PEO Series

Rachel Radomski of Harmony Biosciences

13/7/2021

Rachel Radomski is senior director of patient advocacy at Harmony Biosciences. She talks to RARE Revolution about how patient advocacy is at the heart of everything they do and about how proud she is to help elevate the patient voice

The PEO series: meeting the beating hearts behind the RARE brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

I joined Harmony in the early part of 2019, while the company was pre-commercial and very much in ‘start-up mode’ to build our patient advocacy function from the ground up. The most exciting part of this opportunity was Harmony investing so early in the patient communities and incorporating the patient-voice into our work through our advocacy work.
Our advocacy function also exists completely separately from our commercial team, which is very important to me as a patient advocacy professional. I have had the privilege and opportunity to lead Harmony’s work in the narcolepsy and sleep disorder advocacy space, but as we grow and have entered the clinic in two more rare disease areas (Prader-Willi Syndrome and myotonic dystrophy), it has been exceptionally exciting to develop our Advocacy team and build out a footprint in those patient communities.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

No week is the same at Harmony, but a typical week is a mix of engaging externally with our advocacy organisation and non-profit partners combined with working with my internal colleagues as we work to keep patients at the heart of all we do. Of late, some exciting projects have been building and facilitating internal patient advisory boards in disease communities where we are interested in exploring clinical possibilities. I firmly believe you cannot understand any disease until you are living with it, either as a patient or caregiver. These patient advisory boards allow my colleagues at Harmony to understand a person’s journey to diagnosis and learn about their ‘normal’, including daily life, symptom impact, experience with health care professionals, treatments, and clinical trials. By actively listening, we can bring these experiences and the patient-voice into the drug development process.

3.	What were your personal motivations to taking up a role in patient engagement?

I began my professional career working in public policy at the federal level. As a political junkie growing up, it was always my dream to work in Washington, DC and ‘make a difference’ somewhere. When I got to DC and had the opportunity to work in healthcare, specifically the biotechnology and pharmaceutical industry, I found what excited me most about my job was engaging in partnerships with the patient and non-profit community to work together towards a common goal. After about 10 years working in a government affairs role, I was able to transition my career to a new and growing role of patient advocacy and engagement and I have never looked back!

4.	What makes the role of patient engagement officer important to your organisation?

At Harmony, we have five core values, with the first being keep patients at the heart of all we do. I see my role as the literal embodiment of that value. Whether I am working in the field at a non-profit organised event or bringing the patient-voice into an internal project, I am always working with the lens of what matters most to the patient communities we serve. I also think something special I have found at Harmony is that our advocacy function and the patient-voice is valued by senior leadership and we are consistently asked to have an active voice in all levels of our business.

5.	In your role, how do you ensure the patient voice remains central?

I mentioned it before and I will again, but I think it is most important to actively listen to understand and appreciate what makes someone and their disease experience unique in the rare disease community. I also think it is important to remember that people living with rare diseases are often accompanied on their journey by loved ones, caregivers, and other advocates, all of whom bring their own perspectives. I always try to bring that holistic view into my work.

6.	How do you reconcile operational business needs with elevating the patient voice?

Frankly, I do not find this to be a challenge at all at Harmony. Our first core value is keeping patients at the heart of all we do and the patient’s needs are our business needs.

7.	What are the most rewarding aspects of your role?

I think the COVID-19 pandemic has shown me, in all aspects of my life, personal interactions are the most important and meaningful. I recently returned from my first business trip in 15 months where I had an opportunity to visit a family summer camp for children living with Prader-Willi Syndrome (PWS). Spending a day with the kids and families in the PWS community was eye-opening and heart-warming. I feel immensely lucky I get these first-hand experiences, which serve as a daily motivator to keep working on behalf of these rare disease patients and their families.

8.	What is your proudest moment in your career thus far

When I joined Harmony, I wanted to create a patient advocacy function that went above and beyond traditional industry and non-profit partnerships. Harmony leaned into the idea and two amazing annual grant programmes were born- Patients at the Heart in 2019 and Progress at the Heart in 2021. These grants allow for innovative, patient-centric programmes to be funded through a competitive process that also engages cross-functional Harmony employees during the review and selection of winners. To date, Patients at the Heart has awarded $139,000 in funding and this year, Progress at the Heart will award Harmony’s first grants to address disparities, injustice, and inequities in the rare disease and sleep disorder community. I could not be prouder!

9.	What advice would you give someone considering working in the rare disease space?

The rare disease space is diverse, but I have found the communities so cohesive and excited to partner with each other, the umbrella rare disease patient organisations, and industry. I think that is the most important takeaway is breakthroughs, whether they are happening in lessening time to diagnosis, public policy wins like the 21st Century Cures Act, or the successful development and launch of exciting new treatments, are not happening in a silo. We are all in this together and we win when we collaborate!

10.

If you weren’t Patient Engagement Officer of Harmony, what was Plan B? What did your 10-year-old self want to do as a job?

As an avid sports fan, it has always been my not-so-secret dream job to be a sports reporter. In my dreams, I see myself on the side-lines of Monday Night Football or covering my beloved women’s national soccer team as they go for the gold in Tokyo at the Olympics this Summer. Alas, Erin Andrews already has my gig. In all seriousness, I do think someday I will work in the non-profit community for causes that are personally important to me, like cystic fibrosis.

To find out more about the work of Harmony, please visit;
www.harmonybiosciences.com

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Jayne Gershkowitz of Amicus

28/6/2021

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Jayne Gershkowitz has been with Amicus since 2006 and serves as their chief patient advocate. She talks to RARE Revolution about how patient advocacy is embedded in their organisation and about how heartened it makes her to see it being elevated to a key business function in many companies

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

The role of patient advocacy at Amicus is anything but new; it’s been ingrained in our culture since the company’s inception and is one of our foundational, core beliefs. Having a parent advocate at the helm in Chairman and CEO John Crowley has been vital in infusing the passion and the urgency felt every day by individuals and families living with a rare disease.

As Amicus has grown over the years, both in terms of numbers of employees and our global footprint, we are attracting people who either have or want to further their understanding of the patient. They want to understand the lived experience and be able to contribute to that by making a difference. This central function of patient advocacy attracts experienced people from other sized companies or other kinds of corporate cultures.

Over the years, the biggest change I’ve seen with patient advocacy is how it is becoming elevated as a strategic business function akin to other key business functions. Not everywhere, but at many companies. To me, this demonstrates how critical patient advocacy is in the life sciences. That’s what I have always advocated for—this evolution of the field.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

I don’t really have a typical day or a typical week. Variety is what’s typical, and that is part of the challenge, the joy and the excitement.

First and foremost, for my team and for me, is to be available when patients or their caregivers reach out. People with a new diagnosis reach out—and not always with a disease for which the organisation currently has a focus. We speak with parents whose children have been recently diagnosed, partners of affected adults, sometimes extended family and even friends. I also meet with leaders of patient advocacy organisations—it can be part of an international roundtable or a one-to-one conversation. One week might involve an internal executive committee meeting, a strategy session, or spearheading a cross-functional team review of requests for charitable contributions. Another might involve listening to one of our Patient Advisory Boards discuss disease experience, meaningful outcomes and clinical study designs. It might also involve working with my team to finalise an educational resource on gene therapy for rare diseases or presenting at an industry conference on advocacy in clinical trials.

