Great article by BioCentury’s Steve Usdin on the FDA’s new approach to facilitating appropriate and responsible expanded access to experimental drugs. Rather than buying into the political pabulum of “Right to Try,” the FDA is taking the reins with the right way to try.
Here are some snippets from the BioCentury article:
FDA to facilitate access to unapproved drugs
How FDA plans to help patients get expanded access to unapproved drugs
by Steve Usdin, Washington Editor
FDA plans to launch a new program in 2019 that will help patients gain access to unapproved therapies. The agency will field telephone requests from physicians and patients, streamline the application process, and act as an intermediary between physicians or patients and drug manufacturers.
Legislation is not required, and FDA has sufficient funding to conduct the pilot. Richard Pazdur, director of FDA’s Oncology Center of Excellence, proposed the initiative in early 2018.
The goals of the program, FDA Commissioner Scott Gottlieb told BioCentury, are to remove impediments that prevent physicians and patients from seeking access to investigational drugs and to communicate FDA’s support for manufacturers providing access.
An FDA internal working group has been meeting for two months to develop implementation plans and to iron out legal issues for the initiative, which FDA staff have dubbed Project Facilitate. The project involves the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).
Under the initiative, the agency will provide a telephone number that patients and physicians seeking compassionate use -- which FDA calls expanded access -- can call. FDA staff will answer calls and fill out the form required to apply for a single-patient IND request. It will send the completed paperwork to the physician for signature and then forward the request to the manufacturer.
Manufacturers will be expected to respond to requests within a specified time period. FDA has not yet determined the response deadline. Drug companies will continue to have the discretion to approve or deny requests, but for the first time “they’ll have to give the reason for denying access,” Pazdur said.
Legislation is not required, and FDA has sufficient funding to conduct the pilot.
Drug companies have an “obligation to consider expanded access, especially in areas of unmet medical need,” Gottlieb said.
In addition to streamlining requests and incentivizing companies to grant access, placing FDA at the center of the expanded access process will give the agency insight into demand, drug company behavior and outcomes.
Under the current system, drug companies have no obligation to report data to FDA or the public about the number of compassionate use requests they receive or grant, or about the outcomes patients experience. Under the new program, FDA will collect data on requests for unapproved drugs to help close the knowledge gap and make it easier to formulate policy. And if FDA learns that a drug company is receiving numerous requests for access to an unapproved drug, it may recommend that the company open an expanded access protocol designed for an intermediate or large population, or a clinical trial, Pazdur told BioCentury.
FDA’s expanded access program will, for the first time, create a systematic process in the U.S. for learning about the outcomes of access to unapproved drugs.
While biopharma companies often express concern that adverse experiences from expanded access may lead FDA to delay or derail drug reviews, the fear is largely unwarranted, according to FDA officials. The idea that expanded access will harm a development program is “urban lore,” Peter Marks, director of CBER.
An FDA review of expanded access data from 2005-2014 found two instances in which adverse events from expanded access contributed to FDA’s decision to impose a clinical hold. “It is very hard to find instances where something identified in the setting of expanded access raised questions that were an impediment to a review,” Gottlieb said.
Bob Temple, deputy center director at CDER, suggested that expanded access protocols can produce data that can demonstrate efficacy in populations outside those studied in registration trials, potentially leading to broader indications.
If sponsors create large non-randomized expanded access protocols, “they can actually use the information to expand the label,” Gottlieb told BioCentury.
The complete Usdin article is worth a read. One interesting take-away is that the FDA is now leading the conversation, controlling the playing field and searching for areas of convergence that reward advancing the health of not just individual patients but also the broader public health by rewarding appropriate industry cooperation.
The FDA's new program is a tremendous step in the right direction but many unanswered questions remain, such as who pays -- and how?
Stay tuned.
Here are some snippets from the BioCentury article:
FDA to facilitate access to unapproved drugs
How FDA plans to help patients get expanded access to unapproved drugs
by Steve Usdin, Washington Editor
FDA plans to launch a new program in 2019 that will help patients gain access to unapproved therapies. The agency will field telephone requests from physicians and patients, streamline the application process, and act as an intermediary between physicians or patients and drug manufacturers.
Legislation is not required, and FDA has sufficient funding to conduct the pilot. Richard Pazdur, director of FDA’s Oncology Center of Excellence, proposed the initiative in early 2018.
The goals of the program, FDA Commissioner Scott Gottlieb told BioCentury, are to remove impediments that prevent physicians and patients from seeking access to investigational drugs and to communicate FDA’s support for manufacturers providing access.
An FDA internal working group has been meeting for two months to develop implementation plans and to iron out legal issues for the initiative, which FDA staff have dubbed Project Facilitate. The project involves the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).
Under the initiative, the agency will provide a telephone number that patients and physicians seeking compassionate use -- which FDA calls expanded access -- can call. FDA staff will answer calls and fill out the form required to apply for a single-patient IND request. It will send the completed paperwork to the physician for signature and then forward the request to the manufacturer.
Manufacturers will be expected to respond to requests within a specified time period. FDA has not yet determined the response deadline. Drug companies will continue to have the discretion to approve or deny requests, but for the first time “they’ll have to give the reason for denying access,” Pazdur said.
Legislation is not required, and FDA has sufficient funding to conduct the pilot.
Drug companies have an “obligation to consider expanded access, especially in areas of unmet medical need,” Gottlieb said.
In addition to streamlining requests and incentivizing companies to grant access, placing FDA at the center of the expanded access process will give the agency insight into demand, drug company behavior and outcomes.
Under the current system, drug companies have no obligation to report data to FDA or the public about the number of compassionate use requests they receive or grant, or about the outcomes patients experience. Under the new program, FDA will collect data on requests for unapproved drugs to help close the knowledge gap and make it easier to formulate policy. And if FDA learns that a drug company is receiving numerous requests for access to an unapproved drug, it may recommend that the company open an expanded access protocol designed for an intermediate or large population, or a clinical trial, Pazdur told BioCentury.
FDA’s expanded access program will, for the first time, create a systematic process in the U.S. for learning about the outcomes of access to unapproved drugs.
While biopharma companies often express concern that adverse experiences from expanded access may lead FDA to delay or derail drug reviews, the fear is largely unwarranted, according to FDA officials. The idea that expanded access will harm a development program is “urban lore,” Peter Marks, director of CBER.
An FDA review of expanded access data from 2005-2014 found two instances in which adverse events from expanded access contributed to FDA’s decision to impose a clinical hold. “It is very hard to find instances where something identified in the setting of expanded access raised questions that were an impediment to a review,” Gottlieb said.
Bob Temple, deputy center director at CDER, suggested that expanded access protocols can produce data that can demonstrate efficacy in populations outside those studied in registration trials, potentially leading to broader indications.
If sponsors create large non-randomized expanded access protocols, “they can actually use the information to expand the label,” Gottlieb told BioCentury.
The complete Usdin article is worth a read. One interesting take-away is that the FDA is now leading the conversation, controlling the playing field and searching for areas of convergence that reward advancing the health of not just individual patients but also the broader public health by rewarding appropriate industry cooperation.
The FDA's new program is a tremendous step in the right direction but many unanswered questions remain, such as who pays -- and how?
Stay tuned.
