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Con Hennessy of OpenApp

18/9/2020

With a background in IT and the healthcare system, Con Hennessy became CEO of OpenApp in 2017, having joined the company shortly after its inception, in 2002. He talks to RARE Revolution about the importance of patient-centricity and customer focus, and how integral his highly dedicated and collaborative team are to the success of OpenApp

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically OpenApp and what did that journey look like?

My introduction to the world of rare disease was mostly from a technical perspective working with the national health system in Ireland and seeing the significant difference translating a clinical challenge into a usable, long term sustainable technical solution can mean to the system.

OpenApp has been working for nearly two decades in the health domain. In hospitals we supported clinical teams around oncology patients, the data for team decisions and the clinical workflows. Progressing from this to work with the national health system around analysis of large sets of data across multiple aspects of the system including in-hospital data, family doctor data and community data.

It was through this relationship that we were introduced to the European Cystic Fibrosis Society Patient Registry (often acronymized to ECFS -PR) and were contracted to provide development and support for a rewrite of the patient registry platform. ECFS-PR was deployed by OpenApp in 2012 and continues to collect data to help improve standards of care, identify parameters for clinical trials, monitor efficacy of new treatments/medications, conduct research studies, and promote advocacy.

After delivering this platform to ECFS we decided to participate in ECRD in Berlin in 2014. It was here that our eyes were opened to the needs of many other rare disease communities. Since then we have been investing in our registry platform adding features to support the needs of patient groups, clinical requirements, academic needs, regulatory interactions and industrial partners.

Today we provide and support systems for many different types of conditions all over the world.

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

As is the case with most organisations, we anticipate that the consequences of the pandemic will be our biggest challenges. From lack of opportunities to meet patient organisations, to the pressure on the health systems to redirect funding for general health care, to the focus of researchers on many facets of COVID 19—all of these contribute to less opportunities to work with groups in the rare disease arena.

Even with the uncertain current health landscape, we anticipate the registry market will continue to grow. We firmly believe in the merit of registries as a means for organisations dealing with rare disease to bolster community engagement with researchers, advance support of treatments, assess drug efficacy/safety and longitudinal data collection.

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

OpenApp was founded by Mel McIntyre in 2002 and I joined shortly afterwards. In 2017 Mel sadly passed away, and I moved from a primarily technical role, to managing the organisation. My background is quite a technical one, but I have been able to embrace my new role, in no small part due to having such a dedicated, motivated team around me. It has been a pleasure seeing them flourish and progress over the years.

Thankfully, we have been able to grow and develop over the last couple of years while still upholding the company values Mel instilled. I’ve grown myself both personally and professionally, but would never have been able to do so without the help of many others around me.

4.	What and who are your personal and professional inspirations and why?

I do not really put anyone on a pedestal as an idol, but I do like to understand the aspects of those I work with that makes them successful in their respective fields. From former colleagues I’ve learned that focussing on the user and listening to them will open your mind to the actual problems they need you to solve. From Mel, I’ve learned that generosity of spirit with co-workers and clients generates such a strong partnership where we are all pulling in the same direction.

I’ve also been hugely inspired by the rare disease communities, especially when I hear the personal stories and the battles people have fought. Indeed, I challenge anyone not to be inspired and awe-struck by the determination and resilience shown in the face of such adversity!

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

Working in the rare disease space requires a lot of patience—mostly patience with yourself to understand all of the aspects that affect the various stakeholders, from proper data management, to health technology assessments, from GDPR to ethics, from Patient Reported Outcome Measures (PROMs) to phenotypes.

Patience and understanding also with the client engagement as many organisations have limited funds and, rightfully, take their time when deciding who to partner with and how best to allocate their budget.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

I think it is fair to say that it is only in the last few years that most government agencies have started to understand the rare disease communities and the daily burden they bear. As these agencies develop their understanding, they are also coming to the realisation that the barriers are numerous and varied. Some of these barriers need a coordinated approach within the system such as improving the awareness of rare diseases, to the time taken to the correct diagnosis, to the availability of genetic testing and subsequent counselling, to the streamlined care pathways, and community support.

Beyond these there are other gaps that need a wider regional and maybe international and inter-agency collaboration, and definitely the engagement with organisations beyond the health or social care space. These gaps include supporting research, streamlined ethics for research across many institutions, support research in the care setting, support of patient advocacy groups, and newer models of engagement with academia and industry.

7.	What would you say are some of the biggest motivators for your employees?

We have a diverse team of employees hailing from many different backgrounds and nationalities. They are all highly motivated and focussed on the customer experience, to deliver a usable system to the end users, value for the customer, and a consistency of service.

As would be expected, we also have many employees with direct experience of the rare disease communities and the problems encountered there. So they are passionate about helping the communities in meaningful ways too.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

Making decisions knowing that some of them will be wrong can be tough, but it is softened by knowing that the team around me gets behind decisions, and yet are still open minded enough to challenge my approach when the circumstances change or results do not evolve as expected.

One of the more rewarding areas is fostering a relationship built on trust and teamwork with my managers, and seeing the quiet evolution of those around you into highly effective, mature team players.

9.	What would be your one wish for OpenApp for the year ahead?

My one wish for the year ahead would be to expand the number of organisations we are working with, to enable them to collaborate and bring value to their members. Also to build upon our existing client relationships to ensure that we continue to provide the best service possible.

10.

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

My 10 year old self was enamoured with travel and flying and I had ambitions of becoming a pilot and flying all over the world—yes a very common ambition back in the day! I’ve been lucky enough to have travelled quite a bit and to have lived abroad for many years, but the life of a pilot was not for me. With the expanse and togetherness of the rare disease community I can still experience different cultures through my work, albeit in a completely different way than my 10 year old self would have expected.

To find out more about the work of OpenApp please visit;
www.openapp.ie

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Daniel Lewi of The CATS Foundation

11/9/2020

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When Daniel Lewi's daughter was diagnosed with Tay-Sachs, he discovered there was no dedicated UK charity offering support to families such as his, and so along with his wife, set up The CATS Foundation. He talks to RARE Revolution about the incredible support network his organisation has created, and about their involvement in the first clinical trial; ever hopeful of finding a cure

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically The CATS Foundation and what did that journey look like?

My eldest daughter Amélie was diagnosed with a rare disease called Tay-Sachs in 2011 when she was 15 months of age. At the time there was no dedicated charity in the UK or Europe providing information or support about the disease. My wife Patrícia and I started speaking with other families and although we were told at Amélie’s diagnosis that we probably wouldn’t meet another family impacted by the disease, we started to realise that this wasn’t actually the case. Research at the time was still in its infancy, but for us the main thing was that we, as a community, needed support for each other, from each other. So, we put things in place and set-up the Cure & Action for Tay-Sachs (CATS) Foundation to provide support to families affected by Tay-Sachs and Sandhoff disease (known as GM2 gangliosidosis together) in July 2011.

The journey at the beginning was very daunting, but we were lucky that we had a motivated community who wanted support and wanted to help each other. We’ve also been fortunate to be members of groups like the UK LSD Collaborative who have been instrumental in sharing best practices, advice and guidance as we have navigated the realities and challenges of running a viable charity for a rare disease. This is not only focussed on what support we can give, but help on funding and making us sustainable–this is the most important part as families will always need support. Other groups, like Findacure have really helped us with the fantastic workshops they run and the rare disease community in general is very collaborative, we are always helping each other when we can.

Daniel and his beautiful daughter, Amélie

2.

What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

Our biggest challenge is always going to be funding, although I think this is a concern for all small charities. We need to ensure that we are sustainable and that we can continue to do the great work of supporting people. Opportunity wise, we are fortunate that we now have an active research community who are investigating various different treatment options. This in itself will bring challenges because we have to manage expectations of our families about what these treatments can do. But, it’s an exciting place to be and when we look back at how far we have come in nine years it’s a testament to hard work and a close-knit community!

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

The proudest moment is seeing families smiling at our family conferences. It’s the old cliché, but we do all become one big family and the genuine feelings of warmth that we have for each other always brings a tear to my eye. We have families who have lost children to the disease, have a newly diagnosed family member or they themselves suffer from Tay-Sachs and Sandhoff who attend our family conferences and it’s amazing to see everyone relax and just be themselves.
Another proud moment was the recruitment of the first trial. We have been involved in this from the very beginning and it’s remarkable to see a treatment started that we, as a community, had so much involvement in.

4.	What and who are your personal and professional inspirations and why?

Amélie and my other children Grace and Seth are a big inspiration to me. How she coped with suffering from Tay-Sachs for eight years was inspiring. She showed such determination and strength each day and was an amazing role model to her siblings. When she died, she died on her own terms peacefully at home with her mum holding her hand. It’s the worst thing to happen to you as a parent to lose a child, but we really believe she left us on her own terms.

Grace and Seth are massive drivers for us. They were always aware that Amélie was different and needed more help from us and it is truly remarkable how much empathy they both have at such a young age. Grace, who is now the age that Amélie was when she passed away, is such a force and she is constantly getting involved in the projects that we do at The CATS Foundation–it makes us so proud.

My other inspirations are the families and children we have met over almost 10 years. They are all so strong and continue to be so and we’ve made friends for life within our community.

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

It is an all-consuming environment! It’s not just a job, it’s actually enforcing change and helping people who are, in some cases, going through the worst moments in their life. You can 100% make a difference, but you have to be willing to commit. The rewards are amazing though–the first trial we got started is one of those moments I will never forget and to know you are part of something like that really is a key driver.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

You can never do enough for the rare disease community. Sadly, it’s up to the charities and our communities to be constantly banging the drum to get our voices heard on a local, regional and national level. My major concern is that this trend of people having to fight for a treatment option will continue and I hope that the government will step up, take responsibility and stop putting the price of someone’s life against the cost of a treatment. I find that morally wrong and it must stop.

7.	What would you say are some of the biggest motivators for your employees?

Everyone at The CATS Foundation has a personal connection to Tay-Sachs or Sandhoff disease. We have all lost a child to these awful diseases and are fully committed to doing anything to help families after a diagnosis and to drive forward the research for treatments.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

As a charity CEO the toughest part of the role is handling the responsibility of an entire community. I’m fully aware that we are fighting for our children all the time and every time we lose a child it breaks my heart as it means we still don’t have that all elusive cure. It’s something that will never get easier. But each year we are getting closer and the work by doctors, researchers and pharma will, I’m confident, get us there in the near future.

For me, the most rewarding part is seeing families supporting each other. As a charity we were born out of the fact that there was no other environment where families could turn to each other and discuss the reality of the disease. By that, I mean the harsh reality. The topics like how you handle taking a doubly incontinent eight year old child out for the day, how do you manage having carers in your house, how do you juggle work, how do you function on sleep deprivation. We now have this community and I find that the biggest success. Our research has moved forward, treatments are close, but families no longer feel lonely. That to me, is a success.

9.	What would be your one wish for The CATS Foundation for the year ahead?

In the next year I’d really like us to expand our support offering so that it has a real nationwide reach. The current pandemic has restricted our ability to hold face-to-face conferences and catch ups, but I really hope that this time next year we will have had a family conference for 2021!

10.

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

Before I was drawn into the rare disease world my life was very different! Like many people who set-up rare disease charities we never had an intention to enter this world, but now we are here to stay and make a difference.
However, as a child, I wanted to be an archaeologist. Or, as my mum tells me I used to say, a dinosaur digger-upper!

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

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Debra Miller of CureDuchenne

31/8/2020

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Debra Miller, CEO of CureDuchenne, co-founded the organisation along with her husband, with the aim of helping her son and others like him. She talks to RARE Revolution about the importance of combining passion with strong business acumen in order to advance research and achieve their ultimate goal of a cure for Duchenne

CEO series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically CureDuchenne and what did that journey look like?

My entry into the world of rare disease was a result of our son, Hawken, being diagnosed with Duchenne muscular dystrophy at the age of five. I had no intention of founding a non-profit, but there were no organisations focused specifically on finding a cure for Duchenne. My husband and I took the first step forward and have not looked back in the last 17 years.

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

We have no shortage of quality research and clinical programs to fund. Almost every day we come across impactful projects that could accelerate progress toward identifying a cure. Funding is the greatest challenge we have. Approximately 10,000 families are affected by Duchenne, it’s not a large disease community, so we have to find ways to educate and engage the general public in our mission of curing this disease.

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

A career highlight so far has been when CureDuchenne searched for the best scientist to develop CRISPR for Duchenne. We partnered with Dr. Eric Olson at UTSW and co-founded a company alongside him called Exonics Therapeutics. We committed to an investment that was large at that time for our organisation, but we were counting on other Duchenne organisations to help us raise the funds. Unfortunately, none of them contributed, however, we worked hard and were able to fund this project alone. Within two years, Vertex Therapeutics acquired Exonics for $245 million and committed vast resources to do their best to bring this experimental, yet hopeful therapy to Duchenne patients. If anyone can bring a CRISPR treatment for Duchenne, Vertex can. This project demonstrated the commitment of the CureDuchenne team and truly validated our Venture Philanthropy model.

4.	What and who are your personal and professional inspirations and why?

My biggest inspiration is my son, Hawken! He is an amazing 23-year old young man that lives life to the fullest, with Duchenne. He never complains, he looks to the future and has an amazingly positive attitude. He graduated with honors from USC, he is working as a writer for the Washington Post, along with several freelance writing gigs.