What keeps me engaged is that it's not the same thing all the time and that I continue to learn.

3.	What were your personal motivations to taking up a role in patient engagement?

I started my patient advocacy career chapter after extensive work in nonprofit organisational management, marketing and fund development, including various health and social services, community programming and advocating for adults with disabilities and immigrant populations. Being executive director of a rare disease nonprofit, I embraced being out of my comfort zone—science was not my forte. It was a personal challenge and an extremely rewarding experience learning and becoming entrenched in an area that I was unfamiliar with.

What really motivated me, though, was seeing the effect advocating could have for both the youngest children and adults living with a life-limiting, degenerative disease. I worked hard to increase the size, the scope and the financial health of a long-standing organisation in an incredibly challenging disease area. We supported the families, the research and hope for the future, but had not yet hit a collective stride. It was still building a foundation with vision as the blueprints.

Moving to Amicus in its earliest days kept me in the same disease space but to a grouping of disorders that were more advanced therapeutically and for which more immediate help and hope were possible. We were looking at paradigm-shifting ways of thinking about what might cause - and how you might treat - a disease. It was a heavy educational lift for patients and families, for healthcare providers and for the company. It led to the understanding and shaping of the intrinsic role that advocacy can play in drug development and, initially, clinical-stage programs.

4.	What makes the role of patient engagement officer important to your organisation?

Amicus takes patient advocacy very seriously; it has been embedded in our culture from the very beginning. The fact that my role as chief patient advocate is a C-suite position reporting directly to the CEO shows this. It helps drive our mission-focused behaviours of extraordinary patient dedication and patient-first thinking.

5.	In your role, how do you ensure the patient voice remains central?

The work that we do is, fundamentally, helping people living with a rare disease become their own best advocates. Our credo in patient advocacy – at least how I view it – is being about people, not product.

We always talk about engaging with patient communities in terms of establishing and nurturing relationships and understanding needs. Empowerment is looked at through the lens of sharing education, information and resources where we might identify mutual knowledge gaps. We can then build solutions together to fill these gaps.

Understanding this lived experience helps people feel more empowered in who they are as a patient or caregiver and what is their objective for their relationship or partnership with their healthcare providers. It also helps to energise people to go out there and do more for themselves, for their organisations and for their communities.

6.	How do you reconcile operational business needs with elevating the patient voice?

In my experience, I don’t think there have been many instances where the patient voice and business needs are mutually exclusive. That's partly because of the way we at Amicus have conducted business all these years. If they were mutually exclusive, then one would have a company that was out of touch with the people it is trying to help.

At Amicus, we've always tried to understand the person, the family and the disease community through our Patient & Professional Advocacy function. We have one of the longest standing Patient Advisory Board programs in the industry. We invite patients, families, and caregivers to the company – or, most recently, virtually – for Lunch & Learns and internal roundtables to educate our entire team, across all functions. The patient community is given a forum to have their voices be heard and to speak directly to the people who are developing the clinical studies, designing the support services and striving to ensure access. This minimises the chance to have contradictions between what the patient community wants and what we're trying to achieve on their behalf.

7.	What are the most rewarding aspects of your role?

It has been incredibly rewarding to see what we have built as a company and seeing the difference that can be made to people's lives. To get there, we've done a lot of listening and a lot of learning. It is meeting these people and seeing how they live with their health challenges with such strength and such grace. Those are the lessons that are transferable across so many different aspects of one's life—professionally and personally. And it keeps us grounded. No matter what our successes are, we must think about what success is for each person in each family affected by rare disease and how can we help them achieve it.

8.	What is your proudest moment in your career thus far?

I’ve been privileged to be part of the uptake and growth of patient advocacy as an important corporate strategic function in biotech, and to co-found Professional Patient Advocates in Life Sciences (PPALS), a nonprofit organisation that I’m confident will continue to advance education for people in or wanting to enter patient advocacy, particularly within industry. A particularly proud and probably the most emotional moment for me at Amicus was when we received approval of our first medicine. It proved that all the effort, tenacity and perseverance it took to make this medicine available to patients was well worth it.

9.	What advice would you give someone considering working in the rare disease space?

Strap yourself in for the ride! It's a special place. Most people who start working in the rare disease space stay in it. For those who come to it later, it makes its way into their hearts, and they stay. Know that you're going to be shaken to your core, because of what you learn from people living with rare diseases, which is an honour to witness. If you don’t have the lived experience of those big health challenges, you're going to see things that may well change how you think about life and the needs of people with complex health issues. You also have to be sure that you stay open to all of that, but also keep your objectivity. This industry brings out the best in people. It takes an honest and genuine approach that is maintained in all aspects of our ecosystem. I think advocacy can help to influence and shape the way people think in every single function of the business.

10.

If you weren’t Chief Patient Advocate of Amicus, what was Plan B? What did your 10-year-old self want to do as a job?

My Plan B would probably have been corporate communications or working at a PR agency. As a teenager, I always I wanted to do something with writing and communication. I knew I wanted to be a journalist; I got my degree in journalism and, for a short while did just that. I enjoyed using my curiosity to ask questions, write and convey tough topics. Letting somebody else tell their story or share their passion and then relaying that to others made it interesting for me and validating for them. I always enjoyed that, and I don't think I'm doing anything that's too far off from that now. Except now, as a patient advocate, I can echo their voice, make sure others hear it, and with dedication and resolve, help bring something back to them that potentially can improve their health and their lives.

To find out more about the work of Amicus, please visit;
www.amicusrx.com

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Andrew Bolan of ProQR

10/5/2021

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Andrew Bolan is the associate director for patient & medical community engagement at ProQR, and regards his role as bridging the gap between the patient's lived experience and the science lab. He talks to RARE Revolution about the exciting times we are living in, in terms of scientific development, and the crucial role the patient plays in driving these advancements

PEO series: Meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

Patient engagement has been a part of the ProQR organisational DNA since its founding in 2012. ProQR was founded by our current CEO, Daniel A. de Boer. A few years prior to this Daniel and his family were faced with the diagnosis of cystic fibrosis for his newborn son; an incurable rare genetic disease. Daniel sought help from experts in the field of drug development and formed ProQR to help people like his son.

All ‘ProQRians’ remain passionate advocates for the rare disease community and are determined to make a difference in the world. To achieve this goal, ProQR strives to integrate the patient voice into our decision-making throughout the drug development process as we believe that a patient-focused strategy is crucial to our success.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

Being the associate director for patient & medical community engagement I work across the organisation in a range of projects. From medical affairs discussions about our scientific platforms and how we talk about our science, to our clinical programme teams sharing the patient perspective in our protocol development and clinical trial recruitment. I also work very closely with our communications team to feature patient blogs, community statements and accessible educational materials on our webpage. We have a dedicated patient community page which I take great pride in being a one stop shop for anyone wanting to understand how we work at ProQR.

A specific project I am very passionate about is the ProQR Patient & Caregiver Steering Committee. This is a committee, launched in January 2020, which features 10 or so wonderful individuals living with Leber Congenital Amaurosis Type 10, Usher Syndrome Type 2a and Retinitis Pigmentosa. No subject is off the table and it has been a wonderful forum throughout Covid-19 to keep our company feet to the fire. We use the forum to speak about a range of topics, mostly as you can expect, throughout 2020 has been how we can help battle the Covid-19 isolation which has had a big impact on the inherited retinal disease community.

