Scott Gottlieb testifies at FDA nomination hearing by Senate Health, Education, Labor and Pensions Committee.
The Food and Drug Administration is touting the agency’s efforts to streamline and beef up a longstanding program designed to provide access to unapproved drugs outside of clinical trials, more than five months after President Donald Trump signed a law designed to provide access to such drugs outside the agency’s purview.
In a statement Thursday, FDA Commissioner Scott Gottlieb wrote that over the last year, the agency has taken steps to expand and update the expanded access program, in response to feedback from members of Congress and others on how to make it more effective. These include streamlining the supporting documentation that doctors must submit in order to gain access to unapproved drugs, reducing application time to around 45 minutes and a single file attachment. Furthermore, while offering a drug under expanded access used to require review from an entire Institutional Review Board, requests now require review from only one IRB member.
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Expanded access, also known as compassionate use, enables patients to access investigational drugs when they do not meet the eligibility criteria for clinical trials. Patient advocates and other experts have pointed out that the FDA approves 99 percent of expanded access requests and reviews most of them within 24 hours.
Additionally, in response to the Right to Try Act that Trump signed into law shortly after its passage on May 30, the FDA also announced Thursday the launch of a page on its website designed to help patients understand that pathway. The law has drawn criticism from patient advocates and other experts due to the bypassing of regulatory oversight that it enables and potential for Right to Try treatment protocols to interfere with drugs’ clinical development and FDA review.
Despite expanded access’s record for approvals and review times, the libertarian Goldwater Institute has called the program “so rigged with bureaucracy and disincentives that it is bound to fail in most cases,” as it said in a February 2016 article that nevertheless linked to another article noting that the application time had already been reduced to 45 minutes. Furthermore, one of the chief proponents of the law, Republican Sen. Ron Johnson of Wisconsin, wrote in a June letter to Gottlieb that the law was intended to diminish the FDA’s authority.
In order for their products to qualify for Right to Try, companies must have passed them through Phase I clinical development and have initiated a trial to test them for efficacy or have an active application to do so under FDA review. The products must be unapproved and in active development. However, Right to Try removes a number of the FDA-mandated safeguards that are required under expanded access, and drug companies are not required to honor requests made under the law.
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One company that has sought to take advantage of the law is Oceanside, California-based Therapeutic Solutions International, which said May 31 that it would provide access to its StemVacs dendritic cell vaccine for cancers under Right to Try, adding in September that it would use Phase I data for the vaccine to allow for that.
The company said in its September announcement that the data showed no treatment-associated adverse events and, in some cases, tumor stabilization and immune stimulation. “This Phase I safety data strongly supports our continued clinical development of StemVacs, of which part of the development pathway includes providing patients access to it now under the Right to Try Law,” CEO Timothy Dixon said in a statement. The data were for 10 patients with various advanced cancers and were collected at the Pan American Cancer Treatment Centers, in Tijuana, Mexico. The company did not respond to a request for additional details about the data.
Photo: Zach Gibson, Getty Images