I’m proud of his desire to help other rare disease patients navigate this journey and share his faith with so many people. In the Duchenne community, Hawken is advocating for the disease through his passions in gaming, streaming, writing and recording podcasts.

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

To succeed in the rare disease space, you need to be powered by passion. There is no other way. It is very challenging to cure a rare genetic disease and giving up is just not an option. I believe it needs to be a life’s work, otherwise, you will not be able to deal with the valleys of despair and the pinnacle of successes.

Also, you need to be prepared to apply strong business practices to your passion. There are thousands of non-profits that had great intentions, lots of passion, but no plan and not enough financing to succeed.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

I recently presented at a Fireside Chat with Peter Marks, FDA Director of Center For Biologics Evaluation and Research (CBER), and Christopher Austin, Director, National Center For Advancing Translational Sciences (Ncats), National Institutes of Health (NIH). We discussed the need to memorialise the will of the people and the government to find a cure and treatments for COVID-19, after we have treatments and vaccines for this virus. There is much more to be done for rare disease, and it rests on all of us addressing rare disease as an emergency, just as we have treated COVID as a national emergency. As Dr. Austin said, “it is not that we can’t do it, it’s that we won’t”. We need to change the will not too, to can do. The government has many mechanisms to speed access to therapies for rare disease; from funding to working with rare disease organisations on scientific projects to flexible trial design.

7.	What would you say are some of the biggest motivators for your employees?

Without a doubt, a dedication to finding a cure for Duchenne and serving our families are the biggest motivators for the CureDuchenne team. We have been blessed that several of our funding projects have resulted in many clinical trials, approved drugs, and a very successful venture philanthropy model. They are able to point to our progress proudly which attracts donors, scientists, investors and rare disease families.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

Obviously, the sense of responsibility to the families we serve. I do not want to lose any of the boys or young men we have become so close to. There are so many facets to serving a disease community; finding and funding the best research, developing clinical programs and care education to improve the lives of these families, thinking far ahead of our current projects and forecasting what will be needed if the current projects succeed and if they fail. Every day, I feel a bit of whiplash toggling between all of this. And, of course, raising money is a constant priority since it fuels all of our programs. Fortunately, I have a dynamite team of experts that are self-driven, innovative and they teach me so much.

9.	What would be your one wish for CureDuchenne for the year ahead?

We have at least two gene therapy programs entering phase III trials this year. My wish is that they are dramatically effective for the boys and that we can find solutions to the limitations of these therapies which will enable older Duchenne patients to participate in these treatments. My son included, his clock is ticking loudly and we need to move these ahead at warp speed.

10.

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

Before our son’s diagnosis, I was heading down the path of computer publishing. There are a lot of similarities to the hay days of the PC industry and today’s biotech industry, I most likely would have stayed in technology, unless I discovered biotech through a different means. I have always been good at raising money, so this has obviously been a strength for CureDuchenne. As much as I wish my son did not have Duchenne, he saved me from living a life with little meaning chasing financial success. Every single day I am aware of the blessings of my family, my community and my country. My son Hawken has given me the deep desire to help others.

To find out more about the work of CureDuchenne please visit;
www.cureduchenne.org

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Elin Haf Davies of Aparito

18/8/2020

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With a background in paediatric care and nearly two decades of research and regulatory experience, Elin Haf Davies, CEO of Aparito, founded the company to help rare disease patients get access to treatments. She talks to RARE Revolution about how COVID-19 has made remote patient monitoring a priority and about Aparito's truly patient-centric approach

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically Aparito and what did that journey look like?

I started my career at Great Ormond Street Children’s Hospital, which meant that nearly all the patients we cared for had one rare disease or another. I started in neurology, then neurosurgery before moving to neuro-metabolic. There I got involved in supporting children recruited to clinical trials, often when this was the only hope left available to them and their families. This triggered my passion to complete my MSc and PhD, and later propelled me to work at the European Medicine Agency, regulating the plans for paediatric clinical trials.

Aparito was never planned, but an end result of 18 years working with children and not seeing the change in remote patient monitoring and the ability to convey disease impact in a way that was meaningful and easy for children and their families.

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

Covid-19 has changed the need for remote patient monitoring from a nicety to a necessity overnight. But it also brings a lot of uncertainty and new challenges. It therefore feels that we have a very narrow window of opportunity to mould or create a new business as usual that works better for all patients. IF we get it wrong, I dread to think what we’ll do.

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

That’s a tough question. Graduating with a PhD was very special on a personal level. Taking a patient to the theatre play to celebrate her 18th birthday when we had worried whether she would live into adolescence was very special. Being appointed the Chair for Metabolic Support UK. But it’s probably being able to celebrate Dr Emma Foottit’s PhD graduation after our row across the Atlantic Ocean had funded her research in PNPO (a very rare metabolic disorder). That was definitely very special indeed.

4.	What and who are your personal and professional inspirations and why?

There are so many. All the patients and families that battled on through clinical trials. Florence Nightingale and Betsi Cadwaladr as strong early nursing roles figures. Dame Steve Shirley as a female entrepreneur who was way ahead of her time in the tech industry. And personal friends Vanessa Sloane, Pip Hare, Karen Higgins and Dame Inga Beale who are all achieving amazing things today in their day to day lives.

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

Go for it! It’s a very special community. Even at a global level, it’s very close knit and you form relationships that are lifelong. But most importantly, you really get to see the impact and value of your work first-hand. Each rare disease community is unique in their own way, but the solidarity is palpable.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

I’m not sure I can articulate publicly what I feel about the Government on any topic today. I have pretty much given up hope on having any real support from them for health, social or education. So, I’m relaying on activism and grass route efforts to achieve what needs to be done.

7.	What would you say are some of the biggest motivators for your employees?

A few of my team have been touched by a rare disease themselves, and so they have their own motivators. Our team changed a lot during lockdown. We went from nine to 20, with three of the original nine leaving. And I can honestly say that I am so proud of how we all gelled as a team under lockdown. My team now is highly talented, with a work ethic and passion that means that they motivate themselves and each other without me. We introduced OKRs (objective, key results) throughout the company and have talked a lot about imbedding core values and principles over the years, I sense that we’re now really living and breathing them.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

Being a CEO of a start-up is a very lonely place at times. You’re often caught between staff, clients, investors and the bank balance. People love the idea of a start-up but not what the reality of creating one means. But the most rewarding is the ability to carve out a completely new path – and that’s very motivating.

9.	What would be your one wish for Aparito for the year ahead?

In today’s uncertain times, I would say survival. I never take it for granted or assume that it couldn’t all be lost or taken away with a blink of an eye.

But beyond that I would wish for positive global impact on supporting patient care. We’ve just launched our Patient Group Accelerator to co-create digital endpoints and outcome measures that are important to them. I can’t wait to see how we get on.

