“Don’t tell me what you can’t do, tell me what you can do”: Putting patients at the heart of the conversation
The MPS society is a large, UK-based rare disease organisation that operates internationally to support people with MPS, Fabry and related diseases. Here, Bob Stevens, group CEO of the MPS Society and chair of Rare Disease Research Partners (RDRP), explains his path into advocacy, the work of his organisation and the opportunities for change that he sees
“The MPS 2 (mucopolysaccharidosis type 2) diagnosis came in 2003 when doctors found my eldest son, Ollie, who was then two years old, had a heart murmur which was absent at birth. Both my sons were tested, and we were sent to Great Ormond Street for the diagnosis,” Bob explains.
When he and his wife, Claire, were told both their sons had this rare lysosomal inborn error of metabolism, it felt like their world had collapsed. The MPS Society’s support at this time of shock and crisis was game-changing, Bob says. It helped them to put their lives back together.
“Within days, we had someone who understood the disorder and, importantly, the effect of it on families—someone who did not judge us and who was there to help us through a grieving process.”
This vital support gave Bob a space where he could mourn the loss of his future dreams as a parent. “I realised very quickly that my two boys were never going to play rugby—a sport I am passionate about—and this triggered the feeling that I may not have anything to look forward to,” Bob explains.
On reflection, Bob realises it was healthy and vital to feel such sadness. And he realises how the support taught him about empathy—the glue that binds rare disease families together and is the driving force for advocacy groups.
Inspired, Bob gave up a career in construction and started his journey into advocacy, determined to make a difference for his sons and others affected by rare diseases.
That new direction started with a job in local government, where he supported adults with learning disabilities to find and maintain their employment opportunities. This role showed Bob what he could offer the disabled community and, more specifically, the charity sector. “It gave me the idea that I could use my business experience to help charities be in a better financial position, and that I could specialise in helping them to become viable.”
Soon afterwards, Bob met Christine Lavery, who had founded the MPS Society in 1982, and she proposed he become an MPS Society trustee.
It wasn’t long before Bob saw an opportunity for a new initiative at the MPS Society that would build on the support it was already giving to families. “We observed that patients in clinical trials were not having the greatest experience because their expected commitment wasn’t always cognisant of patient factors, such as mobility issues.” To address this unmet need, the MPS Society set up MPS Commercial, now called Rare Diseases Research Partners (RDRP).
“Rare Disease Research Partners is a clinical trial support company specialising in health technology assessments and providing patient-centred perspectives to pharmaceutical companies and other stakeholders,” Bob explains.
“We wanted to challenge the system to initiate and run clinical trials with patients at the heart of their design.”
Bob says that Christine, “a remarkable woman”, saw in him the next leader of the organisation. When she passed away in 2017, Bob built on her legacy and, with the kind support of mentors and staff, built the MPS Society to a staff of 60 people, who are all “helping to change the world one conversation at a time”.
He is proud that teams from the MPS Society and RDRP co-designed the first managed access agreement for an MPS treatment in the UK, enabled access to the first gene therapy in the UK with a patient submission, and prouder still that patients in clinical trials around the world are better supported.
Bob’s view is that looking at rare diseases through the same lens used for non-rare diseases does little to reduce the disease burden of individuals and families. Properly understanding the daily challenges posed by rare disease is essential, he believes. He challenges the adequacy of existing measures, such as the quality-adjusted life year (QALY): “That doesn’t tell me how much my son’s life is worth.”
What is a QALY?
- One quality-adjusted life year (QALY) is equal to one year of life in perfect health.
- A QALY weights each year of life a person is expected to have after a treatment or intervention with a quality-of-life score (on a 0 to 10 scale).1
He also strongly believes that the psychological burden faced by patients and families could and should be better understood. Patient advocacy groups, Bob says, can step into this void and generate additional data to fill it.
“Too many support systems are configured from the perspective of non-rare diseases,” Bob says, “for example, our families are often going through reassessment once they have completed disability forms because the forms are not designed for rare diseases.” This increases the burden of disease, he says.
“Living a rare life is a challenging one—getting through the day sometimes is just about as much as one can do.”
Bob would also like to see better collaboration between pharmaceutical companies and patient advocacy groups regarding clinical trials, because “the way they are currently organised does not account for the impact they have on people’s lives”.
“One of the things that scares me is being unable to recruit the right patients because they are trial- or survey-fatigued. We have a finite population, which is why we need to continually challenge people in industry to think about this,” Bob says.
Bob keeps close to his heart not only the prospect of his own sons’ future but the families whose loved ones have planted an oak tree in the MPS Society’s wood of remembrance in Sherwood Forest. To date, more than 400 trees have been planted in memory of people who have passed away from MPS. For Bob, planting remembrance trees on bereavement weekend is “one of the most difficult experiences each year”, but it is also a reminder of how the prospects of the community have improved.
“I am comforted by the fact this wood used to be called the Childhood Wood of Remembrance. Now, because of advances in care and treatment, people with MPS are living longer, and it is now known as the Wood of Remembrance and Hope.”
Bob’s sons, Ollie, 23, and Sam, 21, stoke his fighting spirit every day. “Sure, every day you fight and challenge, but the greatest lesson I have learnt from being a rare dad and advocate is to remember to live for today and hold on to all the joy it can bring.”
“Tomorrow is another day,” concludes Bob, adding that his mantra to stakeholders in the rare disease community remains: “Don’t tell me what you can’t do, tell me what you can do.”
 Glossary. NICE. Accessed March 8, 2023. https://www.nice.org.uk/glossary?letter=q
This digital spotlight has been made possible with financial support from Chiesi Global Rare Diseases. With the exception of a factual accuracy check, Chiesi Global Rare Diseases has had no editorial input or control over this article’s content, which was independently created by RARE Revolution Magazine, with the interview and writing support of Rhonda Siddall of Onrush Productions. All opinions are those of the contributor. RARE Revolution Magazine and Chiesi Global Rare Diseases are not responsible for the content of any external sites linked to within this article. RARE Revolution Magazine retains all copyright.