3.	What were your personal motivations to taking up a role in patient engagement?

I have been incredibly lucky to work for some very passionate patient engagement directors throughout my career. Learning from them and understanding that as a patient engagement person in a pharmaceutical company, you have a huge responsibility to listen more than talk. I personally have always enjoyed learning about people and their personal journeys—the fact I can use this in my work to make a company’s patient engagement better is a huge motivation.

Starting at the Association of the British Pharmaceutical Industry (ABPI) in London, I saw the power of the patient voice across healthcare policy. That gave me a real passion to helping bridge the patient community with the life sciences industry bringing forward innovative treatments for areas of high unmet need. I continue to be an ardent believer in harnessing the growing importance of the lived experience of patients to improve and quicken the pace of medical research.

4.	What makes the role of patient engagement officer important to your organisation?

I feel a large responsibility of bringing people who we are ultimately trying to help with our RNA therapies in front of my hard-working scientific colleagues. Bridging our RNA science and petri dishes with people and families outside the laboratory. In pharmaceutical companies, having worked in small, medium, and large organisations, the people living with the conditions you are trying to find solutions for sometimes are left out of the process. A patient engagement role really has an external and internal mandate to connect the real world with that of the scientific workspaces.

At ProQR I have really worked hard to bring those families into our weekly work. Since I joined in November 2019, we have hosted over 20 community speakers, many on Zoom due to the Covid-19 situation. However, it is not just community speakers which play a role in patient engagement. It is the daily, weekly, and monthly discussions with advocacy organisations who are working hard on health policy changes to increase genetic testing in different countries to make sure when therapies and solutions come along, people have accessibility to them.

5.	In your role, how do you ensure the patient voice remains central?

Unlike other companies I have worked for, ProQR gives me access to all levels of the company from senior management to clinical team colleagues daily. Regularly updating all levels of ProQR about the importance of the patient voice and how it can improve what we are doing, speeds up our clinical research and helps with clinical trial participation.

In my role I get to work with wonderful patient advocates, patient organisations and our own ProQR Patient & Caregiver Steering Committee. Making sure their feedback is central to all updates I give to our clinical teams, regulatory and medical discussions. Working in inherited retinal disease being a sighted person, I must work closely with the community to make sure I am representing their voice correctly.

6.	How do you reconcile operational business needs with elevating the patient voice?

Being founded as a company by Daniel, following the diagnosis of cystic fibrosis in his newborn son, the patient perspective is really elevated higher than I have seen it in any of the other pharmaceutical company I have worked in. Alongside this is working closely with the patient community to bring educational awareness to inherited retinal diseases (IRDs), which is a real win-win in helping people understand the big scientific paradigm shift happening in the disease space.

7.	What are the most rewarding aspects of your role?

Getting to work with such passionate and dedicated patient advocates and individuals living with rare conditions. Being a non-scientific person, I studied politics at Swansea University, I see a big aspect of my work to showcase the fantastic genomic science we are doing in a language anyone can understand.

We are living in quite an amazing time for new treatments, techniques, and genomic science. However, this is not worth a lot if we don’t bring people along with it.

8.	What is your proudest moment in your career thus far?

That is a tough question. Probably helping my parents understand what RNA (Ribonucleic acid) stands for and how much of a crucial role it plays in protein development. It took a few cups of tea over Zoom to get them there!

I am very lucky to have worked across a wide range of conditions in my career to date, from asthma and chronic lung diseases, the central nervous system, Alzheimer’s Disease and IRDs. I would not be able to pin down a single proud moment, as I think the more I can do as a non-scientist to bring the impact of genomic level research to the public is an achievement every day. If I can help someone navigate what an RNA future therapy might mean for them, then I am very happy.

9.	What advice would you give someone considering working in the rare disease space?

I find the rare disease space one of the most powerful spaces to work in. I would encourage anyone who has a passion for hard work, tough discussions and a get stuck in attitude to really think about a career, role or time supporting the rare disease community. As the advancements in science continue, more and more treatments are coming for some of the hardest and rarest conditions. It is a very exciting time to get involved.

10.

If you weren’t Patient Engagement Officer of ProQR, what was Plan B? What did your 10-year-old self-want to do as a job?

If I could turn back the clock, I think my plan B would definitely have been either a professional cricketer, striding out to a packed house at Lord’s to face the Australians in the Ashes, or singing in front of a packed Albert Hall covering big band classics like ‘For once in my life’ and ‘Beyond the Sea.’

To find out more about the work of Pro QR, please visit;
www.proqr.com

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Naomi Litchfield of Bionical Emas

29/4/2021

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With a desire to help and advocate for patients from a young age, Naomi Litchfield began her career as a paediatric nurse. She brings this invaluable understanding and insight, along with experience of clinical research and rare disease, to her current position as global patient advocacy lead at Bionical Emas. Her dedication to patient advocacy is clear as she talks to RARE Revolution about the importance of her role within the company and in early access programs, and how she ensures the patient voice remains at the heart of everything they do

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

When the Early Access Programs (EAP) division of Bionical Emas was started over three years ago, we began the story with the patient advocacy department. We believed patient centricity was the right foundation for our business model. Three years later, I run a global advocacy department and we are EAP industry leaders. I hope to take advocacy in EAP to the next level now. Increase our thought leadership awareness, educate and support more global patient organisations and ultimately ensure the patient voice is always represented throughout our early access programs and that they are designed with the patient in mind.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

Every week is different which I love, always working with our advocacy partners on different projects. The most rewarding part of my role is working with rare families and giving them a platform to voice their unique stories. I invite a rare patient, a caregiver or a healthcare professional into Bionical Emas on a regular basis to speak to our company about the impact of their condition on their daily lives and their thoughts/experience on access issues. We have all learnt so much from listening to these powerful stories and having the opportunity to ask questions to influence the design and delivery of our programmes.

One of the projects I am currently working on is a video to explain ‘What is an EAP?’ for patient organisations, healthcare professionals and industry. It is a complex area with different terminology and regulations in each country. Our aim is to create a simple animation video to help people new to early access understand the pathway and purpose of an EAP.

Another exciting project I am absolutely honoured to collaborate on is with Medics for Rare Diseases creating a clinical trial and early access lesson for their 101-training programme.
Daily, I work closely with the EAP operational team to enable patient voice representation within our programmes. This could be training on a disease area and product delivery using my nursing background, discussing real world data collection points relevant to that condition, programme communication plans etc. Where relevant, I also work closely with our sponsor’s patient advocacy representatives to discuss our programmes and define advocacy activities.

3.	What were your personal motivations to taking up a role in patient engagement?

Naomi nursing at GOSH

My brother Ben died of a rare condition and my sister Bryony survived four rare cancers. I watched Bryony grow up in hospital and knew from a young age I wanted to be a paediatric nurse and advocate for patients.

I have had the privilege of caring for many rare patients and their families in Oxford Children’s Hospital where I trained and Great Ormond Street Hospital where I became a senior clinical research nurse caring for rare patients participating in clinical trials and early access programs. I have administered many different investigational medicines and seen the real impact they can have on patient’s lives.

