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3 Ways Patient Voice is Impacting Rare Disease Research, According to Advocates

The patient voice transformed rare disease drug development with the Orphan Drug Act (ODA), a pivotal law that de-risked rare disease research and development (R&D). Four decades later, the voice of rare disease advocates remains a vital part of the development of drug development and ensuring that new drugs and the trials that support their approvals meet the real-world needs of patients.


In 2022, Syneos Health surveyed a select group of leaders from large and small rare disease patient advocacy organizations to better understand their priorities, perceptions and activities related to incorporating the patient voice into the drug development process. Here is what we found out:


  1. Connect with advocacy groups. According to advocates, the best way to communicate with the community about clinical trials is to leverage the tools and channels already provided by the advocacy organizations, including email, social media and community events. They also suggest that companies offer greater guidance to clinicians around how to talk to patients about trial participation early in their disease journey.
  2. Partner to conduct more diverse research. Almost all rare disease advocates prioritize increasing diverse participation in research yet are at different stages of implementation maturity. Of survey respondents, 47% have established and 43% are actively considering programs designed to identify, train and prepare community members to engage with researchers and clinicians to support this kind of innovation and increase participation and diversity in clinical trials.
  3. Acknowledge advocates. Rare disease patient advocacy groups want to be recognized by industry for their time and expertise; 30% of organizations said they did not receive any financial, acknowledgement or other recognition when helping industry with R&D. More than one organization noted that they refuse to work with contract research organizations (CROs), or other third-party research organizations hired by pharma/biotech that do not compensate them for their contributions.

It is incumbent upon the biopharmaceutical industry to actively seek out opportunities for collaboration and partnership early and often throughout the clinical trial development process—from inception to  execution and beyond—to support patient communities, and to design trials and deliver new treatment options that align with patient priorities and realities.


Are you interested in learning more about how patient advocacy groups are influencing clinical development? Explore the Syneos Health Patient Voice Consortium and read more from our experts on rare disease.

Raymond A. Huml MS, DVM, RAC
Vice President | Medical and Scientific Strategy
Head, Rare Disease Consortium
Syneos Health


Keri McDonough MA,
Vice President | Medical and Scientific Strategy
Head, Patient Voice Consortium
Syneos Health


Nermina Nakas MD, MPH
Executive Medical Director | Medical Management
Syneos Health


Meredith Wilson
Management Supervisor
Syneos Health


Jordan Pietz
Director | Scientific Strategy
Syneos Health

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