FDA Discusses Pilot Program for Single-Patient INDs

May 20, 2019

The FDA has launched a pilot program to help patients access needed investigational drugs outside of clinical investigations.

Project Facilitate, part of the agency’s Expanded Access program, features a call center to help oncology providers navigate the process of obtaining permission for their seriously ill patients with no other treatment options left, including clinical trials.

“Access to clinical trials and access to novel therapeutics for patients is still a problem,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence, at a public workshop on the pilot program last week. “We know that not all oncologists or healthcare providers have the regulatory expertise or resources to navigate these single-patient IND processes.”

The pilot program will give providers a single point of contact to guide them through the expanded access request process, helping them locate IRB resources, find the correct contact at the company that holds the IND for the requested treatment and complete the necessary FDA forms.

A public database tied in with the program also will list relevant information supplied by drug companies, including each company’s policies on expanded access.

A secondary aim of Project Facilitate, Pazdur says, is to identify pharmaceutical companies who decline or have policies denying certain INDs and determine their reasoning.

Under the FDA’s Expanded Access program, providers whose requests are approved have the status of physician-investigator—in a one-subject trial—and are required to follow all the agency’s rules for conducting clinical trials.

In fiscal year 2017, the FDA approved all but five of the 1,637 expanded access requests it received.

Read materials from the May 16 public workshop here:

Merck Flags Concerns on Widening Cancer Trial Eligibility
Merck is urging the FDA to look into potential safety concerns as the agency seeks to widen patient eligibility criteria for oncology drug trials of HIV, hepatitis B and hepatitis C patients.

In a comment on the agency’s draft guidance on expanding trial eligibility criteria, the drugmaker cites concerns over immuno-oncology (IO) drugs because of the potential for serious immune reactions and impacts on liver function in patients with active viral infection.

The company suggests adding to the guidance “the availability of adequate safety data” as a consideration when developing eligibility criteria.

In another comment, the American Cancer Society Cancer Research Network recommends the FDA modernize eligibility criteria to ensure any racial or demographic group is not excluded, for instance, “upper age limits, or excluding comorbidities more highly associated with demographic or socioeconomic subgroup unless specific rationale for exclusion exists.”

In total, the agency received nine responses in the public comment period, which ended May 13.

Read the draft guidance, Cancer Clinical Trial Eligibility Criteria: Patients with HIV, Hepatitis B Virus, or Hepatitis C Virus Infections, and comments here:

Icon Releases New Patient Engagement System
Drug development services provider Icon has launched its new patient engagement platform, a web-based system that helps improve patient enrollment and experiences in clinical trials.

The solution connects patients with specific study information and the investigative site closest to them, improving the patient recruitment capabilities of sponsors and sites by identifying potential study participants.

The system’s mobile-optimized website gives patients the ability to discuss possible trial participation as a clinical care option with family and caregivers at home. Additionally, the platform lets site staff view how many pre-qualification questionnaires have been completed as a means of tracking progress.

Sites Boost Economy Across All 50 States, Report Says
Clinical trial sites generated more than $42 billion in economic activity across the U.S. in 2017, according to a new study.

Business consultants TEConomy Partners LLC examined the activity of clinical trials by state, finding each of the 50 states, plus the District of Columbia and Puerto Rico, benefited in some part from $15 billion in direct investment by biopharmaceutical companies and an additional $27 billion in indirect expenditures, such as sites’ payments to vendors and suppliers.

The five states that experienced the greatest economic impact from active trials were California ($5.3 million), Florida ($4.6 million), Texas ($4.6 million), New York ($2.3 million) and North Carolina ($1.8 million). At the bottom of the list were Wyoming with just $100,000 and Puerto Rico with $500,000.

TEConomy based its estimates on enrollment data gleaned from and sites’ average expenditure per trial participant.

Read the report here: