Recent conversation regarding expanded access has focused heavily on the necessity of the FDA approval process. Right to Try legislation, which would remove the FDA from the expanded access process in the US, has been passed in 37 states and is now being considered at the national level.
However, many within the industry are now focusing more on the role of the drug companies, as it is the companies themselves who have the power to decide whether or not their products are provided through expanded access.
Because there are no rules forcing companies to provide their products, nor are there any industry-wide principles helping them navigate the expanded access process, there is significant variation in company policies and procedures.
The 4 Key Expanded Access Program Strategies
To address this issue, a recent meeting, held by The Engelberg Center for Health Care Reform, brought together a range of industry stakeholders to discuss potential organizational strategies all companies should adopt moving forward. The group was made up of leaders from pharmaceutical companies, bioethicists, a senior FDA representative, and a patient advocate. Together, these individuals agreed on four key principles for companies to follow, which they termed ‘The 4 A’s’: Anticipation, Accessibility, Accountability, and Analysis
Read More: An EAP Versus A Clinical Trial
It was agreed that a critical first step for companies should be to anticipate the need for an expanded access program, especially when developing drugs for severe, debilitating diseases. As part of these efforts to anticipate, companies should develop a written expanded access policy which clearly outlines how they plan to deal with expanded access requests. The policy should include the inclusion and exclusion criteria, an outline of the decision-making criteria, appropriate contact information, and a defined time frame for request responses. Companies should also designate a decision maker or decision-making team who will be responsible for reviewing every expanded access request.
Once a policy is in place, the meeting attendees agreed that the next step should be to make this policy accessible to all individuals who may qualify for expanded access. Under the 21st Century Cures Act, all companies with drugs in phase II or phase III clinical trials are now required to post their expanded access policy on their website. However, the act does not specify where. As such, companies should carefully consider the most appropriate place to feature the policy, so that it is easy to find by visitors to the site. It is also important to make sure the policy is written in a clear manner, that is jargon free, so that patients of any education level can understand it.
Just as important as making the policy accessible, is making sure the policy is carried out as promised every time. Companies are accountable to the patients sending expanded access requests. Companies must ensure that they review requests following the specified review criteria and must respond within the specified timeframe.
Finally, the group agreed that an important fourth principle should be to release an analysis of the data from an expanded access program, where possible. Encouraging companies to share anecdotal or preliminary safety and efficacy data, as well as lessons learned from conducting their programs, should foster internal analysis and consequent improvement.
To find out more, click here to access the full meeting report.