The clinical endpoint, or the outcome used to judge a particular clinical situation and designate whether therapy was delivered to the patient/subject, can be difficult to define for rare, life threatening, and chronically deteriorating diseases. When studying a new drug intended as therapy for these kinds of indications, there may not be a clear clinical endpoint other than eventual survival. Therefore, using a surrogate endpoint may be the only way to determine if a drug is efficacious in a cost-effective manner. The National Institutes of Health defines a surrogate endpoint as “a biomarker intended to substitute for a clinical endpoint.” Reliance on surrogate endpoints to develop products for serious, life-threatening conditions could decrease the development time to market, if the product qualifies for the accelerated approval pathway.
The FDA currently has an accelerated approval process to facilitate the approval of products to treat serious diseases, and that fill an unmet medical need based on a surrogate endpoint, in the hopes that drugs can be marketed faster to treat these populations. However, some believe that drugs aren’t being marketed timely enough and more can be done to help move the process along. Senator Kay Hagan has recently proposed the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act to market the accelerated approval considerations in the hopes that more people have access to drugs to bring them to a better state of health. Information can be found within Senator Hagan’s website, http://www.hagan.senate.gov/, which includes Senator Hagan’s objectives for the TREAT Act. The TREAT Act would amend the Food, Drug, and Cosmetic Act to include a section on accelerated approval. TREAT would add that a product may be approved upon a determination it has an effect on a surrogate endpoint through epidemiological, pathophysiological, therapeutic or other evidence developed using biomarkers. The anticipated goal is an expansion of accelerated approval to attract more use and bring more treatments to market. The bill also directs FDA to issue draft guidance within a year to implement its amendments and to follow the draft guidance with a final guidance within a year. This bill appears to be bringing attention to the accelerated approval process, and making amendments to generate more knowledge and enthusiasm within the product development industry. According to some background information on the site, “The TREAT Act does not authorize any government funding and is not expected to receive any funding through the appropriations process. It’s to transform FDA’s internal processes to ensure more consistency.”
For more information on the TREAT Act, go to http://www.hagan.senate.gov/files/TREAT_ACT_Background.pdf
For more information on the regulatory strategies to develop therapies that treat serious diseases, go to http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm