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Leveraging Data to Address Patient Scarcity in Clinical Trials: World Orphan Drug Congress

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Last week the World Orphan Drug Congress virtually brought together industry leaders, patient advocates, and researchers to discuss topics related to rare disease, including advanced therapies, clinical development, manufacturing and commercialization of cell and gene therapies, and AI and digital solutions.

Medidata’s Sheila Diamond, scientific business development, Acorn AI, and Alicia Staley, senior director, patient engagement, moderated a discussion focused on leveraging data to address patient scarcity in clinical trials. Attendees contributed to the stated goal of learning from each other on how best to address patient scarcity in rare disease trials. The conversation included challenges around patient scarcity in rare disease clinical trials and how data and advanced analytics can help mitigate these. 

The moderators opened three guided questions to the group:

  1. What challenges are you facing around patient scarcity in rare disease clinical trials?
  2. How can data and advanced analytics help mitigate these challenges?
  3. What trends are you seeing in leveraging ‘omics and historical clinical trial data to address patient scarcity?

1. What challenges are you facing around patient scarcity in rare disease clinical trials?

Communication emerged as a thematic challenge that affects key stakeholders across the clinical trial process for rare diseases—physicians, researchers, and patient advocacy groups can all benefit from more communication before, during, and after trials. Overall investment in disease awareness would help tremendously. Communication between companies and patients often begins too late—when enrollment starts—but it should function instead as an ongoing dialogue about the disease and commitments between companies and patients.

Ongoing communication can help address another major challenge for rare disease trials: patient enrollment. Regulators require a high number of patients per trial, which is difficult to achieve for a disease with a small population or a pediatric population. Compounding this issue, screen failure rates are quite high for rare diseases. With global trials, language and regulatory barriers further complicate patient outreach.

Patient advocacy groups and educational webinars help attract patients, but there are still hurdles with those approaches. There may be some competing patient advocacy groups not even aware of each other which could benefit from mutual alliances. And some patients do not use social media or engage with patient communities. Consequently, some patients end up siloed or limited by geography. So the question arose: How can the industry come together to help unify multiple silos of patients and advocacy orgs?

2. How can data and advanced analytics help mitigate these challenges?

The conversation highlighted a peculiar paradox for rare disease trials: The more personalized and precise data collected and ingested adds insights but also limits sub-populations. This makes it harder to identify and enroll patients in a timely manner since enrollment criteria becomes more specific. Industry participants said patient registries provide a lot of data for those who opt in, and data from care center networks adds value as well. Yet scarcity issues remain—patients are wary of switching from their current treatments to try new, uncertain ones.

For registries in particular, addressing patient concern is a priority. There’s a big push to capture data for registries but lots of regulatory and data protection around it. Registries are expensive to create and require future proofing along with mapped for FDA and EMA approvals. Patient groups are affected by the complexity of these requirements, and they get inundated with requests for registries. To ensure mutually beneficial outcomes, registries can provide education and communicate data insights back to patients and advocacy groups. Providing patients with data insights is essential for them to benefit from the value of registries.

3. What trends are you seeing in leveraging omics and historical clinical trial data to address patient scarcity?

Omics data can be aggregated and analyzed to understand disease subpopulations with different response characteristics to therapies, and there is the opportunity to leverage historical clinical trial data to maximize the number of patients who can receive the therapy being tested. 

Patients can be reticent to join randomized controlled clinical trials when they are uncertain of whether they will receive the placebo or the new experimental therapy. In addition, trials require a certain number of enrolled patients in order to maintain high scientific rigor needed to understand the effect of the new drug and this can be challenging to reach. In these cases, creating alternative/synthetic control arms from historical patient data can help improve future trials.

Alicia Staley chose five words to encapsulate the lively discussion: trust, education, awareness, connection, and collaboration. 


Learn more about how data and analytics can mitigate challenges for rare disease.

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