10.

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

The 10-year-old me always wanted to be a children’s nurse. My childhood aspirations never dreamed of anything other than being a children’s nurse. I had however imagined that I would be working in Africa for Red Cross, Gavi or Save the Children. I had volunteered for Save the Children in Lesotho four months after finishing school. Other life events got in the way and distracted me from that, but I still think I might one day go back and work in Africa as I absolutely love being under the African night sky.

To find out more about the work of Aparito please visit;
www.aparito.com

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Helen Springford of Illingworth Research Group

18/8/2020

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With a background in both business development and nursing, the CEO of Illingworth Research Group, Helen Springford, brings to the role a strong desire to help and support patients. She talks to RARE Revolution about what motivates her, and about her firm belief in encouraging her staff to reach their full potential

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically Illingworth and what did that journey look like?

It was joining Illingworth which led me to being involved in rare disease clinical trials. I wanted to join Illingworth as I was keen to get back to a role where I could have a more direct impact on the lives of patients and their families. I have always been more interested in trials seeing it from the patient’s point of view than what the molecule did and how it performed. After over 20 years in project management and business development roles, having started my career as a nurse and working as a clinical trials co-ordinator in hospital, I felt I was going back to my roots taking the position with Illingworth.

Helen speaking at the MENA launch in Cairo

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

The good news is that, at last, the message surrounding the need for more patient focused trials appears to be spreading and gaining support within the pharma industry. This clearly is a great chance for Illingworth to promote what we have been doing for over 21 years now and to knock on doors which are more ajar than they were in previous times.

The biggest challenge will be growing competition. With the term patient centricity used often now, many companies are considering either acquiring or growing their own mobile research nursing service organically. Although I do not believe that this will have a detrimental impact to Illingworth in the short term (after all, it is not easy to match a company who has over two decades of experience in this field and who has grown organically over that time), I do envisage over time we will have more direct competition for work. However, I do not believe over two years we will see a huge change.

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

I have many. I will point out two which spring to mind. The first one is keeping in touch with the very first transplant patient that I prepared for theatre when I worked in a heart transplant unit in Melbourne, Australia when I was nursing. Unfortunately, the patient passed away a few years ago but I was fortunate enough to meet up with him and his wife several times over the last 29 years. The last time I met his wife and daughter was in 2017, when I went over to Australia on business and was told how much they still thought of me and what I had done for the family as a whole. That summarises for me why I went into nursing and why I love what I do now to make the lives of patients and families with rare diseases easier.

A more general moment would be seeing junior members of staff grow and develop their careers. I firmly believe in the importance of encouraging and training staff so that, in turn, they can enjoy fabulous careers of their own.

4.	What and who are your personal and professional inspirations and why?

My goals, not unsurprisingly, have changed over the years. I am much more motivated by a role I enjoy and that allows me to continue to help patients than anything else now. I am inspired by those who follow their dreams and live the life they want to life, rather than the life they think people expect of them. Individuals who have passion for what they do and stick to their principals inspire me. I have been accused in the past of not being a yes man and I am proud of that. Those who stand up for what they believe is right, even if it is unpopular, impresses me.

I am also hugely inspired by individuals who have rare diseases and the amazing resilience they show.

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

Do it! It can be difficult to hear some of the stories like families trying to get a diagnosis for their child or the general difficulties many face living with a rare illness. However, for me, the determination that they show and unending challenges they overcome is very special to witness and very humbling.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

I don’t feel knowledgeable enough to answer this question.

7.	What would you say are some of the biggest motivators for your employees?

I would say that it is very similar to mine, in that they want to do the very best for patients and caregivers. Indeed, I would expand on that and say that they are driven by doing the best jobs they can do for everyone, including of course sponsors. I feel that we are lucky in that Illingworth is not a large company and, as a result, we maintain close communication with one another. During COVID-19, we have held, and are still doing so, online end of week drinks which has been very popular. Although a small thing, it allows people, irrespective of the level they are in the organisation to share their thoughts and offload.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

Having to have more of an oversight role rather than doing things yourself and delegating to others can be an alien concept to people like me! At times, you have to take decisions and make choices that are not always popular. However, I find by explaining the rationale behind your decisions can help others understand the thought process behind the actions you take. I am acutely aware that the direction I take the company in could have a negative impact on people and therefore any decisions I do take are not knee jerk and are always considered.

Being at the helm allows me to communicate to the whole organisation and during the COVID-19 lockdown, ensuring the staff are safe and well, both physically and mentally has been extremely important to me personally. Also, I love being able to facilitate staff development and help talent grow.

9.	What would be your one wish for Illingworth for the year ahead?

Although COVID-19, and the lockdown as a result, has been devastating for many on multiple fronts, our service offering of a suite of patient focused solutions to aid recruitment and retention into clinical trials has meant that we have been more in demand than ever before. My hope is that the pharmaceutical industry will continue to embrace technology and services to reduce the patient burden, whether or not COVID-19 is around. This will, in turn have an ongoing positive impact on Illingworth’s growth and allow us to share our vision with a wider audience.

10.

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

I consider myself very fortunate in that I always knew I wanted to do something medical. For me, being a nurse opens so many doors. I graduated 30 years ago and I still have three close friends who went to university with me. One is a lecturer, one a matron in paediatrics and one a midwife. I, on the other hand, decided to utilise the qualification to allow me to travel and then move into research. I almost trained as a surgeon’s assistant but stayed in clinical research, a move I do not regret. Life is far too short for those!

The well travelled Illingworth bears!

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

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Durhane Wong-Rieger of the Canadian Organization for Rare Disorders (CORD)

7/8/2020

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With a long-held commitment to advocacy, and familial experience of rare disease, Durhane Wong-Rieger became CEO of the Canadian Organization for Rare Disorders (CORD) in 2012. She talks to RARE Revolution about the challenges that lay ahead as a result of the COVID-19 pandemic, and about the fundamental importance of collaboration for rare disease

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically Canadian Organization for Rare Disorders (CORD) and what did that journey look like?

I have two children who were both born with rare conditions. Different conditions that had very different pathways to diagnosis and treatment. My daughter demonstrated physical and cognitive challenges at birth, including low muscle tone and inability to make contact. Her symptoms could have aligned with many conditions but we could not achieve a specific diagnosis. She did get a diagnosis of thalassemia minor at age two but that could not explain the other symptoms which we learned to manage with rehabilitation and supplemental educational support. At the time, advanced genetic testing and importantly genomic testing were not available. My son was born with a congenital heart defect, diagnosed at birth and he had a clear monitoring and treatment pathway, at least until he reached 18 years of age when the paediatric cardiac system dumped him into adult cardiology with absolutely no expertise or support for childhood heart conditions.