I cherish my 15 years nursing and it has given me a unique foundation compounded by my personal experiences to now passionately advocate for patients and families in my role.

4.	What makes the role of patient engagement officer important to your organisation?

Our mission is to positively impact the lives of patients and their families by bringing life-changing medicines to patients all around the world. My role is at the heart of this mission and I am proud to drive it every day and work with such a caring and dedicated team.

The positive impact on the patient is integrated into all our EAP functions from design to delivery. We understand the importance of being informed of the patient community history and the individual patient’s condition.

We really do understand there is often an urgent need for access to investigational medicines and because of this there is a need for transparent, timely and compliant communication with all stakeholders throughout our programs.

5.	In your role, how do you ensure the patient voice remains central?

Ensuring the patient voice remains central to all our work is something I am extremely passionate about. I educate and discuss personal and professional case studies (with consent) but the most powerful and beneficial impact on our team comes from rare patients and families themselves which is why I regularly invite them to speak to our team so they can listen, learn and engage.

I am proud to be continuously driving our company mission, supporting our teams to feel knowledgeable, empowered and to really understand the conditions of the patients on our early access programs and how important the impact of access to the investigational medicine can be.

We have a feedback wall in all our office locations around the world that showcases positive feedback from healthcare professionals and the impact the programs have had on their patients. The team find it extremely rewarding to read these messages and I believe this to be vital to drive our team to regularly show them the positive impact their work is having on patients and their families' lives.

6.	How do you reconcile operational business needs with elevating the patient voice?

The two things aren’t mutually exclusive. Our mission of ‘positively impacting the lives of patients and families by bringing life-changing medicines to patients all around the world’ is central to everything we do and drives our business/operational behaviours. We know that if we are truly winning for patients, then the rest takes care of itself. In simple terms, we have created an amazing business by doing great things for patients.

7.	What are the most rewarding aspects of your role?

Working with rare patients and their families, educating on rare conditions and access to investigational medicines to engaged groups both internally and externally.

8.	What is your proudest moment in your career thus far?

Administering a phase one investigational medicine to the first UK rare patient and seeing the positive impact it had immediately on that patient and their family. I will never forget that moment and the effect it had on the family, my team and myself.

9.	What advice would you give someone considering working in the rare disease space?

Go for it! Being part of the rare disease community is so rewarding, challenging, exciting, and emotional. Speak to people working in rare you will soon see what a great opportunity it is. I feel very privileged to work with such inspirational people and really have a positive impact on rare families lives.

10.

If you weren’t Global Patient Advocacy Lead at Bionical Emas what was Plan B? What did your 10-year-old self want to do as a job?

For as long as I can remember I wanted to be a paediatric nurse, it was my dream job and it came true. However, when I was 10 I didn’t know patient advocacy roles existed and now I have had the opportunity of experiencing both, I’m delighted to say my current advocacy role is the best job I’ve had for many reasons, but mainly because I truly believe I positively impact more rare patients and families.

To find out more about the work of Bionical Emas, please visit;
www.bionicalemas.com

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Nikki Backus and Emma Murphy of The CATS Foundation

30/3/2021

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Having been supported by The CATS Foundation through their own personal grief, Nikki Backus and Emma Murphy know the importance and power of such support. Both women want to give back to the charity through their patient engagement roles and help other families, drawing on their own lived experience

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

Nikki
The Family Support Officer role was created in 2014 when I came onboard for the new
position. I’ve been in the role in varying degrees since then, but the role really evolved in early 2020 when the team expanded to include my colleague Emma. I think we make a great team and work well within the dynamics of the other charity personnel.

Emma
We have had a family support officer for eight years now. I started my position in March 2020 to work alongside Nikki in the role. Since joining at the beginning of the pandemic we have started providing a lot more support online. Through coffee and chat sessions, Christmas events, awareness week and a remembrance event all via zoom. This works well as we are all very spread out across the UK. We hope to build a strong network of support for families not only virtually but through face-to-face meet ups again when covid allows. The group coffee and chats, and the one-to-one chats really help us to feed back personal experiences to help shape better care in the future for patients.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

Nikki
Each week is a little different and changes with the needs of the charity and the families we support. Typically, though it will include:

Contact with families – checking in one to one or group activities such as our fortnightly CATS coffee chat zoom call.
Administrative tasks such as updating contact spreadsheets, creating services surveys and monitoring feedback, sending out news via emails.
Planning new projects for the upcoming months.
Working on ongoing tasks such as sending out birthday cards and gifts.
Working on upcoming/annual events such as the recent Remembrance day, Christmas zoom party and our awareness week. These events are so worthwhile as they engage the community and raise awareness, but they take an awful lot of planning and preparation in the background.

Emma
In a typical week I will reach out to families individually to see if there is anything they need help with and to make sure things are okay.
I spend time sourcing equipment that families may need that is tricky to get or not available on the NHS.
We send out handmade cards to families to celebrate children's birthdays and to remember children that have passed away. I love this personal touch and enjoy making the cards.
We have also been busy recently sending out care packages to families isolated by the pandemic and those who have been stuck in hospital with their children.
I speak with the rest of the team a lot and we are constantly thinking of new ideas and ways of connecting with and supporting families.

3.	What were your personal motivations to taking up a role in patient engagement?

Nikki
The CATS Foundation supported my daughter Ruby who suffered from Sandhoff Disease. They were a lifeline for us and I wanted to give back to the charity and the wider community after Ruby passed away. I was honoured to take on the Family Support Officer role when approached by CATS.

Emma
My son Stanley was diagnosed with Tay Sachs disease in 2014 and sadly passed away from the illness in June 2019 at just four years old.
All I want to do now is support others going through similar heartache, help to improve experiences with my own firsthand experience and push forward in helping to find a cure for these awful diseases.

4.	What makes the role of patient engagement officer important to your organisation?

Nikki
The CATS Foundation does some extremely important work alongside professional bodies and organisations towards research and a future treatment for Tay-Sachs and Sandhoff Disease. It also strives to improve the patient experience within the medical community, but fundamentally the charity is all about family. We support families from diagnosis through the course of the disease with advice, a supportive shoulder, we connect families together providing a support system and help in many, many practical ways. All of this is coordinated with the help of the Family Support team.

Emma
I believe the role is essential to every part of what we do; supporting and empowering caregivers and patients is at the root of everything we do.

5.	In your role, how do you ensure the patient voice remains central?

Nikki
We ask for feedback from our families to always strive to improve our services. We constantly try to offer more support, in different ways year on year.

Emma
The charity is all about the patient and making their journey as easy and as comfortable as it can possibly be. Feeding back the patients’ needs and wants is key to moving forward and learning and growing.

6.	How do you reconcile operational business needs with elevating the patient voice?

Nikki
The patient voice is key and central to our efforts, while operational needs are essential to the smooth momentum of the charity, and they are executed around the support services.

Emma
Since we are focused on the patients’ needs, the operational business needs has to fit in with reaching out to patients and gathering their thoughts and ideas on how things can be improved.

7.	What are the most rewarding aspects of your role?

Nikki
Knowing that I helped make a difference to a family affected by these diseases.

Emma
I love to empower and encourage the patients and caregivers to use their voice and make sure they push to be the invaluable part of the medical team around them.
Giving people that confidence to speak up is so rewarding.

