A Year in Review for Expanded Access
A full rotation around the sun has brought us to the close of another successful year of expanded access. 2017 has brought many advances for Expanded Access, further pushing the field into its full potential to help patients gain access to investigational therapies in the case of serious or life-threatening diseases.
In July, the Reagan-Udall Foundation of the Food and Drug Administration launched the Expanded Access Navigator. Along with guidance on expanded access for patients, caregivers, and physicians, this essential tool is a one-stop-shop for the expanded access policies and contact information for pharmaceutical companies. Companies with their policies listed on the Expanded Access Navigator are considered compliant with the 21st Century Cures Act.
On August 3, led by Senator Ron Johnson (Wisconsin), the Senate unanimously passed the S.204 Trickett Wendler Right to Try Act of 2017 which has been linked to the Goldwater Institute's similar endeavors in passing right-to-try laws in 38 of the 50 United States. The bill allows for physicians requesting access to investigational medicine to bypass FDA in the request for investigational medicine and instead interact solely with the pharmaceutical company offering the drug. The passing of this legislation was followed by the Hearing on Examining Patient Access to Investigational Drugs held by the subcommittee of Energy and Commerce on October 3. The progress made within the expanded access environment was highlighted through the efforts of institutionalized working groups, the FDA, and pharmaceutical companies. The FDA's rapid response rate in granting access and efforts to ease the application process for requesting drug was brought up to further present improvement in the area. The frustration that many faced in the process of expanded access was acknowledged, but solutions such as the Reagan Udall Navigator, the shortened expanded access application, and industry guidelines where brought forth. The bill now sits in the house pending a vote.
The same day of the October hearing, came published updates to Expanded Access to Investigational Drugs for Treatment Use- Questions and Answers: Guidance for Industry The updates offered clarification on the significance of expanded access adverse event data, acknowledgement of the expanded access clause of the 21st Century Cures Act, and new exceptions when it comes to IRB approval of an individual patient expanded access IND. The FDA requires a submission of a results summary after treatment of individual patient expanded access. The summary must include all adverse effects and serious and unexpected adverse reactions only if they are relevant to the investigational drug. FDA expressed an understanding of the conditions that expanded access is granted upon and clarified that because the design of expanded access programs is not intended to measure drug efficacy, it will be rare for the approval of an IND or the progress of a clinical trial to be interrupted due to adverse events brought forward through expanded access. The guidance also introduced a waiver which would allow the bypass of a full IRB review in the case of individual patient expanded access when the physician making the request has been granted accord by an IRB chairperson or designated IRB member.
With expanded access gaining greater momentum across the pharmaceutical industry members of the community joined together to further drive this energy through conversation and dialog. Experts in the field gathered to discuss, debate, and learn the multi-faceted characteristics of expanded access at conferences such as the Drug Information Association Annual Meeting in Chicago, the Pre-Approval Access Conference in Philadelphia, the Managed Access Programs in London, the Wide Trials Conference in Boston, and the FDA/CMS Summit in Washington D.C.
Further compliance to 21st Century Cures Act and the FDA Final Rule have been seen within the biopharmaceutical company network. Increasingly, pharmaceutical companies have been posting their expanded access polices on company websites, providing guidance to physicians and patients on the process of submitting a request for investigational drug. Clarifications have been made on the turn around time after the submission of an expanded access request. Links to Expanded Access listings on www.ClinicalTrials.gov have been made available.
2018 holds much progress for Expanded Access. With educational workshops, conferences, and meetings having been planned for the year, 2018 will see fewer burdensome discrepancies associated with the process of applying for expanded access. With more widespread knowledge and greater use of available resources, companies are more likely to implement their own expanded access programs. This may be the year for national Right to Try legislation. Changes are on the horizon and Early Access Care is here to assist pharmaceutical companies in the optimization of solutions for Expanded Access programs.