 Maria interviewing Dr. Janet Woodcock
 The definition of multi-tasking! Maria giving a talk with her young son on her hip
 To find out more about the work of TREND Community please visit www.trend.community
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The beautiful hemophilia memorial in the AIDS Grove
Kimberly with our Editor, Nicola Miller, at Global Genes Rare Patient Advocacy Summit, September 2019
 To find out more about the work of Global Genes please visit www.globalgenes.org
 To find out more about the work of Genpharm please visit www.genpharmservices.com
  To find out more about the work of ThinkGenetic please visit |
 | Continuing in our CEO Series, Becky Jenner, CEO of Rett UK, shares her very personal journey that saw her give up a career in retail to co-found the charity Extratime and eventually become CEO of Rett UK CEO Series: meeting the beating hearts behind the RARE brands |
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| 1. | What made you want to move into the wide world of rare disease, and then specifically Rett UK and what did that journey look like? |
| My career spans two halves really. Firstly in retail management starting as a Saturday assistant for Tesco, moving up to be an assistant manager with them and then with a company now called M & Co, initially as a store manager and then as an area manager, a post I returned to part time after having my first child Rosie … until D-day! D-day or diagnosis day was the day I found out my beautiful, smiley and happy fourteen-month-old had Rett syndrome, a rare, complex neurological disorder which mainly affects females. After a period of near-normal development up to the age of around one year to 18 months, the child regresses, in Rosie’s case overnight, losing key skills including the ability to walk, talk and have any purposeful hand use. With time, many medical comorbidities emerge including epilepsy, scoliosis, dystonia, gut and bowel dysfunction and severe breathing problems. To say life took a turn at that point is probably an understatement. I resigned that day … I still remember saying hysterically to my boss at the time, ‘I will still be changing nappies when she is 30!’ Sounds ridiculous now! I spent the next few years concentrating on her, finding out how I could help her achieve the best she could and trying to find a way forward. I became a trustee of Rett UK, a post I held for 7 years, had two more children, did a business degree part time, then at the point when I wanted to return to work and found there was not any childcare available that would meet her needs (by now very complex) and that my two boys could go to too, I started a charity with a mum in a similar situation; Extratime has just celebrated its 15th year of providing inclusive after-school and holiday activities for children and young people aged 5-25 years. When the vacancy of CEO at Rett UK came up, it was a natural step for me to combine my business, charity and personal experience … especially into something that I have such a personal investment in. That was in 2014, after 12 years at Extratime. |
 Celebrating 15 years of Extratime |  |
| 2. | What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years? |
| The biggest challenges for us, like any small charity, is funding; finding new sources, making it go further but seeing demand for our services increase as cuts to statutory services that our families rely on continue to bite and cause them stress and hardship. We have worked hard to build alliances and partnerships where we can as we see the greatest opportunities here to ensure our families get the best we can offer them. We are a small community and we need to join with others to have a bigger voice and more influence about the things that matter to our families. |
| 3. | What is your proudest moment in your career thus far? I think that has to be winning Voluntary Sector Champion for Brighton and Hove in 2013, voted for by parents in the city and other professionals. It was fantastic to know how much people valued Extratime and what I had achieved with the team there. |
| 4. | What and who are your personal and professional inspirations and why? |
| Rosie is my inspiration, my motivation and my drive every day to improve things for her and others affected by this devastating disorder. What she has to cope with on a daily basis is unimaginable for most people but she has an incredible zest for life, a wicked sense of humour and is the heart of our family. If she can keep going with what she has to deal with – anything I do is a walk in the park by comparison. |
 | Becky with Rosie |
| 5. | What advice would you give someone considering working in the rare disease space? |
| Do it! Especially if you are a medic or a researcher! We need more of you! We need new people to pick up the baton for rare diseases and take an interest in the research. There is a lot going on especially with gene editing and gene therapy – these are exciting times and there will be some life-changing treatments in the next few years. |
| 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 things are improving with the combined efforts of the rare disease community but we need to keep the pressure on. Who knows with Brexit how that will affect the European playing field? A lot of research is done in Europe involving UK researchers and vice versa. How will those programmes be managed, funded? Will we lose some good people – probably! And at a time when so many advances are being made with gene therapies in particular, this is not great. The government nationally needs to ensure that this vital research is not compromised and also take a long term view that investment in rare diseases may be expensive initially but the longer term payback of getting the right treatment and therapies for this community is greater. Locally, our community is suffering because of the financial austerity which has decimated care and support services especially respite. Families crumble without regular breaks. |
