Sometimes I like to think that we’re making progress in the rare disease community. The “we” being the collective we – the interconnected and interdependent rare disease community that involves government, pharma, advocacy groups, health care providers, and of course, patients and their families. In fact, I was heartened back in 2012 at the US World Orphan Drug Congress when I kept seeing the visual of the patient at the center of the “patient-centered care” model.
I, in fact, don’t believe it should be patient-centered care, but instead, patient-partnered care. (see my original blog post about what we want from (big) pharma and the ensuing discussion on Forbes). Patients don’t necessarily need to be catered to and taken care of in a paternal way, but instead, we seek to become partners in our own care and in the care of our loved ones.
It is that model that I spoke of when I spoke at the World Orphan Drug Congress in 2012 (video below). And at that event, I was pleased to see the involvement of several other patient speakers and attendees.
But if you attend enough disease-specific or general rare disease conferences, the actual patient speakers are much harder to find. There are always representatives from larger patient advocacy organizations like NORD and Parent Project Muscular Dystrophy and parents of children with rare disease who are otherwise players in the rare disease space like John Crowley of Amicus Therapeutics and Wendy White of Siren Interactive, and my discussion is not intended to belittle their experience, work, and roles. Patient/caregiver involvement is growing, but it is still incredibly meager (with the notably exception being the Global Genes Patient Advocacy Summit, since that event is truly about the patient).
But at most conferences and events, where are the speakers who spend their days in and out caring for that child who may be the “market” that these companies are seeking? Isn’t that experience a valid perspective to be reminded of at the (now ever plentiful) rare disease conferences that often feature some of the same speakers year after year? There are a myriad of patients and parents with a story, a will, and valuable insight that would be worthwhile to tap into.
But maybe I’m asking too much? This is a commercial business after all.
Orphan drugs are such a hot investment right now in the pharma space that I fear…
– the role of the patient will be swallowed by the role of the dollar,
– the patient-centered model will be lip service and not life service,
– new companies entering the space will see the potential revenue long before they see the potential patient.
Don’t think that I’m too far off. Ask me how many people I’ve met working in rare disease in some capacity have never met a live person with the disease they are impacting. So even where we stand today, that needs to change.
Should I take comfort that it’s not just in rare disease that this is a problem? It is the entire health care system frankly. I have connected with a great number of people involved in promoting the role of the patient such as Regina Holliday and e-patient Dave deBronkart, among the most famous (I encourage you to follow their movement because it is really our movement). They have even succeeded in making “The Role of the Patient” as one of the 20 Great Challenges in Health & Medicine in the TEDMED program.
So I would challenge industry and patients to consider, what is the appropriate role of the patient in the pharma/biotech industry? Clearly there are some patients/parents who have vaulted into drug development themselves (probably most famously John Crowley, but of no less drive is Lori Sames‘ fight for a cure for her daughter’s rare disease GAN – Giant Axonal Neuropathy) so it’s not that we’re all too “emotionally involved” to make strategic business decisions or point research in the right direction.
From my perspective, patients/caregivers should be involved at each of these stages, whether in a formal individual role or a focus group. Companies and FDA should recognize that need and create a formal pathway for it to occur.
Do you agree? Why or why not? What are the barriers that have prevented it?
On that topic, I conducted a roundtable at this year’s World Orphan Drug Congress on patient involvement in clinical trial design and will report on those discussions in a later post.
I’d love to hear your thoughts.