8.	What is your proudest moment in your career thus far?

Nikki
Not just one, but many. Helping to organise the European conference, delivering the online Christmas party, creating a Remembrance video honouring our children who have passed…. helping families in honour of my daughter Ruby.

Emma
I feel immensely proud to be a part of our incredible families lives every day.
I am proud to be part of the rare disease community.

9.	What advice would you give someone considering working in the rare disease space?

Nikki
The Rare disease community needs warriors and while it can be challenging to make our voices heard the rewards are knowing that all our small steps forward will one day make a huge difference to the life of a child diagnosed with a rare disease.

Emma
The whole rare disease community is full of such inspirational and proactive people, to be surrounded by such passion and positivity can only enhance your life.
My advice would be to do it, you will not regret it.

10.

If you weren’t Patient Engagement Officer of The CATS Foundation, what was Plan B? What did your 10-year-old self want to do as a job?

Nikki
My 10-year-old self didn’t really have a plan! While I enjoyed my former IT career I can honestly say working with families at a time when they need support the most, understanding how they feel as it wasn’t that long ago that I was in their position, making a difference, no matter how small, is so much more rewarding.

Emma
I have always wanted to help people since a young age. Before Stanley was diagnosed with Tay Sachs disease and became poorly I worked as a family support worker for Action For Children, which I feel helped to prepare me for this role in so many ways.

To find out more about The CATS Foundation please visit;
www.cats-foundation.org

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Maysa Ghammachi of Genpharm

30/3/2021

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Maysa Ghammachi's role as patient executive officer at Genpharm is a newly evolved role and very much a first for the region. Maysa provides valuable support to patient organisations and through awareness campaigns and events, works hard to break down barriers and tackle the taboos surrounding rare disease in the region

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

The role of patient engagement isn’t only fairly new at Genpharm but it is the first patient advocacy role within a pharma company in the region. However, with one of our core values being “We always put patients first”, patient engagement has always been at the centre of our day-to-day actions. As the organisation has grown with time so has our resources and networking. This has given us the possibility to tackle more projects and collaborations. With the trend of digital healthcare expanding, I can envision Genpharm joining that movement. Our future project to tackle: How can the digital health tech space be used for patient advocacy?

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

With the rare disease patient associations in the region being in their infancy, part of my role entails providing support and resources. I get in touch with our partners abroad, such as in the US and Europe, and use our networking to bring tools to them. We had the opportunity in the past to collaborate with Treat NMD. We collaborated by bringing to the region and translating their neuromuscular diseases material into Arabic. Recently diagnosed families were given the resources once diagnosis was made by their physicians. Another big aspect is to use our current social platforms and create campaigns to raise awareness of rare diseases.

3.	What were your personal motivations to taking up a role in patient engagement?

Coming from a digital creative background, I was taught that design is not only about aesthetics, but it is about function and purpose. From there the idea of purpose evolved and led me unconsciously towards the role I hold today. My position allows me to use my creativity but with a bigger purpose. I know that with each awareness campaign, project or event we are one step closer to changing someone’s life. That is my purpose.

4.	What makes the role of patient engagement officer important to your organisation?

Globally, there is often a predisposed prejudice attached to pharmaceutical organisations. I believe that a PEO’s role is to break this. Patients and biotech companies are closely intertwined; one needs the other. We need to take into consideration patient needs to be able to provide correct support and treatments. When we look at our region in the MENA, rare diseases are still considered taboo in many corners of our society. Part of having the role of PEO at Genpharm is to break down these barriers and obstacles. By raising awareness and educating, we are able to open the conversation.

5.	In your role, how do you ensure the patient voice remains central?

A simple thing that can be overlooked is the importance of listening. I feel taking the time to listen to families and their stories plays a big role in keeping patient centricity. The more you take time to understand what the family has gone through, where they currently are and what changes they need, the more likely you are able to give the support needed. From there, we are able to identify the correct platforms to give them a voice as well as guide them to support centres. An example is our recent collaboration with a Rare Revolution Magazine and MENA Spotlight Edition which includes interviews with families as well as the patient associations.

6.	How do you reconcile operational business needs with elevating the patient voice?

By raising rare disease awareness and supporting our community, we aim to facilitate the knowledge and diagnosis pathway of rare disease patients and their families. It undoubtably is a wholesome alliance.

7.	What are the most rewarding aspects of your role?

The most rewarding aspect of my role is knowing that however big the part I played, someone’s life was changed for the better. The feeling is indescribable especially once you are able to see the results and direct impact of your actions.

8.	What is your proudest moment in your career thus far?

My proudest moment so far in my career is being able to be a part of this journey. I am proud to be able to work in an organisation who makes promises of change and delivers them. An example is a SMA educational initiative that Genpharm launched. Its purpose was to visit local schools and educate teachers on the early signs of SMA.

9.	What advice would you give someone considering working in the rare disease space?

My advice is to not think twice. Join and join now the rare disease journey. It is a long one but with committed efforts we can shorten it together.

10.

If you weren’t Patient Engagement Officer of Genpharm, what was Plan B? What did your 10-year-old self want to do as a job?

It is a hard question to answer as I believe my role as a PEO has been an ongoing and evolving journey. From my early beginnings, we had internally identified a need for the patients to have a dedicated voice. Filling that gap has been a natural transition but nevertheless almost predetermined.

Growing up, I always was interested in being a doctor or a lawyer. I feel by being the patient executive officer at Genpharm, I have a middle ground between both. I closely work with the health sector and by being an advocate, I am able to lend a voice to support those who need it the most.

To find out more about the work of Genpharm please visit;
www.genpharmservices.com

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Sue Krug of Soft Bones

18/3/2021

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Sue Krug, patient engagement officer of Soft Bones, knows all too well the fear that can come with a rare disease diagnosis. In her role she draws on her own lived experience with rare disease to help alleviate the fears of patients and their families and supports them throughout their rare disease journey, empowering them with knowledge and information

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

The patient engagement role has been part of the fibre of Soft Bones since it was founded more than 10 years ago. My role started as a friendly welcoming face to hypophosphatasia (HPP) for newly diagnosed and joining members. Now it has evolved to helping patients and families learn about the huge spectrum of symptoms of HPP and connecting them with other staff at Soft Bones and our various resources.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

There is no ‘typical’ week at Soft Bones, which is what I love about my job. One week, I may have a long conversation with a newly diagnosed patient who has been searching many years for an answer to all their symptoms that did not seem to connect until now. Then another day, I may help a scared parent understand what this diagnosis could mean for their unborn child. Throughout the variety of patient engagements, one constant is my role of calming their fears and helping them to know they are not alone in their journey.

3.	What were your personal motivations to taking up a role in patient engagement?

I’m motivated to help patients from feeling lost and alone. Having a rare disease (hypophosphatasia) myself, I understand their fears about feeling like the only one in the world with a condition. My motivation to stay in this role is seeing patients grow in confidence and knowledge of their rare disease, and learning to self-advocate for themselves with their doctors.

4.	What makes the role of patient engagement officer important to your organisation?

One of the most important roles of any patient advocacy organisation is to empower patients with information to allow them to advocate for themselves. At Soft Bones, we aren’t just here at the point of diagnosis, but we partner with patients along their entire journeys.