| 7. | What would you say are some of the biggest motivators for your employees? |
| Without doubt it is our families, their lives, their stories – our staff are moved every day by the challenges they face and they genuinely want to help make a difference. When they receive a thank-you card from someone, and they get a lot, they are so touched that they found the time to write it! |
| 8. | What are the toughest parts of being a CEO, and conversely what are the most rewarding? |
| The toughest part for me is dealing with the death of someone with Rett syndrome. I have been involved with Rett UK for over 22 years as a trustee, a family member and now CEO – many of my friend’s daughters have already passed. Of 7 girls that Rosie was at school with who had Rett syndrome only two are still alive. It is also hard for me to switch off as so much support and fundraising is now done on social media. The expectation of an instant response, especially outside of ‘normal’ work hours, is a real challenge for a small team working nationally so invariably that falls to me. The most rewarding is getting out of the office to meet the families at our events and seeing how much they value what we do. The feedback we get is fantastic! |
| 9. | What would be your one wish for Rett UK for the year ahead? |
| That they run for government, sort Brexit out and put rare diseases at the heart of any plan. |
| 10. | If you weren’t CEO of Rett UK, what was Plan B? What did your 10-year-old self want to do as a job? |
| Plan B is to get a piece of land, grow beautifully scented British flowers to sell to anyone who appreciates them! My ten-year-old self wanted to do something that involved working outside but the only thing the ‘career advisor’ at the time could suggest was working for parks and gardens! My youngest son, now 18, has just completed the most amazing BTEC course in Adventure Sports – now he can go anywhere in the world teaching sailing, kayaking, mountain bike leading, climbing! How I would have loved to do that! |
To find out more about the work of Rett UK please visit
www.rettuk.org
www.rettuk.org
  | Second up in our brand new CEO Series is OPEN Health co-founder and COO, Sandy Royden. Sandy shares how she has been guiding the brands growth in the rare disease space and her aspirations for the future. CEO Series: meeting the beating heart behind the RARE brands |
1. | What made you want to move into the wide world of rare disease, and then specifically OPEN Health, and what did that journey look like? |
| Given my pharmacy background, I have always been interested in the pathophysiology of disease. Rare Diseases therefore fascinate me – as they ’fall outside of the norm’ in terms of common ailments and conditions. I strive to understand their genetic links or inherent causes, and then thrive on the opportunity to help communicate and explain this to a wider audience – HCPs, patients, family and carers. Being able to rationally explain a disease and potential treatment options, particularly in the rare disease space where their population rarely get any support, is hugely gratifying. This personal interest is also reflected across OPEN Health, where there are a number of like-minded people with similar interests. In particular, our director of rare disease, Gavin Jones, does a great job of really understanding the impact of a rare disease from a patient’s perspective, and communicating this across our business, so that we all share the same empathy, passion, and desire to have a positive impact on the treatment of these conditions. |
2. | What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?From a rare disease perspective, I think it is a case of constantly challenging ourselves to find innovative ways of supporting our customers. The landscape is rapidly evolving with significantly different ways of treating patients very much on the near horizon. You just have to look at the imminent introduction of gene therapies to see that healthcare communications and value recognition need to rapidly evolve. I think it is exciting that rare disease could be seen as the platform to promote new relationships between the varying stakeholders in healthcare including patients and their advocacy groups. We would like to be seen at the forefront of this new world and are busy working to create solutions to support improved communications and outcomes both now and long into the future. |
3. | What is your proudest moment in your career thus far?My proudest moments are when you actually hear about ‘real’ patients who have benefitted from the treatments we have worked on. At the end of the day our goal is to ensure stakeholders are educated on relevant aspects, across a variety of different diseases and specific treatments, and that by effective communication initiatives we can optimise patient access to medicines. |
4. | What and who are your personal and professional inspirations and why?I take personal inspiration from Audrey Hepburn – there was nothing she couldn’t do - she was a humanitarian, dancer, actress and member of the Dutch resistance. What I take from her is ‘endless optimism’ –‘Nothing is impossible’ – in fact the word itself says I’m possible! From a professional perspective, with pharmacy at my core, I want to ensure we drive patient access to new and innovative medicines and technologies. |
