It's an understatement to say that social media has made an impact on almost every aspect of rare disease. From patients being able to find each other quickly, to their ability to promote fundraising and advocacy campaigns, social media has opened doors that heretofore did not exist. For pharmaceutical companies, it poses a unique opportunity to listen and understand their patient communities better, but it also causes trepidation in some circles with respect to how it impacts their clinical development programs.
Rare disease patients involved in clinical trials do not suddenly sever their social media connections nor carve that clinical part of their lives out of conversations. Because so many rare diseases are lifelong, debilitating - and often in children, conditions that take up is a substantial part of the patient’s or caregiver’s life - social media is used for support, information sharing, fundraising, and a sense of community. In fact, being involved in a trial, in some cases, heightens a patient or caregiver’s involvement in social media because of the extensive or unique demands of a trial (creating a need for a subset of community support), their community’s desire for information about new potentially lifesaving treatments, and the desire for increased activism to move research forward at a faster pace.
Pharmaceutical companies may or may not prefer their clinical trial population to connect via social media, but there is little they can do to change it. The best practice would be to understand the potential effects of social media upon their clinical development programs and plan how best to proactively address them.
TRIALS - six ways social media impacts rare disease clinical trials
– Information about your trial – data, participants, side effects – will likely become public long before you publish the results. Whether it’s through blogs, twitter, or even patient testimony at FDA meetings, the broader patient community, investment community, and/or public will be able to find this information, good or bad, if they look hard enough.
– On the positive side, social media is an invaluable means through which patient communities share information about upcoming or ongoing trials with potential participants. It can also dispel myths for patients who don’t fully understand how clinical trials work (see my post on myths in clinical trials
). Unfortunately, social media can also spread inaccurate information that could hinder recruitment; thus, it’s important for sponsors to monitor social media as well as engage in concerted efforts to share accurate information about enrollment in their clinical trials.
– Rare disease clinical trial participants will
share information with one another and their disease community. In many cases, they will unblind trials, especially if their lives or the lives of their children hang in the balance of a drug development program. To expect otherwise is either patronistic, naïve, or both. As someone said recently at the Global Genes Patient Advocacy Summit, “some may look at trials as experiments, but patients look at them as treatment options.” So as long as sponsors anticipate this occurring, they can at least consider how this might impact their program.
– In certain cases, patient communities will use social media in advocacy campaigns related to an investigational therapy. One example is the “Race to Yes” campaign promoted by the Duchenne muscular dystrophy community to support accelerated approval for Sarepta Therapeutics’ exon-skipping drug eteplirsen. Garnering over 100,000 signatures and a response from the White House, one might correctly argue that patient advocacy through social media certainly helps some clinical programs. On a separate note, advocacy via social media has grown exponentially in the arena of expanded access or compassionate use. Sponsors with investigational drugs for devastating progressive disorders should expect that they may see these campaigns before they reach the NDA stage and be prepared for how they want to respond.
– In the course of information sharing via social media, patients have become very adept at discerning relevant insights that can be overlooked by clinicians and sponsors. Sponsors should consider implementing a formal feedback loop for patients/caregivers to share these insights, in addition to encouraging informal conversations between the investigators and trial enrollees to discuss such issues.
– Perhaps most importantly, it should be noted that participating in a rare disease clinical trial can be very challenging. Patients and caregivers need each other for support. It shouldn’t surprise anyone that most rare disease clinical trials have their own private online groups since these are the only people who truly understand the medical, emotional, and financial challenges, as well as the uncertainty, involved in participating in that particular trial. In certain cases where patients have been urged to isolate from discussing the trial because of implications that it might undermine the data, some have felt undue emotional isolation, even questioning their ability to remain in the trial. Sponsors and regulators must recognize that in devastating rare diseases, where the only support available is online support since no other patients live nearby, social media plays a key role, whether its discussing disease management, a clinical trial, or the emotional challenges of caring for a dying child.
Social media plays an important role in every rare disease community, one that can potentially impact clinical trials in both positive and negative ways. Keep these six avenues in mind when preparing for your next clinical trial, considering how to address any negative implications before they happen.
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