FDA’s Modernized Review Process: Improved Guidance Documents 
​​[Posted on: Thursday, March 1, 2018]

Last week FDA announced that it is modernizing multiple aspects of its review processes in an effort to make quick decisions, work collaboratively with sponsors and take patient perspectives into consideration for clinical trial design and market approval decisions. To provide examples of its new approach FDA released five new Guidance Documents for neurological disorders which were created by teams of FDA experts with diverse experience and contain precise, specific and clear suggestions. These new guidance documents are shorter, with one guidance document containing less than 3 pages of content while the longest one has about 7 pages of content. These documents are devoid of lengthy narratives that are characteristic of most earlier similar documents. Most of these disease specific new guidance documents address three broad areas for consideration when developing new drugs for either of these neurological diseases: (1) Brief outline of early development considerations such as safety in first in man trials, (2) specific considerations for the efficacy clinical trial design, and (3) General considerations for nonclinical studies, PK/PD data and labeling issues. The second category is the longest in these guidance documents. This reflects patient focus as the main area of interest and concern to patient populations is the design of efficacy trials, while other issues have traditionally been the interest of regulators such as the nonclinical studies but not patients. Patient groups who have always argued that too many resources are spent on exploratory phases or nonclinical studies should be happy with the new emphasis on efficacy trials. Two of the five new guidance documents relied heavily on draft guidance documents developed by the respective patient groups. FDA is working on several similar new guidance documents that will be released in the near future. Assuming the guidance documents are indicators for Agency-wide treads, it would safe to assume that FDA is shifting its review focus away from the traditional sequential approach of gradually amassing data from early stage trials, nonclinical studies and then pivotal studies to one where the pivotal efficacy studies take center stage and all other research becomes supportive, greatly increasing the speed of patient access. The announcement does not mention biologics and medical devices but the signs point to similar philosophical reorganization in those branches of the Agency as well. The conservative reviewers may argue that speeding up patient access at the cost of traditional nonclinical and early phase trials is a riskier approach but there are sufficient checks and balances in the system of post-market safety monitoring to protect the patients. And for unmet medical needs, patients are certainly very supportive of shorter development timelines. However, this faster process will likely not happen for most large blockbuster indications with multiple existing treatment options.