5. | What advice would you give someone considering working in the rare disease space?Whatever role you are considering playing in the rare disease community I would say go for it! It is very fulfilling to work in this field and supporting improved outcomes where there is significant unmet need. You will experience a close connection while making a difference for rare disease patients and their families. Whatever role you are in, my advice is that you develop a clear mantra in what you are trying to achieve and the benefit this will have within the rare disease community. By living and breathing this mission you will find that your voice will more likely be heard and you can have a greater impact. |
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 one of the most concerning gaps is the challenge of gaining appropriate funding for the use of medicines and therapies in rare disease. Given the numerous emerging new treatments and technologies there is rightfully a lot of hope within the rare disease community. However, these medicines will often come with a high price tag and their value to health systems may not be realised for some time after they are introduced and initially used by patients. This combined with health technology appraisals that are not best suited to assess value in rare disease and we have an issue. This is playing out at the moment with a number of high-profile cases not gaining approval for use in various health systems. We need to have more open dialogue across the different stakeholder groups in rare disease so we can ensure equitable access to the most effective treatments for patients. |
7. | What would you say are some of the biggest motivators for your employees?I believe our culture is something that is incredibly important to our employees. We have a very diverse range of capabilities within our business and with that come wide ranging skill sets and personalities. Collaboration is so important to us in enabling people to positively work together in supporting improved outcomes for our customers and the patients they are striving to serve. Everyone in our business, from our creative directors to our admin staff, recognises that they all play a part in improving healthcare for patients which in turn motivates all of us to make every effort in making a difference. |
8. | What are the toughest parts of being a CEO, and conversely what are the most rewarding? Being a CEO can be extremely rewarding - particularly seeing and supporting people within our organisation to grow, develop and achieve their personal and professional aspirations. But alongside that comes some of the tough decisions – reducing resources and cutting your cloth during the challenging times. |
9. | What would be your one wish for OPEN Health for the year ahead? I would love to see OPEN Health continue to grow and further enhance our reputation in the rare diseases space. I want us to be able to deliver truly innovative programmes that have a measurable and positive impact on patients’ lives. |
10. | If you weren’t CEO of OPEN Health what was Plan B? What did your 10-year-old-self want to do as a job? I always wanted to be a pharmacist, and once qualified and more experienced in the various career opportunities. I sought out a clear path through marketing and healthcare communications. To be honest, if I had failed to achieve the grades to study for pharmacy, I was going to have a total career change and study hotel catering and management………who knows where that would have taken me! |
To find out more about the work of OPEN Health please visit www.openhealthgroup.com
  | We are thrilled to launch our brand new CEO Series with friend of the revolution, Rick Thompson, With a surprising journey from moles and dinosaur digging to CEO of the patient loved charity, Findacure, Rick Thompson shares his journey into rare disease and his hopes and challenges for the year ahead CEO Series: meeting the beating heart behind the RARE brands |
1. | What made you want to move into the wide world of rare disease, and then specifically Findacure and what did that journey look like? I’ve been into science for as long as I can remember, and really just followed my nose in my studies. I did a Biology degree, and ended up specialising in Zoology. From there I spent time at the Natural History Museum in London, before starting a PhD all about moles. Now, moles are truly fascinating animals, and studying to be a doctor (the useless kind) was a fantastic experience. But by the end of the five year period it dawned on me that very few people in the world really cared about my work. It would never make anyone’s life better, or richer. It was this realisation that drove me to look for work in the charity sector, applying my scientific background to the medical field. The position at Findacure was a really exciting one – it offered me the chance to learn more about the biomedical field, while interacting first hand with people living with rare diseases – people who rarely get any support. This was a huge opportunity for me to make use of my studies in a meaningful setting, and when I was lucky enough to be offered the job I seized it with both hands. I’m acutely conscious that the majority of people working in this field have personal experience of rare conditions – something that I fortunately do not have. There are times when I worry that this disconnect could limit my ability to connect with the patient experience; however, I also hope that it helps to keep Findacure from bias towards any one condition, and drives us to find and listen to as many patient stories as possible. |
2. | What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years? |
| The biggest challenge for everyone in this sector is funding and sustainability. A huge priority for us right now is to secure more long-term funding so that we can plan our training programmes in a more strategic manner, and hopefully grow our activities so that they can benefit more organisations. We hope that this type of sustainability will allow us to dedicate more time to our beneficiaries, but also more time to partnerships. There are a number of fantastic cross-rare disease organisations out there, Rare Revolution and Medics4RareDiseases to name two, that I hope that Findacure can begin to work with more closely in the next few years. This will help to build a tighter and more unified rare disease community, help us all to share resources and expertise, and hopefully ensure the sustainability of all organisations. |
3. | What is your proudest moment in your career thus far? |
| This is a tough question. I was obviously very proud to take over Findacure as CEO, and I am proud whenever I get to walk out on stage and talk about our work. My proudest moments though tend to be more related to the impact of our work – seeing the energy in the room at the end of our conference or workshops always fills me with a sense of pride and well-being. I was also particularly proud when my mentee Liz got up on stage and delivered her first ever presentation to a live audience at last year’s peer mentoring summer meet-up event. She did an amazing job, and it felt like a huge personal achievement for her, and a great tribute to all of her hard work. |
4. | What and who are your personal and professional inspirations and why? |
| David Attenborough is a national treasure and a true hero. I saw him talk in person recently, and his delivery, humour, and desire to share science in an accessible way is absolutely priceless. His passion, dedication, and openness is something we can all aspire to. Professionally, there is no one inspiration, it is more the knowledge that nearly every patient group leader that I work with is doing a similar job to me while living with the impact of a rare condition. Their positivity, energy, and drive is inspiring and a lot to live up to! |
5. | What advice would you give someone considering working in the rare disease space? |
| Talk to patients and talk to parents as often as you can. Never lose sight of the fact that they are the people that you are working to support, and their voice cannot be heard too frequently. |
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? |
| In a word, no. Fragmentation of any given rare disease patient population means that most fall through the cracks of local support – whether care for individuals, or for charities seeking funding. At the national level, aside from major concerns about the impact of leaving the European Union (I will not use that word…), there remain serious worries about how the NHS will find ways to pay for many of the new rare disease treatments on the horizon. While prices are high in many cases, it is crucial that companies also feel able to secure a return on their investment in rare diseases. If they cannot, we will see more and more companies moving out of orphan drug development. The government have to find a way to balance this very tough situation. |
7. | What would you say are some of the biggest motivators for your employees? Without doubt the whole team are motivated by running events, and interacting with patients, carers, and charity professionals. We are part of a vibrant rare disease community, and love spending time hearing about all of their work, successes, challenges, and exceptional persistence. |
8. | What are the toughest parts of being a CEO, and conversely what are the most rewarding? |
| Another charity CEO told me that in our sector, CEO stands for Chief Everything Officer, and I think this nicely summarises the challenges and rewards of the role. As CEO of a small team my job is to steer the ship, and plug the holes. If anything goes wrong, it is my responsibility to fix it, and deliver the work as quickly and seamlessly as possible – whether I know what I am doing or not! This can lead to some stressful moments, but also some huge successes. Luckily for me I am supported by an exceptional team at Findacure, so most of the time I get to bask in the reflected glory of their success! |
9. | What would be your one wish for Findacure for the year ahead? |
| The easy answer is a nice long-term sustainable source of funding – but this really goes without saying. Aside from this, I would like to see us gain recognition amongst the medical professionals who run rare disease specialist centres around the UK. This would give us fantastic connections with the UK’s rare disease specialists, but also help us to find more new founded patient groups, and more newly diagnosed patients seeking support. |
10. | If you weren’t CEO of Findacure, what was Plan B? What did 10-year-old Rick want to do as a job? |
| Well ten year old Rick probably wanted to be digging up dinosaurs. I never quite managed that, but I did something pretty close both by digging up 30 million year old fossil mammals in Spain, and writing a paper all about Ankylosaurs – so I certainly can’t complain! |
To find out more about the work of Findacure please visit
www.findacure.org.uk
www.findacure.org.uk
Author
RARE Revolution on behalf of The Humans of RARE
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