Rare Diseases: The Role of Social Media in Patient Recruitment

Since the passing of the Orphan Drug Act in 1983, the rate of drug development in the United States has seen a significant increase. The act directly impacted funding – a major challenge associated with rare disease drug development. This in turn has led to a number of life-changing treatments for patients and an increasingly lucrative market for drug developers. However, there is still much more work to be done. Rare diseases impact 30 million people in the United States alone —more than the number of patients diagnosed with AIDS and cancer combined. Out of the 30 million patients, 95 percent do not have an FDA-approved treatment and at the current rate of drug development, it is estimated it could take over 450 years to treat every rare disease.

Mark Baglin, vice president and product strategy team leader at Shire Pharmaceuticals and Kevin J. Anderson, associate director of Global Clinical Operations at Alexion Pharmaceuticals, led a really interesting “Rare Disease” track during the recent Festival of Genomics in which they explained the unique challenges and opportunities in rare disease drug development. According to the speakers, unlike with big pharma, patient involvement plays a vital role in rare disease R&D and clinical trial design. Here we take a look at ways to best leverage physician and patient recruitment when it comes to rare disease drug development.

Patient Recruitment

The most obvious challenge facing the rare disease industry is recruiting patients and principal investigators (PIs) for clinical studies. Treating physicians don’t fall under a rare disease bucket, making it difficult to know what doctors should be involved. Because many of the PIs used for these trials are considered key opinion leaders within the field of rare disease, they can be expensive and harder to work with due to their demanding schedules. Furthermore, there are a limited number of experienced PIs outside of the U.S. and Europe, which limits the globalization capabilities of a trial.

Another major issue is it can be extremely challenging to find patients given the rarity of the disease. To gather meaningful data, patient recruitment requires the participation of several sites across many regions, as 50- to 75-percent of sites will not enroll a single patient and if they do, the average is typically only one patient. Delays in the recruitment process not only add costs but also create uncertainly around the feasibility of the program.

To leverage patient recruitment in clinical trials, traditional methods need to be altered to employ a more patient-centric approach. Patient recruitment should depend of the prevalence of a particular rare disease in a region, rather than launching sites across several regions waiting for patients to enroll. This not only decreases costs, but also is more efficient than the current method.

Patient Involvement

Historically, the patient’s greatest barrier to understanding the prevention, diagnosis and treatment of rare disease is an insufficient knowledge of the mechanisms and history of the disease. The key to developing patient knowledge is open communication. Doctors need to share the mission with the patient and help them understand all elements of disease research and treatment, including funding and patient services. Rare disease patients tend to be the most vocal and well informed and often have the support of active advocacy groups. Partnering with these groups and ensuring transparency among developers and site investigators is key to maintaining involvement.

SEO and Social Media

As enrollment rates continue to drop even as more clinical trials are introduced, it is clear that clinical trial coordinators need to develop new approaches to enrollment. Search engine optimization and social media offer the ability to alter the landscape of clinical trial recruitment. Social media is becoming increasingly important in filling the missing health information, particularly surrounding experiences using particular therapies and associated side effects. According to the Ogilvy Healthworld report “Connecting the dots: Which Pharma Companies are Succeeding in the Social Media Space,” the average number of tweets by pharma companies has increased by 530 percent since 2013.

But followers aren’t enough. The report shows that companies with the most effective engagement and that generate the most interest do not necessarily have the largest community. Smaller biotech companies need to take advantage of this opportunity and use social media and SEO for site and patient recruitment. Several companies have found recruitment success by reaching out to Facebook groups that may not have sufficient funding to form their own advocacy organizations and connecting with the organizers about becoming patient advocates. PatientsLikeMe is another online resource that many patient advocacy groups turn to and can serve as a useful outlet for recruitment efforts.

Developing a rare disease drug is no easy task, but the returns offer great rewards. Developers still face hurdles when it comes to funding and showing value to payers who now more than ever are looking for ways to save costs on drugs. For patient recruitment, patient advocates and social media can prove to be a valuable source when used effectively.