These personal experiences have helped me to understand the needs of rare disease patients and families but were not the precipitating factors for taking on the role. I am actually a psychologist by training and taught about 20 years in university, doing patient advocacy on the side, until 1999 when I left academia to take on advocacy full time. I was a volunteer for the Canadian Organization for Rare Disorders (CORD) for many years, and initially asked to write a Canadian ‘orphan drug policy’ (similar to the USA and EMA policies) for CORD to use as advocacy with the federal government. I became Chair of the board to lead the advocacy for the policy but also to fight for access to rare disease drugs, which were coming to Canada but having tremendous challenges getting funded in the public drug plans. I transitioned to a part-time executive director role on 2012 and have remained in the position to this day.

I have had a lot of international patient advocacy experiences and the contacts are essential in rare diseases. In 2015, I helped to establish Rare Disease International, the global alliance of rare disease organisations to advocate for and effect change on behalf of rare diseases in the global environment, for example, setting up a Non-governmental Organisation for Rare Diseases in the United Nations, achieving a specific reference to rare diseases as a target group within the UN Document on Universal Health Coverage, and signing a Memorandum of Understanding with the WHO to advance rare diseases.

Fight For Our Lives rally at Parliament Hill

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

I wouldn’t have said this at the beginning of the year, but it is clear that we need to keep focus and progress in the midst of the COVID-19 pandemic which threatens to overwhelm all other health issues and usurp all funding and services.

One of our biggest advantages and opportunities comes from having a united global community so we can work and speak with a coordinated voice, within the patient community but also across sectors. Rare diseases have always reached out to other entities, including disabilities, noncommunicable diseases, healthcare professionals, policy makers and developers. Indeed, technology is our other biggest asset, with advances in genetics, drug discovery, gene therapies, and digital health. Finally, there are integrated international platforms, including centres of expertise, collaborative research, data sharing, and patient engagement.

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

Really? I couldn’t say. In this world, there are no individual achievements but only contributions to coordinated and collaborative efforts. What we do at CORD is borrowed from other national organisations and we have increasingly been a source of initiatives for others. CORD is an alliance of patient organisations and we work with the community which translates our work into the grassroots which also support our efforts as an alliance. We also have effective rare disease groups at national and international levels that are much bigger than us so we leverage our efforts off of their support.

4.	What and who are your personal and professional inspirations and why?

Mostly patients, parents, and families who are trying to improve their lives in the face of overwhelming challenges on a daily basis. In addition to my children, I have a husband with Parkinson’s Disease, Familial Hypercholesterolemia (heart condition), and achalasia (motility disorder) with the most positive outlook.

Of course, there are a lot of leaders in the rare disease world and beyond who have been instrumental in advancing our collective cause. Yann LeCam, parent of a child with cystic fibrosis and ED of EURORDIS, Abby Myers, mom of a boy with Tourette’s syndrome and patient champion for USA Orphan Drug Act, Pat Furlong, Duchenne Muscular Dystrophy mom who learned how to crack the professional and policy world and has remained at the forefront even after both of her boys have passed away. Many many more who transformed their personal passion to action on behalf of all.

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

You can only work in this space if you have the personal passion for it. It doesn’t mean you have to have a personal relationship to rare disease but you have to commit as much as someone who does.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

What the government (Canada and most other countries) does is actually work against the rare disease community. Policies that work for the majority and common conditions are applied to rare diseases and work against access and support. It would take volumes to illustrate all the ways but what has been most public is the access to therapies, which are individually expensive but cost-effectiveness is calculated using the same yardstick as for common conditions.

There remains a lack of training for healthcare professionals, there is no national strategy (as there is for cancer, cardiovascular disease, diabetes and mental health) even though collectively there are more patients with rare disease than any of these conditions. There are 7,000 rare diseases affecting nearly 3 million Canadians but no dedicated resources.

7.	What would you say are some of the biggest motivators for your employees?

Everyone who works for CORD and most of our Board members and volunteers have a personal relationship to rare diseases. Passion and first-hand knowledge of the challenges and benefits.

Jonathan Pitre receiving his youth leadership award at CORDs Rare Disease Day Gala

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

Everyone thinks you know what you are doing (and of course you want to project this image) but really you don’t and are making it up as you go along, If you are lucky, you see opportunities and can take advantage of them, so for us, it is important to be agile. We cannot have a traditional three-year strategic plan (although my board keeps asking for this), because the environment is constantly shifting, both in terms of challenges and opportunities. So we need long-term vision and strategic direction but not plans that have to be rigidly followed.

9.	What would be your one wish for CORD for the year ahead?

In 2019, the federal government promised us a $1 billion ‘start-up’ budget to set up Canada’s Rare Disease Drug Strategy. This complements a commitment from P/T gov-ernments to set up a special pathway for access to Drugs for Rare Diseases, in the form of a managed access scheme. Now all government actions have stalled in their pivot to COVID, so we have to pick up the mantle and lead the development ourselves, bringing together all the stakeholders, so we are in place with a strategy and plan for 2022 when the monies were promised to be available. That is what we need to accomplish in 2021.

10.

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

My guidance counsellor said I had way too many options and ideas and my biggest challenge was to focus on one thing and do it well. So here I am.

Rare Disease Day at Queens Park

To find out more about the work of CORD please visit;
www.raredisorders.ca

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Alex Evans of RAREiTi

5/8/2020

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With a background in nursing as well as in the pharmaceutical industry, Alex Evans brings a wealth of experience and expertise to her role as CEO of RAREiTi. She talks to RARE Revolution about the challenges the COVID-19 pandemic has brought to the healthcare industry, and how these challenges can also provide opportunities by learning from the way people have responded to and overcome such unexpected difficulties

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically RAREiTi and what did that journey look like?

I feel extremely privileged to work in the rare disease arena. I qualified as a nurse many years ago. Having that training has enabled me to have a clear view of the challenges that patients and their families overcome. My career also included a number of years in the pharmaceutical industry, working for AstraZeneca across multiple therapy areas and Biogen, specifically in multiple sclerosis. It was when I worked at Nutricia, and then Healthcare At Home, that I first experienced the impact a rare condition can have on a patient and their family.

Joining RareiTi was an easy decision for me to make, because it allows me to be part of a transformation in how companies view managed access programs. There’s tremendous potential here to help rare disease drug developers increase their success rates locally and globally, and at the same time help doctors and patients gain access to new, and often life-changing, medicines.

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

I hate to say the C word, but I think that Covid-19 is going to be a challenge for the health economy globally for the next few years. Some payors are finding themselves with tricky decisions to make in managing healthcare budgets. They need to ensure that their patient populations all get access to the treatments they need, despite the risks that the pandemic poses for rare disease patients.

