A new paper by a team of FDA officials finds that only about 30% of drugs requested for expanded access go on to be approved by the agency.
“Expanded access provides just that: access. There is no guarantee that the product sought will be effective and/or safe, much less that it will be effective and/or safe for the particular patient,” the authors write.
In recent years, the debate over patient access to unapproved drugs has risen to the national stage. So called “right-to-try” laws, also known as right-to-ask laws, seek to bypass FDA to provide access to unapproved drugs and have been enacted in 37 states. In August, the US Senate passed a bill that would expand right-to-try nationwide, though the House has yet to consider the bill.
The debate focuses on a patient’s right to access unapproved drugs to treat serious or life-threatening diseases outside of a clinical trial. Proponents of right-to-try have argued that FDA is a barrier to patient access, while FDA has countered that it approves nearly all requests for access it receives.
Under FDA’s expanded access program, physicians can request access to a drug for individual patients or a group of patients under an expanded access investigational new drug application (IND) or via a new protocol under an existing IND.
While FDA maintains that clinical trials are the best option for patients to get access to unapproved drugs, the agency acknowledges that it’s not always possible for a patient to enroll in a clinical trial.
FDA has also said that in most cases pharmaceutical companies deny requests for expanded access, often citing supply or safety concerns. Right-to-try proponents have countered that drugmakers turn down such requests out of fear that adverse events that occur because of expanded access use could derail their development programs or open the company up to other liability issues.
Expanded Access and Approval
According to the authors, FDA received 6,054 unique expanded access requests between FY2010 and FY2014. After excluding expanded access requests for multiple patients and cleaning the results for duplicate and “nonsubmitted” INDs, the authors were left with 5,394 unique requests, 5,298 of which were allowed to proceed.
Of those, the authors were able to identify 408 unique drugs and fixed-dose combinations.
By 30 September 2015, 122 (30%) of those drugs had been approved by the agency for at least one indication, though not necessarily for the indications sought in expanded access requests.
Six of the top ten most-requested drugs went on to be approved, though the paper does not mention the names of those drugs because of confidentiality reasons.
Looking at it from another direction, 3,365 of the 5,298 requests were for drugs that later went on to be approved, with a handful of drugs accounting for a large proportion of those requests.
The authors also found little evidence to support the argument that adverse events that occur as a result of expanded access treatment can jeopardize a drug development program.
“Over the last decade, spanning almost 11,000 expanded access requests, there were only 2 instances in which a clinical hold was placed on commercial drug development due to adverse events occurring under expanded access. In both instances the development of the drugs continued after these issues were addressed and the holds were lifted,” the authors write.
And the authors cast doubt on the argument that liability concerns keep drugmakers from providing expanded access to their products. A search of three legal databases, Google Scholar and HeinOnline turned up no results for product liability suits against drugmakers for personal injuries as a result of expanded access treatment.
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email firstname.lastname@example.org for more information.
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