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Self-reporting of health data by patients to a community through online registries has become more prominent in recent years and has the potential to inform biopharmaceutical companies’ development of therapeutics. This is especially true in the rare-disease space, where biopharma companies can find a heavily engaged patient community that may help overcome unique drug development challenges.

  • Facilitating Trial Recruitment: A significant barrier to research and development in rare diseases remains the inherently small size of the patient populations, making recruitment for trials difficult and expensive. However, availability of internet-based, crowdsourced data breaks down geographic barriers, allowing biopharma companies access to significantly greater data sets and patient registries which can help identify and stratify patients for trial inclusion.
  • Decreasing Patient Burden: Barriers to participation in online patient and data registries are very low for patients, requiring only access to an internet connected device. Reducing visits to physicians and other healthcare providers can greatly reduce the burden of clinical trial participation on patients, potentially increasing their enthusiasm to enroll in a trial. Ease of use of online platforms may increase patient compliance.
  • Identifying Unmet Needs: Self-reported patient data may allow biopharmaceutical companies to identify trends in off-label treatments, adverse reactions to medication, or unmet needs in a patient population. Reduced costs of data collection may allow drug developers to devote more resources to data analysis and research and development.

The use of online registries has been validated in several rare diseases. A study utilizing the online registry DuchenneConnect showed that data from the registry displayed a clear advantage of the use of steroids to delay loss of ambulation in Duchenne muscular dystrophy patients. Multiple clinical studies over forty years support this finding, demonstrating the ability of an online registry to provide accurate, actionable insights to support drug development. The researchers, also utilizing registry data, identified a potential therapeutic benefit for several supplements and with validation from their steroid finding believe that these compounds warrant further exploration.

The use of lithium carbonate in the treatment of amyotrophic lateral sclerosis (ALS) was at one point a topic of fierce discussion between physicians and patients; a small scale study showed it slowed disease progression. Investigators, curious to answer whether lithium carbonate could slow ALS progression, utilized data from the crowdsourcing website patientslikeme.com to assess this question. The study could not replicate the previous small trial’s results, a finding that subsequent large scale clinical trials confirmed. Beyond repudiation of lithium carbonate as a treatment for ALS, this case study also demonstrates that crowdsourced data can be used to provide fast, inexpensive, and reliable results.

As the popularity of personal health and fitness wearables expands, the data available to biopharma companies through online patient registries will only continue to grow and become more informative. While not eliminating the need for clinical trial validation, online registries and crowdsourced data provide a useful inexpensive tool for biopharmaceutical companies seeking to develop therapies for rare diseases by assisting in hypothesis generation, identifying novel opportunities to bring drugs to market, and informing treatment guidelines.

Our reports on Amyotrophic Lateral Sclerosis and Duchenne Muscular Dystrophy are available.

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