The opportunity for RareiTi is to assist healthcare decision-makers with this task by providing high-quality, in-home services and meaningful real-world data. RareiTi’s unique approach to managed access programmes, based on a foundation of real-world evidence and innovative patient-friendly technologies, delivers some big picture objectives for healthcare ecosystems.

There is a great deal to be learned from how Covid has been managed. Due to the nature of the disease, regulators have had to look to innovative ways to bring treatment and diagnostic kits to market quickly. The same principles should be applied to access for therapies in rare diseases.

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

I am lucky. I have a really great career. I think getting my first CEO Job—this one at RareiTi, stands out as the proudest moment. RareiTi is a truly responsive and responsible Managed Access company that combines a breadth of global experience, cutting edge technologies and analytics that deliver better outcomes for everyone. Our founders, Natalie Douglas and Wendy White are industry veterans with a vision that will truly benefit rare communities. Our global reach and infrastructure mean that our clients can be confident that the services we provide are based on expertise and experience across the world, meeting safety and regulatory requirements in a large range of countries, and providing multi-lingual support.

4.	What and who are your personal and professional inspirations and why?

I’m inspired by the example of Nobel Prize winning geneticist Barbara McClintock, who revolutionised the understanding of how genes work through her pioneering discovery that genes can change positions on chromosomes.

On a personal note, I am studying for a professional doctorate in nursing at the moment, and I have done a lot of research into the history of the profession. I believe that leadership is key to really good nursing care. The women who built the foundation for leadership in nursing—Florence Nightingale, Mary Seacole, Jean Watson and Barbara Carper, are all truly inspirational to me.

However, I also want to mention the inspiration that patients and patient advocacy groups bring. The patient voice is so important in rare disease, RareiTi works in close partnership with key patient advocacy groups to support the important work that they do.

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

I would advise them to definitely go for it. The opportunity to work in such a varied arena with fabulous people is inspiring every day!

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

One of the gaps that is currently impacting rare disease patients is the interruption in clinical research caused by Covid-19. Some research programs are finding their way through this crisis by switching to telemedicine and home care, which is one of RareiTi’s strengths. It is my hope that regulators will be more open to this model even after the crisis is resolved, because so many patients see it as safer and a better fit for their lives.

7.	What would you say are some of the biggest motivators for your employees?

We have a fabulous team, whose ambition is to help patients and doctors get early access to life changing medication. RareiTi specialises in managed or early access programs, which means we work with the regulators to ensure the safe provision of medication under regulated conditions to patients before they have been granted a licence for use. Helping patients and their families get treatment early, can be life changing for some patients who have been waiting for a treatment to emerge or to be approved.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

The most rewarding part of this job is being able to help those hard-to-reach communities of patients and being able to improve their quality of life. Managed access programs are such an important part of the rare disease arena, ensuring that patients can access long awaited medicine, outside of the clinical trial (which also play an important role, but are not accessible to everyone) and can often be a lifeline to patients. These programs offer the clinicians more options when treating patients, and experience with a new medicine before it comes to market. The real world evidence generated by managed access programs also often enhance the data that is required to get approval of the medicine, so supports the approval process too.

Working in this space requires constant learning, innovation, and adaptation, because of rapid changes in both the science and the policy environment. That is one of the most challenging things about rare disease, but it’s also one of the most gratifying.

Because I began my career in nursing, I find it particularly rewarding to see how the work we do provides clinicians with more options when treating patients. It also enables them to gain experience with new medicines before they come to market. The real-world evidence we collect supports the approval process so drugs can get to market sooner.

9.	What would be your one wish for RAREiTi for the year ahead?

To help as many patients as possible get early access to medication.

10.

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

There was no specific plan B. I have always been drawn to jobs that ultimately help patients. I think that the impact that drug developers have on patients’ lives is sometimes misrepresented, and not considered in its purist form. The people I know who work in rare diseases are dedicated to bringing new medications to patients and want as many people as possible to benefit. Being in a position to help companies connect patients and doctors to new life-changing medication is my dream job!

To find out more about the work of RAREiTi please visit;
www.rareiti.com

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Laura Helms Reece of Rho

24/7/2020

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Rho CEO, Laura Helms Reece, gained extensive experience in the world of business and pharmaceuticals, before returning home to join the family business. Laura talks to RARE Revolution about how the biggest motivator for her, and her team, is making a difference to people lives, and about the challenges and opportunities that lay ahead for clinical trials

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically Rho and what did that journey look like?

My parents founded Rho in our basement in 1984. My father was a professor of biostatistics and my mother ran a data management group at UNC-Chapel Hill. They started Rho because, as state employees, they were looking to earn extra money to send their kids to college. When they started, they hired graduate students and moonlighted faculty and staff from UNC to help run projects in our basement. I checked data for allowance money as did my brother.

I did not actually intend to stay in the family business. When I went to UNC as an undergraduate, I was a business major. I graduated with my master’s degree and had to decide which direction I wanted to take—stay on the path to become a finance professor or become a biostatistician. On a good day in finance, you make someone rich. On a good day in biostatistics, you make someone healthy. I decided I felt better about making someone healthy.

I still did not want to work in the family business, so in time between earning my master’s degree and getting my doctorate I worked for Glaxo. After my doctorate, I worked for Astra Zeneca in Wilmington, Delaware, and learned a lot about how to develop a drug. Despite having a wonderful experience, I wanted to come home to North Carolina. That is when I talked to my parents about working for the business.

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

Certainly, over the next several months, and possibly through much of next year, the novel coronavirus (COVID-19) will continue to have a major impact on how clinical trials are run. Patient safety is always a top priority, so it is important we make sure we have the right protocols in place to ensure patients feel safe about participating in clinical trials, especially during this time.

Our teams have helped many sponsors navigate and implement decentralised solutions for their clinical trials and programs. This includes providing recommendations for the level of decentralisation required (e.g. a fully virtual trial or a hybrid approach), as well as the development of strategic plans for remote and central monitoring, technology integration, IP management and site and patient engagement.

Based on our experience, we believe that the best decentralised solutions are patient-centric, customized and rooted in a robust Risk-Based Quality Management (RBQM) framework, as specified by the E6R2 guidance.

Additionally, Rho is coordinating a new study called Human Epidemiology and Response to SARS-CoV-2 (HEROS) to help determine the rate of COVID-19 in children and their family members in the U.S. The study also aims to determine what percentage of children infected with SARS-CoV-2, the virus that causes COVID-19, develop symptoms of the disease, as well as whether rates of SARS-CoV-2 infection differ between children who have asthma or other allergic conditions and children who do not. HEROS is sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH).

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

I am proud of the work we do at Rho every single day. It is one of the blessings of working in our industry. Certainly there are huge highlights like the days that a therapy we’ve supported comes to market or a major publication we’ve supported comes out that will change lives for people.

