Executive Summary

While the US FDA works to cut time necessary to approve expanded access applications, sponsors are creating delays by mandating patients and institutions sign contracts to gain access.
Timetable at the Central Station

Efforts to streamline the US Food and Drug Administration’s expanded access program may be hindered by legal protections that the drugs’ sponsors require of prospective patients and their providers.

Agency officials have been trying to reduce the time and burden necessary for patients and physicians to gain access to unapproved therapies, usually through a single-patient investigational new drug application. However, while the agency has cut the required paperwork and is preparing to launch a program to help physicians navigate the pathway, among the most time-consuming parts of the process remains sponsor requirements that patients sign contracts before access is approved.

During a 16 May public meeting on the FDA’s Project Facilitate, the agency’s new expanded access call center, stakeholders discussed bottlenecks and challenges with the request process. Patient contracts with drug sponsors was one area identified where action is needed, although no ideas were offered during the meeting.

Contract requirements were initially raised as a misperception about the burden of expanded access, but later discussion suggested the issue was a legitimate problem for providers and patients.

A Novartis AG representative said during the workshop that the contract takes the most time when completing an expanded access application, even though the company has tried to reduce it to a two-page letter of agreement.

Contracts usually include liability and indemnification statements and outline when drug access may end, such as when the product is approved or new safety information is available. Delays usually occur as the contract is negotiated between the company and hospital or other provider institution.

The contract hurdle may not be an area that FDA can streamline directly. Sponsors retain the final say on whether to grant expanded access to their products. Agency officials acknowledged during the meeting that more stakeholder education of the situation is necessary.

Project Facilitate is intended to help ease the bureaucratic burden on patients and physicians when submitting an oncology-related expanded access application and by extension open the program to many more patients. (See sidebar.)

The program is expected to launch by the end of May with FDA staff available to answer questions about the process and help requestors complete the necessary forms. The agency also wants to use Project Facilitate to gather efficacy and safety data, along with reasons companies deny applications. (Also see “‘Project Facilitate’: US FDA Plans May Meeting On Expanded Access Call Center” – Pink Sheet, 15 Mar, 2019.)

Confusion Over Expanded Access Eligibility

Expanded access eligibility also was raised as a source of confusion.

Ellen Sigal, chair and founder of the Friends of Cancer Research and chair of the Reagan-Udall Foundation board of directors, said some patients try to use expanded access to circumvent clinical trials, which is a problem for drug sponsors.

“Many patients and many companies have told us when we did our first round of feedback that many of the patients they’re getting are eligible to be on the trial,” she said. “If a patient wants the drug and they don’t want to go on a trial, but they’re eligible, they’re not going to get it through this mechanism. So it’s important to do education on this as well.”

A Pfizer Inc. representative said during the meeting that the company’s expanded access policy includes consideration of whether the patient is eligible and has access to clinical trials for the drug.

Education is among the top priorities for the FDA’s expanded access program, not only for patients, but also sponsors. (Also see ” Expanded Access: US FDA Told To Increase Regulatory Clarity, Early Interactions With Sponsors” – Pink Sheet, 8 Nov, 2018.)

FDA Official Says Eligibility Should Be ‘Looser’

Companies likely would prefer patients not gain drugs through expanded access if they can receive them in a formal clinical trial, where much more data is collected in a more controlled setting.

Some stakeholders worried that expanded access programs were being treated like clinical trials from an eligibility standpoint.

Gideon Blumenthal, deputy director of the FDA’s Oncology Center of Excellence, said there should not be strict requirements because expanded access generally is reserved for patients that are excluded from traditional clinical trials.

Sponsors were required in the 2016 21st Century Cures Act to post their expanded access policies on their websites. The Reagan-Udall Foundation aggregated the available policies into a navigator on its website. (Also see “Reagan-Udall Expanded Access Navigator Ready To Launch” – Pink Sheet, 11 May, 2017.)

“Oftentimes with the single-patient INDs, these are for patients who are traditionally excluded from clinical trials either because they can’t access the site or because of some overly restrictive eligibility criteria,” Blumenthal said. “The eligibility criteria in general should be much more looser for single-patient INDs.”

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