These provisions have been conflated with the federal Right-to-Try (RTT) legislation that passed the Senate on August 3, 2017, which has garnered significant attention.

RTT attempts to increase access to investigational drugs by removing FDA and Institutional Review Boards (IRBs) from the equation. Critics of RTT point out that the FDA and IRBs provide important protections for patients. It is unclear what impact the RTT legislation, if passed by Congress and signed into law, will have on expanding access to investigational drugs because the decision whether to grant access remains with the drug manufacturer, which may be unable to provide access for a number of reasons (e.g., medical, ethical, legal).

The FDA has a process in place for expanded access (also known as “pre-approval access” or “compassionate use”), whereby patients with serious or life-threatening diseases can request access to investigational drugs, prior to FDA approval. Data from the FDA indicate that the Agency approves over 99% of these requests. Expanded access may be sought by a patient who has exhausted all available medical options and to whom a clinical trial is unavailable, either because there is not an active trial ongoing or because they do not qualify for the trial, e.g., due to the advanced nature of their illness.

FDARA takes a fundamentally different approach to access, addressing challenges in the drug development and clinical trials system that can exclude patients in the greatest need. The legislation requires FDA to examine the clinical trial system, specifically inclusion and exclusion criteria. These provisions aim to address how patients with unmet medical needs can be better served.

“A drug development process designed to accommodate a broader range of patients, either through clinical trials or multi-patient ‘expanded access trials’, would reduce the number of patients faced with the burden of requesting access,” said Nancy Beck, a director at Avalere. “Including patients with advanced disease or confounding conditions has the potential to provide valuable insight into how a drug will behave in a ‘real world’ population, once on the market”.

FDARA also reinforces the requirements set forth by 21st Century Cures Act passed in December 2016, which increased transparency around expanded access by requiring manufacturers to publish their expanded access policies within 60 days after enactment (February 11, 2017) or when the manufacturer first initiated a phase 2 or phase 3 study of the investigational drug. FDARA requires manufacturers of drugs that receive breakthrough, fast-track product or regenerative advanced therapy designation to post their expanded access policies within 15 days of receiving the designation.

These transparency requirements are a response to the frustration expressed by patients and physicians, who are unable to locate the appropriate information from manufacturers on how to request expanded access. Prior analysis by Avalere, which reviewed the websites of 100 publicly traded pharmaceutical and biotechnology companies, found that the number of companies posting an expanded access policy more than doubled from 19% in September 2016 to 47% in March 2017 in response to the requirements of the 21st Century Cures Act.

“The increase in transparent expanded access policies is very encouraging,” said Brenda Huneycutt, a vice president at Avalere. “Including transparency provisions in FDARA is another step toward reducing the burdens to patients and physicians looking to understand how to request access for a specific investigational product.”