5.	In your role, how do you ensure the patient voice remains central?

By being a good listener, showing patience, and engaging in conversation. Since our HPP patients have a variety of needs, we have created eight regions in the U.S. that allow us to connect on a local level to provide more in-person opportunities to connect. Soft Bones is a nimble organisation and this flexibility allows us to re-prioritise as needed to ensure we continually provide value to our patients.

6.	How do you reconcile operational business needs with elevating the patient voice?

Our approach has always been to elevate the patient voice first order of business, and we find that if you put the patient first, the operations side always seems to fall into place.

7.	What are the most rewarding aspects of your role?

The most rewarding aspect of my role is when I see a patient, who a few months ago was filled with fear and searching for answers, transformed into an active member of the HPP community. It’s especially rewarding when I see a patient ‘paying it forward’ with knowledge that we gave them, and seeing them armed with new information and confidence where they are able to help out someone else in our community.

8.	What is your proudest moment in your career thus far?

It is not uncommon in my role to have situations where severe newborns can be in a life-threatening state where every minute counts. Seeing a severe newborn turn the corner and thrive because our Soft Bones team helped them to quickly connect, get a diagnosis and access to treatment is an incredible feeling. What can be better than that?!

9.	What advice would you give someone considering working in the rare disease space?

A few pieces of advice I would offer to someone working in rare: first, take nothing personally. Emotions can run high as patients often feel misunderstood or have had doctors think they are faking symptoms because they haven’t been able to find a cause.

Another insight, the rare disease community is very personal and we connect by sharing experiences. So, willingness to share experiences—or to connect in some way that is similar—is very important.

Compassion and empathy, even if you have never been in their shoes, is important. We are very close with each other. Because we are small, I know almost every member of our community, and since I have developed relationships, many times I work at odd hours because I feel the need to be there for them. But even with this challenge, working with the rare community is extremely rewarding as long as you make time for self-care.

10.

If you weren’t Patient Engagement Officer of Soft Bones, what was Plan B? What did your 10-year-old self want to do as a job?

If I wasn’t working at Soft Bones, my Plan B was to teach computer software to seniors. I still do this as a hobby. When I was 10 years old, I wanted to be a forest ranger and smoke jumper. In many ways, I’m navigating the wild frontier of rare diseases and putting out fires every day, so I have no regrets.

To find out more about the work of Softbones please visit;
www.softbones.org

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Asya Choudry of Breaking Down Barriers

17/3/2021

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Asya Choudry is proud to focus on equality and diversity in her role as the community engagement manager for Alstrom Syndrome UK through the Breaking Down Barriers project and talks to RARE Revolution about just some of the projects she is involved with. With an unwavering passion and resolute commitment to making a difference in the rare disease space, Asya says herself, that the best is yet to come!

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

I have been the community engagement manager for Alstrom Syndrome UK (ASUK), working on the Breaking Down Barriers (BDB) project for nearly three years now. ASUK have always had a patient engagement role that was created when it was founded in 1998. My role focusses on helping our member organisations create bespoke projects using the funding provided as part of the BDB project. I support organisations to improve engagement with BAME communities, improve access to genetic services, and help them facilitate the project into fruition and evaluation.

The role has developed and includes more educational and consultancy areas, mainly working within equality and diversity and reducing health inequalities. Working for a small organisation allows you to have a lot of flexibility, working in different areas and making suggestions for which direction we can take. I really enjoy it when I can help with things that I am really passionate about and can really get stuck into.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

As I work for Breaking Down Barriers three days a week and for Ehlers-Danlos Support UK (EDS-UK) as their helpline and medical liaison manager for two days, my week is fairly action packed. On a standard week in BDB, I’m usually having meetings with our member organisations on different aspects of their projects and supporting them through any difficulties. I also provide feedback and review their action plans or evaluations. In general, it’s a very creative role as you are always having to think on your feet and suggest new ideas for the organisation considering their size, demographic and who they are trying to engage from diverse communities. Otherwise, it’s speaking at events to raise awareness of BDB or doing the background work that every organisation needs to help everything tick along.

My other position within EDS-UK is to manage their national helpline supporting those living with a connective tissue disorder called Ehlers-Danlos syndrome. The helpline is not only used by patients looking for support but is also used by professionals hoping to understand more about their patients. I also train healthcare professionals on how to manage patients with EDS and host webinars with medical professionals who are experts in EDS to help educate the community on specific areas of the condition.

One Project I can speak about is the ‘Let’s Talk Diversity’ campaign we are doing as part of BDB. It is something I am very passionate about and I am so happy that it is finally in full swing. The campaign is about interviewing professionals and patients to discuss what diversity means to them. Any professional or person with lived experience who is either from a BAME background or works in the diversity sector, we would love to hear from you! It’s all about exploring their personal experiences growing up, why they chose to work in this field and what changes organisations can make to improve inclusivity. It’s real conversations with real people. I believe it is through these stories that we can impact change of behaviour in our work culture to become more inclusive internally and externally within the diverse communities we serve.

3.	What were your personal motivations to taking up a role in patient engagement?

I had a brother who passed away at the age of six from a genetic condition. My mum had a bad experience with the health professionals at the time and felt she was blamed for her son’s condition. This stopped her from seeking support. This motivated me to learn about genetics and how to communicate the chance of inheritance effectively. No-one should be made to feel how my mum felt, hence why the need for cultural competency amongst health professionals is paramount, as it can be the difference between families feeling heard, supported and engaged.

4.	What makes the role of patient engagement officer important to your organisation?

I would say it is important because I’m the point of contact for our member organisations when it comes to their issues, needs or cultural understanding. Alongside help with facilitating their BDB project. We are here to keep things on track and to help make sure BAME communities needs are addressed and services are adjusted adequately to support them. We are supporting organisations who are working in patient engagement roles to ensure the support they offer is inclusive and helping them to develop the skills needed to support diverse communities.

5.	In your role, how do you ensure the patient voice remains central?

I think this is not isolated to my role, but it is part of the ethos of the organisations I work for. At ASUK and within BDB, we have patients that are part of our advisory board and trustee board, which keeps the patient voice at the central heart of our projects and our learning. We also have patient advocates who are always invited to be a part of our events, conferences and webinars. We always ask each member organisation at the start of their journey how they know a certain project is needed within their organisation and how their members are involved in the planning, monitoring and evaluation of projects. BDB collates examples of good practice from the projects that we fund, and we always encourage quotes and feedback from the communities they serve to form parts of their evaluation so that we can monitor impact.

It is the people and communities that we are all doing this for. They are the ones that determine the success or learning from each project and they help inform how we adjust our projects and funding activities. Patient voice/involvement is and always will be a key priority for ASUK and BDB. My other role within EDS-UK also incorporates these ideas by having patient representatives as ambassadors and volunteers running the support groups in all represented regions.

6.	How do you reconcile operational business needs with elevating the patient voice?

At ASUK we have worked hard to establish a representative and diverse board of trustees in terms of demographics and lived experience. We also conduct a skills audit to ensure we have a positive combination of people and skills to help drive the charity forward and succeed. The needs of our members will always be a key priority and the only way we can determine need is by listening to individual and collective voices and using this to shape our organisational strategy and the services we provide.

