Questions Raised About the Benefits of the Accelerated Approval Pathway
(Thursday, September 8, 2022)
An independent study found that more than 76% of the drugs approved under the accelerated approval pathway (AAP) in the US did not demonstrate any additional therapeutic benefits compared to the previously available treatment options for the same indication. Also, the US FDA approved nearly three times as many drugs under this pathway compared to the European Medicines Agency (EMA) without much enforcement of the post-approval study requirements. The “Therapeutic Value” of a new drug is defined as an additional benefit of the medicinal product over an appropriate comparator in terms of improvement of symptoms and other measures of benefit such as the cost of treatment, etc. The Therapeutic Value of a new drug can be rated, and scores assigned based on various factors, and is a critical factor for a new drugs continual availability to the patients. The US does not have an equivalent system to evaluate drugs post-FDA approval. In the US, all drugs approved under the AAP need to conduct additional post-market studies to further evaluate the benefits of the drug approved under this pathway. However, first, studies conducting a head-to-head comparison of the new drug to the comparators are rarely required, and second, the timelines for the post-marketing studies are rarely well-defined or enforced. Hence, most drugs approved under the AAP stay in the market for years demanding high prices for treatments with no proof of significant benefits over other available (and mostly cheaper) options. With almost 84% of AAP approvals for cancer drugs, and only 35 of the 146 drugs approved under AAP having a demonstrated therapeutic value, it translates to patients getting treated for life-threatening diseases with expensive new drugs where another available option would suffice. The independent study, authored by researchers at Harvard University, raises critical questions about the health economical aspects of the AAP compared to its equivalent pathway in the EU, the Conditional Marketing Authorization (CMA). Unlike the drugs approved under AAP, those approved under the CMA pathway must submit the Therapeutic Value justification for their product to practically stay in the market. The authors suggest a similar measure for the US for the drugs approved under the AAP. Particularly, the authors argue for a stricter enforcement of the post-market study requirements. In the recent past, FDA’s accelerated approval program has frequently come under negative publicity by questions about its potential benefits. The AAP is credited with approval of many drugs, particularly for oncology indications, and likely have benefited the patients by providing additional treatment options, even if equivalent to previously available drugs. However, AAP has also been gamed to get FDA approval for drugs that did not show significant benefit. And it is fair to critique such drugs. The irony is that such arguments should come from the FDA, but they don’t. Although, the US does not have a system to consider the therapeutic value for approval of drugs, this study could be used by payors to object to covering drugs approved under the AAP, particularly if the cost difference between an AAP drug and a comparator is significant.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC