The truth about compassionate use access remains that after the passage of the Right to Try law, it is still very difficult to get access to investigational medicines, and there are unanswered questions on how to make expanded access feasible, cost-effective, and accessible. As Jess Rabourn, founder of the WideTrial, a non-profit that advocates for group-level expanded access programs, said at the first Expanded Access Summit that WideTrial organized last year, “far too few patients are benefiting from preapproval access.”
A year ago patient advocates began suggesting that medium- and large-size expanded access programs can produce data that can be used in approval applications and inform reimbursement decisions.
The upcoming second Expanded Access Summit, to be held in January 2019 in Washington D.C., will build on the discussion on whether expanded access may be the largest source of consented patient outcomes information, can help with further research studies, potentially be beneficial for regulatory claims, and improve provider care decisions.
The summit will feature Janet Woodcock, Director of FDA Center for Drug Evaluation and Research, and Peter Pitts, President of the Center for Medicine in the Public Interest, who will address how a combination of research trials and expanded access trials can be optimized to engage more patients into clinical development process, as per 2017 FDA Reauthorization Act. Other topics include whether Right-to-Try is a big sideshow, lessons from the first 30 years of regulated Expanded Access, and a roundtable on solving the barriers to wider access.
We believe that when patients are better educated about expanded access, the higher the probability is of finding a solution how to make expanded access accessible for all who need it. Wide Trial will provide 100 free tickets for patients and caregivers to attend the summit. More information about the summit is at https://easummit.net/.