Ever since I began, 25 years ago, to want to raise the profile of my rare disease – adrenomyeloneuropathy (AMN) - I’ve been perplexed about how to have a meaningful conversation with a pharma company.
Patients are not usually expert in medicine, science or in the way pharmas work. But they know that they play interlinking roles in developing solutions that could lead to effective therapies or even cures.
Social media has only become a tool that patients can use within the last 10 years. Patient forums – such as the AMN Easier one I look after on HealthUnlocked – have now become vital mechanisms where patients can help each other and also form collectives to press for change. Fellow-sufferers from four continents have embraced AMN Easier. The site offers useful poll results derived from patients as they navigate the forum. Even though they are not rigorous, in the absence of data from other sources, these ought to be useful to doctors. For example, one poll captures the kinds of symptoms that patients experience, and displays this as a pie chart. For a newly-diagnosed patient, such data can help alleviate fears that symptoms are unusual. But my attempts to encourage doctors and pharmas to engage with the forum have largely been unsuccessful. Of course, doctors and pharma personnel are busy people, but I suspect that the real reason has more to do with incentives, or the lack of them.
Rare diseases are now a hot topic for pharmas as they seek new revenue streams. Such diseases appear to have much potential for drug research, often being monogenetic in origin. Yet when I approached one large pharma to determine if my disease was one it was addressing and offering to help, I learned a harsh truth: unless a body of clinical trials is already being pursued, pharmas aren’t interested. Companies develop solutions but do not generally engage in original research. Without evidence that research is happening elsewhere, there is sympathy but not much else.
My attempts to understand my own disease has led me down some interesting paths. I’ve learned that “rare diseases are not rare” as the former Chief Medical Officer, Sir Liam Donaldson stated in 2009. I have also learned that 52% of cancers are also rare.
But the really exciting discovery has been that many rare diseases – and many common ones as well - are linked in hitherto undocumented ways because they share a common disease mechanism. Otherwise unrelated diseases are revealed to have things in common at genetic, pathological or clinical levels. Patients dealing with the condition can therefore discover that their disease may not be so rare after all.
I am therefore optimistic that a new initiative I have developed to explore this phenomenon and to further research in rare and more common diseases might fuel real progress. A prototype system to facilitate discussion among biochemists and geneticists and to capture data about such commonalities is under development, sourced entirely by willing volunteer effort. One pharma has provided a tiny amount of goodwill funding.
Once the prototype is a reality, the next big step will be to use social media to help us move from this pilot project to a live system. The challenge will be to enable scientists, patients, doctors and pharma personnel to collaborate in launching new research to explore these common mechanisms. And I shall be looking for social media to be a key driving force to make this happen. Scientists interested in helping will need to be found. Funding needs to be gathered, and crowd-sourcing begins to look like a very viable option. I want patients to pressurise funders and politicians through Facebook and Twitter. And hopefully pharma will look on, comment and engage.
What’s also interesting is that out of this mix, it may be patients that best understand how social media can help. Patients – ordinary people – use these technologies. Doctors, with some exceptions do not – they are too busy and lack the time and skills. Scientists are too focused on the specifics of their research. But new generations of medical experts and scientists are entering the workforce with the ability to use these systems efficiently. Will pharma be part of the picture?
Chris Ogden is a patient advocate and management consultant.
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