Recently, we discussed the importance of connecting with advocacy groups in order to create more trial diversity. As a result of this connection, well-organized patient advocates are influencing the drug development industry, particularly in orphan diseases. Not-for-profit patient advocacy groups have demonstrated the power to affect significant change, even impacting therapeutic development and approval against expert recommendations.
A meaningful patient advocacy strategy is no longer a nice-to-have, fictional idea for pharma companies; it’s a must-have in order to increase diversity among their trials.
This blog tracks the origins and rise of patient advocacy groups and discusses milestones in the FDA’s acceptance of patient advocacy groups over the past 30 years.
Where it Started: AIDS Activists of the 1980s
After years of government inaction with regard to the AIDS epidemic, people living with HIV/AIDS and their advocates created local and global activist movements in an effort to change public perception and response to the disease. Activists fostered this movement through:
- Adopting the practices of other social movements
- Educating themselves to become their own researchers and lobbyists
- Using fear and anger to impact public perception/action
- Mobilizing, so the public viewed them as real people rather than statistics
Broader visibility and understanding of the disease forced pharmaceutical companies, the U.S. government, and regulatory agencies like the Food & Drug Administration (FDA) to open a dialogue with patient advocates, allocate funding, and expedite a response to the epidemic.
Highly successful, the AIDS activist movement provided a playbook for other patient advocacy groups in healthcare to use for years to come.
A Force to be Reckoned With
Within two years of two major AIDS activism events – a 1988 rally at both the Department of Health and Human Services in Washington, D.C., and the FDA headquarters in Maryland, and a 1989 protest at New York City’s St. Patrick’s Cathedral led by members of ACT Up – the FDA began recruiting patient advocates into its Patient Representative Program.
The Patient Representative Program recruits and trains patient representatives who have experience with diseases. Today, it has voting members sitting on FDA advisory committees and panels covering more than 300 diseases and conditions.
Patients now have a substantial role in the development and progress of potentially life-changing treatments. The FDA even celebrates collaborative milestones as part of its patient advocacy history.
The following list highlights major post-AIDS advocacy initiatives implemented by the FDA to allow patients to have a greater impact on a disease treatment’s regulatory process.
- 1993: The first FDA patient representative serves on an advisory committee.
- 1996: FDA patient representatives receive advisory committee voting rights.
- 2001: FDA begins seeking patient input during early development of medicinal products; FDA patient representative role expands to include consulting to scientific and regulatory reviewers.
- 2008: Patients and consumers are encouraged to report medical product problems using the existing FDA MedWatch system.
- 2012: Safety and Innovation Act is enacted, requiring the FDA to “develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions.” Multiple patient engagement programs launch as a result, including the FDA Patient Network, which facilitates patient engagement with agency decision-makers, educates people on the developmental process of new medications and promotes innovation.
- 2015: FDA issues treatment guidance for Duchenne muscular dystrophy based on recommendations composed by the disease community, marking the first time an external advocacy group (Parent Project Muscular Dystrophy) significantly impacted U.S. government drug policy.
- 2018: Patient Engagement Collaborative (PEC) launches with the Clinical Trials Transformation Initiative (CTTI).
- 2019: Patient Affairs Staff (PAS) online webform and Patients Ask FDA launches.
A New Culture of Drug Development
Today, patient advocacy groups are no longer serving only patients and caregivers – their most meaningful collaboration is now with the healthcare industry. Drug developers are also embracing these groups to increase patient engagement.
The mechanisms through which patient advocates create momentum continue to evolve but still center on social impact. Bloggers, YouTubers, and social media influencers are establishing their reputations and using their expertise not just to raise awareness but to sit on advisory boards, speak at disease-related events, and co-author research papers.