Expanded Access – It Continues to Make Headlines

Expanded Access – It Continues to Make Headlines

The recent Senate passage of S. 204, the Trickett Wendler, Frank Mongiello, and Jordan McLinn Right to Try Act, demonstrated that interest in expanded access (“compassionate use”) continues unabated.  Both seriously ill patients who want to “try” an investigational medicine before it is approved by FDA and companies developing the medicines face challenges that can make the process of access difficult or impossible for both.

Those challenges are the subject of the upcoming (September 15, 2017) WideTrial 2017 Expanded Access Summit in Cambridge, MA.  The foundational question for the Summit is what stakeholders across the spectrum think are best practices, challenges, and solutions to a goal of making expanded access a viable and contributory part of the drug development process.  Could centrally managed “Expanded Access trials” be used to engage groups of patients who are not candidates for clinical research trials but who are within the targeted disease population or range of related populations?   Such programs would be based on established protocols and designed for meaningful numbers of patients.   A “wide trial” protocol would accommodate patients who generally would not be eligible for a clinical research trial– sicker patients, for example, or those with difficult to manage symptoms or co-morbidities.  Data could be collected from these “wide trials” that, while perhaps not directly relevant for determining the approvability of a product for the intended patient population, could inform the design of further clinical trials, identify high-responder subgroups, and provide useful additional information for the drug labeling.

The purpose of the WideTrial Summit is to conduct an in-depth analysis of whether this approach could be workable, as well as to answer key questions, such as the practicalities and the value of a third party managing such trials; the reimbursement landscape for patients receiving an investigational medicine outside of a company’s clinical trial, including the costs associated with administering the medicine, even if the medicine itself is free; pragmatic considerations such as trial design, use of data, informed consent and privacy, integration of the wide trial into the development program, and even shipping investigational medicines.

Summit speakers include representatives from FDA and NIH, academia, patient organizations, law, and the biopharmaceutical industry.  It is hoped that a best practices publication will come from the Summit, which could be especially helpful for smaller companies struggling with the best ways to respond to the needs of very sick patients.

The Summit sponsor, WideTrial, is built to serve as a platform for conducting expanded access programs.  The organization grew out of the ALS Emergency Treatment Fund.  Two of the patients after whom S. 204 is named, Trickett Wendler and Frank Mongiello, were affected by ALS.  Ms. Wendler succumbed to the disease.

 

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