1. Digital technology can improve healthcare.
During COVID-19, telemedicine visits went from tens of thousands to millions practically overnight, said Anne Pariser, MD, Director of the Office of Rare Diseases Research at NIH’s National Center for Advancing Translational Sciences.
“This had a real leveling effect in terms of access,” especially for patients who normally had difficulty traveling to see a doctor.
2. We must protect the Accelerated Approval Pathway.
Developed to allow for faster approval of drugs for HIV-AIDS, the Accelerated Approval Pathway allows limited, more focused clinical trials for drugs that treat rare, life-threatening conditions.
More than 250 vital drugs have been approved through the program since the 1990s, but it’s now under threat. “I think it’s important that people understand, it does not compromise rigor. So it requires that there be the same level of efficacy and safety for products that go through this pathway,” said Annie Kennedy, Chief of Advocacy and Policy at the EveryLife Foundation.
3. When sharing your patient perspective with federal agencies, "focus on the disease."
"Focus on that clinical context. Focus on the disease," and not necessarily a particular drug or development program, said Theresa Mullin, PhD, Associate Director for Strategic Initiatives, U.S. FDA Center for Drug Evaluation and Research (CDER). “It's probably better and more useful to focus on your experience.”
“Try to bring a diversity of perspectives, because we really do want to hear from the full range of the community,” she continued.