The days when we discover a treatment doesn’t work are heart-breaking. And yet, I take pride in our work to test failed therapies vigorously. When we find a therapy doesn’t work we can help clients and patients move on to more hopeful options. I am also proud of the work we do at Rho to build a business that treats employees and clients honourably.

4.	What and who are your personal and professional inspirations and why?

Personally, I am inspired by my grandmother. She was widowed at the age of 38, with a high school education and three small children, during the height of the polio epidemic. She eventually became a very successful businesswoman but had some hard years. My parents, who both came out of childhood poverty and built a successful business, are also an inspiration to me.

My professional inspirations are many and varied. Dean Barbara Rimer has inspired me as she has overseen the Gillings Global School of Public Health at UNC. She has dealt with years of decreasing state funding while increasing the value that the Gillings School brings to public health. In contrast with Dean Rimer, whom I get to talk with regularly, I am also truly inspired by Dolly Parton, whom I’ve never met. She has built an impressive business empire, brought jobs to the depressed area where she grew up, and funded a number of valuable social support charities. And she did that with big blonde hair and a country twang.

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

This is a space that requires innovation and often presents unique challenges. The rare disease community is one that faces a number of hurdles—if given the opportunity, interact with the patients and caregivers and listen to their voices. Patient and caregiver input in the rare disease space is valuable to researchers throughout the product development process. It is also critical to work closely with regulatory agencies, as the challenges of product development in the rare disease space may require creative study designs and agency feedback and support in uncharted territory.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

The rare disease community is a strong and resilient one, and they have worked hard to give themselves a stronger voice in the local government and afford themselves the opportunity to provide feedback. A state’s government has a critical role to play as to how a given state’s programs and policies can benefit the rare disease community. On a more central level, the Orphan Drug Act provides for status of designations such as orphan drug designation and regenerative medicine advanced therapy designation in order to incentivise companies to develop products for rare disease. The biggest gaps I see are accessibility and affordability. Rare disease therapeutics are extremely expensive to develop and in particular we have seen recent gene therapy products approved for rare diseases that have a high price tag. Congress will need to work with pharmaceutical companies and insurers to develop a strategy for financing these treatments. There are innovative, life-changing therapies being developed (and approved!) and the government must now give thought on how to determine the price of these treatments and how to finance them--the benefits of an innovative treatment should not be denied due to affordability.

7.	What would you say are some of the biggest motivators for your employees?

First and foremost, I would say the nature of our work is the biggest motivator for our employees. As I previously mentioned, on a good day in biostatistics, you make someone healthy. The fact that we get to work with sponsors to bring lifesaving or life-altering therapies to market to help improve health, extend life and enhance quality of life.

I think the culture Rho provides is also a key motivator. We are fortunate to have a collaborative work environment where we value a strong team spirit and are loyal to each other and to our clients—and there is a mutual trust and respect across the entire organisation. Rho also encourages a work-life balance that allows employees to bring their best selves to work while also being passionate about their lives outside of work.

Rho's new headquarters

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

The toughest part is that the buck stops here. I am responsible for the decisions that are made at Rho and I take that very seriously. For my first eight years as CEO, I had a co-CEO who was also my brother. Sharing that burden with someone you trust completely makes the burden much easier to carry. I’m glad I had those years. I still have his support, as well as the support of my parents, who founded Rho. But I’m the one who has final responsibility.

The most rewarding part of being the CEO of Rho is getting to work with truly wonderful people. Because almost all of Rho works out of our Durham office (when we aren’t in a pandemic), I get to talk to people throughout the organisation regularly. I love working with the executives and project teams that I interact with frequently. And I also love the conversations I have over lunch in our hub and in the hallways. Rho is full of talented, dedicated, and delightful people.

9.	What would be your one wish for Rho for the year ahead?

I would love for us to increase the number of lives we improve by extending life, enhancing health, and improving the quality of life for the patients. We do that by expanding the number of clients and projects we serve. Our talented teams of experts make a difference in the projects they support. I’d love to help more projects to successfully execute their trials and submit to regulatory authorities world-wide.

10.

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

My 10-year-old self wanted to be a Broadway star. Sadly, as my 9-year-old recently put it, “Mama just can’t catch a tune.” So that isn’t really a plan B for me!

The truth is, I love business. If I weren’t the CEO of Rho, I’d start another business with a worthy mission. I’m just a business geek.

To find out more about the work of Rho, please visit;
www.rhoworld.com

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Kristine Hoestermann and Theresa Thomas of RARE.

24/7/2020

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Kristine Hoestermann and Theresa Thomas' partnership was formed from a shared passion and drive to raise awareness for rare diseases. RARE. is not just a brand but a movement. They speak to RARE Revolution about what led them to creating RARE. and about their mission to make the invisible - visible

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically RARE and what did that journey look like?

Neither of us had a choice when it came to the world of rare disease, although we wouldn’t change it.

Kristine: At the age of 27 I was an avid runner and noticed a pain in my knee. What I didn't realise then was that pain was the beginning of my rare disease journey. I created RARE. initially as a way to express myself and the change that was happening in my life. After some time I realised that RARE. was a tool that not only could help me but could help others as well. The safe place I created was needed by more people than just myself. I began creating clothes and household items that helped raise awareness for rare diseases. Which eventually led me to connecting with Theresa.

Theresa: Two and a half years ago my son Owen was born weighing in at almost 12 pounds. I immediately knew that something was different about my son. He was very large and his tongue protruded from his mouth. Two weeks later we got his official rare disease diagnosis of Beckwith-Wiedemann Syndrome (BWS). A year after my son was born my husband was pushing me to start a blog. He knew I was struggling with our son's rare disease and he felt that writing would help me express myself. Little did I know six months later I would connect with Kristine through Instagram, and ultimately become her business partner and COO of RARE.

Kristine

Theresa and her family

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

We believe our biggest challenges are exposure and reach. Although we have been connecting with so many amazing families there are still so many people suffering in silence with no community or voice. We want to reach them as quickly as we can so they know that they are not alone.

Some of our biggest opportunities have been, and continue to be, working with philanthropists in the rare disease network. We are working with authors such as Deborah G. Edwards, David Fajgenbaum, and Sarah Ramey. We are currently working on a few projects with non-profit organisations as well as other rare disease fighters, in addition to working on next year's Rare disease day event.

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

One thing we value the most is the ability we have created to connect with people across the world. To date RARE. is in 31 states and six countries. We also worked incredibly hard on our shop with a purpose initiative. When you check out on RARE. you now have the ability to choose between several non-profits where 15% of your purchase total will be donated to.

4.	What and who are your personal and professional inspirations and why?

Kristine: Taylor Swift has always inspired me because she built her brand from the bottom up using her own story to touch the masses. She is always authentic and real and connects with her fans despite the criticism and negativity she receives. She continues to stay true to who she is. She built her brand through personal connections with her fans and continues to cultivate kindness and fight for equality which correlates to the brand we are building—one that is authentic and made from genuine connections and stories.

