RAPS is closely monitoring developments in the Coronavirus (COVID-19) outbreak. See our public safety page for the latest updates.

Regulatory Focus™ > News Articles > 2019 > 9 > GAO Reports on FDA, Drugmaker Efforts to Boost Access to Investigational Drugs

GAO Reports on FDA, Drugmaker Efforts to Boost Access to Investigational Drugs

Posted 10 September 2019 | By Michael Mezher 

GAO Reports on FDA, Drugmaker Efforts to Boost Access to Investigational Drugs

A new report from the Government Accountability Office (GAO) finds that drugmakers and other stakeholders are largely supportive of steps taken by the US Food and Drug Administration (FDA) to facilitate access to investigational drugs, both through clinical trials and via expanded access or right-to-try requests.
Specifically, the report looked at actions FDA has taken to broaden clinical trial eligibility, improve access to investigational drugs outside of clinical trials and how drugmakers communicate with patients about expanded access and right-to-try.
To complete the report, GAO surveyed 10 drugmakers and 14 other stakeholders and reviewed the websites of 29 drugmakers for policies related to expanded access and right-to-try.
Seven of the 10 drugmakers surveyed said that steps FDA has taken in recent years to simplify and explain its expanded access program have improved the program.
GAO also found that 23 of the 29 drugmakers’ websites it reviewed had policies about expanded access and/or right-to-try. Nineteen of the 23 drugmakers that had that information publicly available said “they would consider individual requests for access” from patients, though most said they would also require regulators to review the request.
Because right-to-try requests do not require FDA or institutional review board (IRB) review, this indicates that some drugmakers are more comfortable with FDA’s expanded access program than right-to-try.
The remaining six drugmakers all said they were in the process of developing policies for access to investigational drugs outside of clinical trials to publish on their websites in line with the federal requirement to do so.
The 19 drugmakers that did offer expanded access all required physicians to submit requests, but had varying additional conditions, including patient criteria, which drugs they would consider providing and notice that supply of a drug must be sufficient to meet clinical trial needs before expanded access requests may be considered.
Clinical Trial Eligibility
Despite FDA’s recent guidance on expanding clinical trial eligibility criteria for cancer trials, only one of the 10 drugmakers surveyed told GAO they broadened eligibility criteria for a study, while another said it was taking steps to do so. GAO notes that “these officials and others noted challenges to broadening eligibility criteria.”
FDA also released a guidance in June aimed at increasing diversity in clinical trial populations that makes recommendations for drugmakers to review each eligibility criterion and eliminate or alter any that are not necessary for assuring patient safety or achieving the study objectives.
GAO says the drugmaker that expanded eligibility criteria for its trial did so by “removing exclusions after determining they were not critical to clinical trial designs, including exclusions related to liver function, infections (e.g., HIV), and the use of other medications (e.g., steroids).”
The drugmaker that is looking to expand its eligibility criteria said it is doing so “partially in response to FDA’s 2018 pubic workshop report,” and is looking to include adolescents in an upcoming study “if they determine that patient safety would not be compromised.”
Both companies noted that expanding their trial eligibility criteria could lead to drugs being approved for a broader patient population and could ease the enrollment process by eliminating certain screening steps.
While the bulk of the drugmakers surveyed had not taken steps to broaden clinical trial eligibility criteria, six of the 10 remaining drugmakers said they have taken steps to reduce other barriers to participation, such as reimbursing travel and hotel costs for patients who need to travel to a trial site.
One drugmaker told GAO it is planning to conduct a “fully remote” pilot clinical study in the next two years with the aim to increase enrollment in rural areas.
Some of the drugmakers surveyed told GAO that broadening eligibility criteria could lead to challenges such as making trial results more difficult to interpret or delaying the start of a clinical trial as more time is needed to review individual eligibility criteria.
Expanded Access
According to GAO, most of the stakeholders interviewed for the report were familiar with steps FDA has taken to simplify the institutional review board process for expanded access requests.
The report also found that most stakeholders felt positively about FDA’s recently announced “Project Facilitate” pilot that features a call center for oncologists to contact for assistance with completing and submitting expanded access requests for single patient investigational new drug applications.
However, one of the drugmakers surveyed expressed concern that FDA’s increased efforts to facilitate expanded access could “intentionally or unintentionally pressure companies to make their investigational drugs available to patients.”
While seven of the 10 drugmakers surveyed said FDA’s updated expanded access guidance is an improvement, with one saying the guidance contributed to its decision to offer expanded access to its drugs, four of the companies expressed concerns about FDA’s use of adverse event data from expanded access treatment.

Regulatory Focus newsletters

All the biggest regulatory news and happenings.