7.	What are the most rewarding aspects of your role?

Oh there are far too many to note, so I will do my best! As I mentioned I have a personal passion and volition to working in this field so I am far too biased as it is. I love the variety of conditions that I work with and having the continuous challenge of creating new projects and engaging new communities.

I love learning about new faiths, cultures, celebrations and practices. We recently did a social media campaign called ‘share your colours, share your culture’ for Rare Disease Day, where we had patients, professionals and patient organisation representatives take part in talking about what their culture means to them. I also love the fact that I get to use my lived experience as a British Pakistani Muslim who speaks some of the South Asian languages, to help guide cultural understanding and education.

The best part of my job is knowing that you are making a difference to people’s lives in so many ways. Either through improving services or ease of access, or just by giving communities confidence to uptake what the charities are offering. Every family or community that has children who are living with a genetic condition have a lot of hardship in numerous areas of life, if we can improve even one aspect for them, that’s a life well lived. I am also thankful to have amazing colleagues, who trust and support each other, it is this respect that helps us to be productive with our aims of wanting to help people and improve services.

8.	What is your proudest moment in your career thus far?

I am proud of all the organisations that we support, and the hard work and determination that they have put in. I feel proud of their success and achievements. I am also proud of the charity that my family run called Mushtaq Welfare Trust, which offers a UK standard of education to orphaned girls in a village in Pakistan.

I feel that the proudest moment of my career hasn’t happened yet, so watch this space! I have big dreams, so I’m hoping to continue working hard to do some good, and I have faith that the best is yet to come.

9.	What advice would you give someone considering working in the rare disease space?

I would say to keep an open mind and to always remember that there is more that connects us than divides us. Working in rare disease is all about kindness, understanding of others situations and helping improve your little area of work for others, to offer equal access to all. It is important to consider that your staff and organisation should reflect the community that it serves, as this in-house understanding will be beneficial to staff as well as members.

10.

If you weren’t Patient Engagement Officer of Breaking Down Barriers, what was Plan B? What did your 10-year-old self want to do as a job?

I went through quite a few phases before I decided on genetics when I was 16 years old. When I was a little, maybe seven or eight years old, I wanted to be Ricki lake, a chat show host, and help people with their life problems. I was very committed to this idea as I’d walk up and down the windowsill with my plastic microphone (that was actually a pencil case) and give my advice to help people with their marital problems.

In my early teens I then wanted to work in the police force as a criminal profiler, because I liked a detectives’ job in a television show called ‘The Bill.’ Otherwise, I was mostly interested in advertising and marketing. My friends and family would always say that I could sell ice to an Eskimo, and I was always coming up with creative ideas and jingles.

Projecting forward, my end goal would be to work in reducing health inequalities and Equality, Diversity and Inclusion, maybe even for the WHO or UN and here’s to dreaming, becoming Health Secretary!

To find out more about the work of Breaking Down Barriers please visit;
www.breaking-down-barriers.org.uk
If you would like to contact Asya about any of the projects she is involved in, or to learn more, please contact;
Asya.choudry@alstrom.org.uk

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Mileva Repasky of CDCN

10/3/2021

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Mileva Repasky started out on her rare disease journey when her daughter was diagnosed with Castleman disease. With a long-held desire to help others, she is dedicated not only to helping her daughter but others like her and their families, through her tireless work and commitment to the CDCN

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

The patient engagement role has existed in some capacity since the CDCN’s inception in 2012. Over the last seven years I have served as the chief patient officer and proudly advocated for our community. Patients are at the heart of everything we do, and as an organisation we strive to support, empower, and educate those affected by Castleman disease (CD) through both in-person and online resources to battle their CD, but also have a voice in their fight.

The CDCN has spearheaded a revolutionary approach to rare disease research with an eight-step model, where the patient community is prioritised when it comes to setting the research agenda. We recently launched the All In Movement, which is an initiative to crowdsource and prioritise high-impact questions from our community of patients, loved ones, physicians, and researchers to guide the next generation of studies on Castleman disease. This type of collaboration will be increasingly important going forward. In order to continue being the best resource for patients I also want to continuously gain a better understanding of our patients’ needs and experiences so that I can advocate for our community to the best of my ability.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

A typical week for me consists of 30+ hours addressing the needs of our patients and their families, engaging with various stakeholders in our community, and working alongside our team on various projects to drive forward our life-saving research. This takes the form of answering emails and phone calls when patients and love ones reach out to the CDCN for help. I’m always so glad that they find us and are then connected with a group that can make a difference to help their situation. Sometimes it’s about finding the closest specialist/physician to a patient, gaining insight on published literature on treatments and diagnosis to share with their doctors, and connecting for emotional support.

We have a partnership with the Chan Zuckerberg Initiative to advance drug repurposing for rare diseases. I am serving as the patient engagement lead to ensure that the voice of our patients are at the centre of all we do, as we work to share the lessons we have learned while navigating the repurposing landscape with other rare disease organisations by creating a guide for others to follow.

3.	What were your personal motivations to taking up a role in patient engagement?

When my daughter Katie was around 18 months old, we started down a road of so many unknowns to find a diagnosis for what she was battling. We received the diagnosis of Castleman disease before her second birthday. I had no idea what the road ahead would be but what I did know was that I would fight beside her to find a cure–and that led me to finding the CDCN.

Over the past eight years we have watched her endure more than any child ever should, all while feeling like we are in the dark with so many questions and so few answers. With the diagnosis I knew that I had to dedicate my life to doing my part, which is helping patients and their loved ones who are going down the same road we were on. I will continue to dedicate my life to support our patients, their loved ones, and fight alongside them and our amazing team. I know that we will find a cure in my daughter’s lifetime!

4.	What makes the role of patient engagement officer important to your organisation?

This role is so important to our organisation as patients are truly at the heart of everything we do. The patient perspective is a vital piece of the puzzle of Castleman disease. This role allows me to advocate the needs of our patients while also facilitating the needs of the organisation allowing our patients to have a voice in all that we do.

5.	In your role, how do you ensure the patient voice remains central?

My role gives me the ability to connect with our community and create meaningful change that will continue to benefit them. I am constantly in communication with our patients and their families, to provide them with the resources they need to fight their battle while also sharing new published papers, research updates, and ways they can join in the fight.

We have a Warrior community that connects virtually throughout the year and in person each summer at our Patient and Loved One Summit (although we have done this virtually in 2020 and 2021). We have an amazing group of patients that are also dedicated to contributing to a cure, however they can–and it looks a bit different for everyone! Some patients contribute blood and tissue samples for research, some register in our natural history study, others fundraise and spread awareness, and some do it all!

6.	How do you reconcile operational business needs with elevating the patient voice?

Our operational business needs are one and the same as helping our patients and their families. We exist to do the research that will cure Castleman disease and help patients live full lives. We can’t do this high-impact research without patients donating their samples, medical history, and perspectives to shape the agenda.

7.	What are the most rewarding aspects of your role?

The most rewarding aspect of my role is the ability to help patients and their families on their journey with Castleman disease. The feeling I had when my daughter was diagnosed with Castleman disease was awful–the medical unknowns and overwhelming lack of information was crippling. Now having the opportunity to support patients and their families so they do not have to experience that same feeling brings so much joy and purpose to my life.

I meet so many patients and their families in person and get regular updates on how they are doing which is also so wonderful. I love to hear treatment success stories and when things are going well. But the low points also continue to drive me to continue this mission–there is always more work to do.

8.	What is your proudest moment in your career thus far?

This is a difficult question as there have been so many moments in my career that I am proud of, but all revolve around our patients and the success of our team. A few of my favourite are attending the first Patient and Loved One Summit (after I was in this role) and being able to hug all the people I spent the year talking to and helping. Successfully engaging a group of our patients to fundraise enough money to fund a study. Working alongside the Chan Zuckerberg Initiative to expand our collaborative approach to other rare disease and also our new project on drug repurposing. And being a part of the CORONA project and doing my part to aid in the fight against COVID when the pandemic hit!

One of my proudest patient related moments involved working alongside our team to help a paediatric family whose child was battling idiopathic multicentric Castleman disease with TAFRO (a variant of CD) to get a definitive diagnosis and treatment plan. Having the ability to support them in the hospital and provide resources to them within hours of hearing about this case was so special to me. I remember the moment when I walked into his hospital room and hugged his parents, I could feel their devastation and it just was a very real reminder of why I am doing this!! He is now doing amazing and on the same treatment that my daughter Katie is on!

9.	What advice would you give someone considering working in the rare disease space?

Patients and their families battling a rare disease need organisations to give them a voice and stand beside them. Working in the rare disease space is such a privilege and requires hard work, passion, and dedication to the cause, but it is so rewarding and such an honour.

10.

If you weren’t Patient Engagement Officer of CDCN, what was Plan B? What did your 10-year-old self want to do as a job?

For as long as I can remember I have had this innate desire to help people. I received my bachelors and master’s degree in psychology with the intention of becoming a psychologist. When my daughter was diagnosed, I was able to take my passion and my education and use it to help others suffering with the same disease. It has become the most rewarding job I have ever had as it is allowing me to fight alongside an amazing group of people working every day to find a cure to save my daughter and so many others.

To find out more about the work of the CDCN please visit;
www.cdcn.org

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Juliet Hulse of Illingworth Research Group

4/3/2021

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With a background in nursing, Juliet Hulse followed her passion for research and is the patient engagement officer for Illingworth Research Group. She discusses her responsibility to ensuring a patient centric approach, her commitment to patient groups and the importance of the patient voice in the success of a trial

PEO series: meeting the beating hearts behind the rare brands

1.	How new is the patient engagement role in your organisation, how has it evolved and what are your hopes for the role in the future?

The role is relatively new, however as a patient centric solutions organisation, Illingworth Research Group has always had the patient at the centre of the clinical study they are involved in. My current role now means I can encourage a patient centric process within the planning, design and delivery of a research trial and apply meaningful solutions for patients.

2.	What does a typical week entail for you, and are there any specific projects you can tell us about?

My week starts with a review of current Requests for Proposals (RFP). Some protocols may already mention a remote decentralised approach with off-site visits included in the schedule, however other protocols may need further discussion on remote visits to be included within the patient program. It may be a rare disease indication where a collaborative approach is necessary involving not only mobile research nursing but also our concierge service PatientGo, medical imaging or some technology input.

I oversee a team who will work together to suggest areas where the indication and trial may benefit from the inclusion of decentralised remote visits. Reaching out to patient groups to understand the patient population is imperative. We are working on several Duchenne muscular dystrophy studies currently, and I will often ask the project managers on these studies about how they are progressing and any challenges they may be facing, collecting feedback from advocacy groups to apply to lessons learned. We gain very rewarding feedback from families impacted by our nurses' visits and this is always encouraging to share!

The current pandemic has had a significant impact on patients and their families who are taking part in research trials but who may be shielding and vulnerable. The need to find solutions to enable studies to continue has encouraged regulatory authorities to rethink the design of studies integrating remote visits and telemedicine into studies. I often find myself working across various time zones with several colleagues around the world as far as Australia!

3.	What were your personal motivations to taking up a role in patient engagement?

Quite simply it was my desire to ensure that all patients should have the ability to take part in clinical trials, and to guarantee that we make their journey as straightforward as possible during a study. Paediatric studies are particularly important, roughly 50 percent of rare diseases affect children, 30 percent of whom do not survive past their fifth birthday.

4.	What makes the role of patient engagement officer important to your organisation?

Ensuring Illingworth understands patient engagement and why patient engagement really matters is fundamental to our culture and growth. Engaged patients are more likely to adhere to study requirements, increasing study retention.

Patient engagement is also a strong feature of the professional standards for clinicians enshrined in the International conference of harmonisation – good clinical practice (ICH GCP).

5.	In your role, how do you ensure the patient voice remains central?

Listening to patients, ensuring their involvement in decisions, providing information to patients with explanation and empathy are fundamental.

6.	How do you reconcile operational business needs with elevating the patient voice?

I feel it is important to focus on measurable success from the studies perspective by incorporating the patient into the trial design from the start. The patient voice can contribute to many key areas, and overall improve the delivery of care in the future. Greater recognition of the value of the patient voice will allow a redirection of clinical practice to ensure the priorities of CROs and biotechs are aligned to the needs of those experiencing the disease.

7.	What are the most rewarding aspects of your role?

Feeling that we are really making a difference to a patient who is taking part in a study, listening to their challenges and needs and being able to offer solutions that mitigate some of those challenges.

8.	What is your proudest moment in your career thus far?

Apart from achieving my initial nursing qualifications, speaking at conferences in the US and Sydney gives me a sense of pride.

9.	What advice would you give someone considering working in the rare disease space?

Although hugely challenging at times, working in this area is also very rewarding. Obtaining a deep understanding of the rare disease process can be difficult, families often go through a diagnostic minefield given the clinical and genetic heterogeneity of rare disease. Commitment to patient communities and collaboration within multidisciplinary teams are a driving force behind drug development and provide crucial insight for biotech. Every single patient counts!

10.

If you weren’t a Patient Engagement Officer of Illingworth, what was Plan B? What did your 10-year-old self-want to do as a job?

Obviously the 10-year-old me wanted to be a nurse! I used to wrap up my teddy bears and dolls with bandages and steal my brothers Action man to perform operations on!

I have been fortunate enough to enjoy every aspect of my career from the first day I started my training to my current role. I initially worked on a busy coronary care unit and cardiology ward, but over the years realised my interest lay in research, initially in cardiology trials.

I have been privileged to be able to travel extensively and understand aspects of off-site research nursing globally, including Egypt and Australia. Maybe a travel writer….

To find out more about the work of Illingworth Research Group please visit;
www.illingworthresearch.com

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Rachel Radomski of Harmony Biosciences

Jayne Gershkowitz of Amicus

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Nikki Backus and Emma Murphy of The CATS Foundation

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Sue Krug of Soft Bones

Asya Choudry of Breaking Down Barriers

Mileva Repasky of CDCN

Juliet Hulse of Illingworth Research Group

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Author

Archives

Categories

Rare Revolution is published by NRG Collective, a not-for-profit media company specialising in rare disease contentContact Us​rstewart@rarerevolutionmagazine.com

PEO
Series

Rare Revolution is published by NRG Collective, a not-for-profit media company specialising in rare disease content
Contact Us
rstewart@rarerevolutionmagazine.com