White Paper Shows Role of Ongoing Patient Engagement to Improve Rare Disease Clinical Trials, Costs, and Success Rates

WASHINGTON, Jan. 23, 2025 /PRNewswire/ -- The Global Genes Corporate Alliance, an initiative that brings together biopharmaceutical companies and academic institutions working to improve access and treatments for people with rare diseases, today released a white paper that shows the critical role ongoing patient community engagement through the discovery and drug development process plays in improving the cost, time, and success rates of clinical trials for rare disease therapies. 

The white paper, Early and Often: Reimagining Patient Community Engagement to Improve Clinical Trials Feasibility, argues that by adopting a patient-centric approach, biopharmaceutical companies can enhance protocol designs, improve recruitment and retention, and increase the likelihood of rare disease clinical trials succeeding. The white paper is available to download for free at globalgenes.org. It represents more than a year-long effort of the Global Genes Corporate Alliance and included the participation of members of Global Genes' Global Advocacy Alliance.

Drug developers often face clinical trial problems because of difficulty recruiting and retaining study participants. This can delay patient access to needed medicines and have economic consequences to drug companies through costly clinical trial protocol amendments, longer times before realizing revenues, and erosion of their opportunity for market exclusivity. 

Often, these delays result from a lack of understanding of what it is like to live with a specific rare disease, the daily challenges people with a condition face, and how clinical trial protocols may affect the willingness or ability of people to participate in a study. By engaging with the patient community before critical decisions about a therapy and studies of it are set, companies can identify obstacles and address them before they are put into place.

"Rare disease drug developers continue to waste precious time and millions of dollars because of their failure to engage early and often with the patient community," said Wendy Erler, Vice President and Global Head of Patient Experience, Patient Advocacy, and Patient and Caregiver Insights for Alexion Rare Disease and Co-Chair of the Global Genes Corporate Alliance. "This white paper makes a strong business case for why integrating the perspectives of people living with rare diseases into the discovery and development process will save time and money, ensure drugs are meaningful to patient populations, and improve enrollment and retention rates in clinical trials by addressing unnecessary barriers and burdens for participants."

Members of the corporate alliance undertook this effort to develop a tool to educate colleagues about the importance of regular engagement with the patient community and how that can better inform rare disease drug development. While patient organizations vary widely in their focus, they are becoming increasingly sophisticated in drug development. Many organizations are directly involved in research and are hiring veterans from the biopharmaceutical industry to help de-risk drug development in their disease.

Rare disease patient organizations are investing in natural history studies, biomarker identification, registries, and animal models for their diseases. By leveraging their expertise and the resources they have developed, drug developers can gain insights into the best clinical trial sites for treating people with their disease, specific challenges patients with a condition may face, and identify inclusion and exclusion criteria or protocols that may unnecessarily make it difficult to recruit participants. 

"Engaging the patient community throughout the development of medicines for rare diseases is critical. By doing so, we stand to ensure that our research addresses real-world needs, limits barriers to participation in clinical studies and ultimately delivers better medicines that provide better outcomes for patients and their families," said Edward M. Kaye, M.D., CEO of Stoke Therapeutics. "This white paper shows how doing the right thing for patients is good business because it drives efficiencies, saving time and money by aligning all those involved around a common goal."

The white paper shows:

• Early patient engagement is essential: Early collaboration with patient advocates can provide valuable insights into disease characteristics, patient needs, and potential barriers to trial participation.
• Patient insights save time and money: Without patient engagement, companies may not know which sites to prioritize, leading to delays and slow start-up times. Investing in thorough patient engagement can mitigate these risks.
• Qualitative insights matter: Real-world patient experiences can reveal critical factors affecting trial design and participation. They can help identify and address obstacles that may deter participation.
• Companies can make wrong assumptions when they don't ask: Patient insights can reveal unexpected preferences or concerns specific to a rare disease population that may not be apparent to researchers without direct community engagement.
• Industry needs to expand the roles of their patient advocates: Patient advocates working for biopharmaceutical companies should be recognized as more than a marketing function. These professionals should be able to sit at the table with colleagues across various functions within a company, be heard, and engage with them throughout the discovery and development process to inform it with patient insights.

Such an approach improves drug development and fosters trust and collaboration between industry and patient communities.

"Too often, drug developers wait until they are ready to begin enrolling participants in a clinical trial before they engage with a patient community. By then, they have established the protocols for a study, selected clinical trial sites, and made other decisions without benefitting from the unique insights the patient community can provide," said Charlene Son Rigby, CEO of Global Genes. "This white paper serves as a call to action for biopharmaceutical companies to rethink their strategies for working with patient advocacy organizations and embrace early and ongoing collaboration throughout the discovery and development process."

About Global Genes 
Global Genes is a 501(c)(3) nonprofit rare disease patient advocacy organization dedicated to providing patients and advocates with a continuum of services to accelerate their path from early support and awareness through research readiness. Using a collaborative approach that involves biopharma, researchers and advocates with data as a central core, Global Genes also enables research and data collection through the RARE-X research program. Through this effort, Global Genes is building the largest collaborative patient-driven, open-data access initiative for rare diseases globally.

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