Theresa: My inspiration has always been my older sister Michelle. She was born with one arm but has never let it hold her back. She deals with everything head on and is now a paediatric nurse at one of the top Children’s Hospital. She has faced adversity her whole life but you would never know it. She has always been a source of inspiration and strength for me.

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

Listen first, be patient, and respond with kindness.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

We feel there are gaps from diagnosis all the way through to workplace accommodations. Until we put the patient at the centre of the solutions these gaps will continue to grow.

7.	What would you say are some of the biggest motivators for you?

Kristine: The biggest motivators for me are the kind messages and affirmations that RARE. receives. We are constantly reminded that the work we are doing is reaching the people who need it most.

Theresa: My biggest motivator is my son Owen. Watching him grow and go through life with a rare disease, one that most people have never heard of, continues to motivate me to create change and awareness for him and other BWS children.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

One of the hardest things about being a CEO, one with a rare disease, is allowing myself the time to rest and recover. When you have a dream and a business and tons of ideas it can be challenging when your rare disease gets in the way.

The most rewarding things are seeing the impact that our products and our brand have made. They have helped people find their voice.

9.	What would be your one wish for RARE for the year ahead?

Our wish is that we are able to continue our journey of cultivating change, impacting lives, and creating designs that people resonate with.

10.

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

RARE. actually was our plan B.

Kristine: Prior to getting sick I was a clinical social worker.
Theresa: Prior to having Owen I was a registered cardiovascular invasive specialist.

Our 10 year old selves both wanted to do jobs that made us happy and had a tremendous positive impact on others. We both were in fields that accomplished that, however our lives took a different turn and we are now able to continue to live out our 10 year old dream through RARE.

To find out more about the work of RARE. please visit;
www.findyourrare.com

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Patrick Howie of MediFind

7/7/2020

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Patrick Howie, CEO of MediFind, knows all too well the challenges patients can face in trying to obtain the right care and learn about new treatment options. Motivated by personal experience, he founded MediFind, to try and ensure precious time isn't wasted; by helping patients find the expertise and treatments they need, faster

CEO Series: meeting the beating hearts behind the RARE brands

1.	What made you want to move into the wide world of rare disease, and then specifically MediFind and what did that journey look like?

I was at brunch with my brother, Dennis, who had been fighting a rare cancer for four years at that point, when he said, “I’m running out of options, any ideas?” I literally couldn’t breathe for a minute. I also knew that I had to come up with some ideas. While I’m not a doctor, as the head of Global Analytics for one of the largest pharmaceutical companies in the world, I did have access to unparalleled data capabilities and an intricate understanding of the world of healthcare. I realised then that he had lost a lot of time trying to find experts and trying to learn about new treatment options, and that I could leverage analytics to help him, and patients like him, find better care, faster. That’s how MediFind was born.

Determining expertise

One of a kind—example data sources

2.	What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

Our biggest challenge will be to make sure everything we do is easily understood by patients and their caregivers. Healthcare can be extremely complex, especially the terminology. We are working very hard to translate this complex medical terminology so patients can more easily understand and act on it. Many patients want to be more actively involved in their treatment decisions, and once the medical jargon is removed, they will better be able to use their voice to get the care they need.

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

As we started to build out MediFind, an American colleague of mine heard about what I was doing and asked for help. His son had some significant health issues from birth and was being treated at one of the medical centres considered to be a world leader in his condition. They were recommending his son start a treatment, but the father wanted a second opinion. I pointed him to some experts in Europe, who willingly reviewed his son’s history and records, and told the father that the treatment being recommended had recently been shown to not work. The European doctors consulted with the local doctors to come up with a different treatment plan that is dramatically better than the original plan.

4.	What and who are your personal and professional inspirations and why?

Obviously my original inspiration was my brother, but I am now inspired by the millions of people like him around the world who tackle their medical situations head on and are looking for a better way. Everyone deserves the best chances at the best health.

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

In my opinion, passion and persistence are requirements to working in the rare disease space. Tackling a rare disease is particularly challenging because it is hard to get enough critical mass to really drive progress for any single rare disease. The good news is that the rare disease communities are extremely passionate and frequently have an impact much bigger than their numbers would indicate.

6.	Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

Given that I am based in the United States of America, our approach is obviously quite different from much of the world. While every government faces a situation where there are more demands on resources than can be fully met, I do believe governments are missing out on a big opportunity to help rare disease patients (and, quite frankly, all patients) by not investing in maximising the impact of generic medications. There are thousands of generic medications that alone, or in combination, might be able to help rare disease patients but there is no economic incentive for private companies to explore those options. A government program to maximise the impact of these low-cost treatments could have a huge impact to patients and could reduce costs to the system.

7.	What would you say are some of the biggest motivators for your employees?

Just about every one of our employees has a friend or family member who has struggled with a rare disease. For them, the motivation is easy, as they understand the impact we can make. For those that don’t have that direct connection, I believe they all understand the greater human impact we can have, even if it isn’t as personal for them. We are also solving some pretty tough challenges, so many get an added motivational boost from working on difficult problems.

8.	What are the toughest parts of being a CEO, and conversely what are the most rewarding?

The most difficult part for me is having to spend more time than I would like on operational tasks. I know they are needed, which is why I do them, but I want to spend as much time as I can trying to solve those problems that will help the most patients.

9.	What would be your one wish for MediFind for the year ahead?

I want to release the first complete and constantly updated list of treatments for every rare disease. I believe that one of the greatest opportunities we have in the near term is to learn from the experience of others and which treatments, or combinations of treatments, have helped patients with a specific disease or condition related to a disease. This information is hard to get, and difficult to synthesize, and we have been working for the past three years to develop a system to capture this information in a comprehensive and ongoing way. This is related to the notion of medication repurposing—many doctors are having success using combinations of readily available treatments to treat rare disease but it is hard to find out what has been tried and what is working (or not).

10.

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

The truth is that I didn’t seek out this job, but I knew that there was an important need that we could uniquely solve. My 10-year-old self was blissfully ignorant of rare diseases and I didn’t have a personal connection to them until my 40s, but helping patients with rare diseases has become my life’s mission.

Patrick's brother Dennis - his inspiration

To find out more about the work of MediFind please visit;
www.medifind.com

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Con Hennessy of OpenApp

Daniel Lewi of The CATS Foundation

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Helen Springford of Illingworth Research Group

Durhane Wong-Rieger of the Canadian Organization for Rare Disorders (CORD)

Alex Evans of RAREiTi

Laura Helms Reece of Rho

Kristine Hoestermann and Theresa Thomas of RARE.

Patrick Howie of MediFind

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

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