Oct 21, 2015

About the author

Dr. Carol Stephens

Dr. Carol Stephens has over 30 years of experience in drug development and support with focus on regulatory planning, negotiation, execution, and compliance. In her spare time she likes to design new gardens and spoil her dog Moxie.

Topic tags

Accelerated Approval Pathway, Breakthrough Therapy Designation, drug development, expedited development, expedited programs, Fast Track Designation, FDA, Priority Review Designation, regulatory, regulatory authorities, review programs

5 Things You Need to Know About Expedited Drug Development

When you are looking for ways to make drug development as effective and efficient as possible, take advantage of all the acceleration options from regulatory authorities. They may be more helpful than you think.


The trend is unmistakable

Increasingly, FDA and similar regulatory agencies are offering a range of expedited development and review programs, and companies are applying for and receiving the benefits. In the last few years, an average of approximately 60% of recent drug development has been in therapies approved by FDA for at least one of the expedited programs. Of recently approved drugs, about a third qualified for two or more of the programs.1


The options are diverse, and the benefits useful

FDA’s four expedited programs for drugs treating serious conditions represent the largest opportunities for most companies. Fast Track Designation may be granted for a qualified infectious disease drug or one with nonclinical or clinical data demonstrating the potential to address an unmet medical need. Breakthrough Therapy designation requires preliminary clinical evidence indicating the drug may demonstrate substantial improvement in a clinically significant endpoint(s) over available therapies. These designations confer more frequent FDA interactions, potential for rolling reviews, and eligibility for accelerated approval and priority review. For Breakthrough Designation, companies receive intensive FDA guidance on their drug’s development program. For the Accelerated Approval Pathway, companies can use and gain approval based on a compelling surrogate endpoint or an intermediate clinical endpoint reasonably likely to predict a drug’s clinical benefit. The Priority Review Designation allows shorter submission review clock of 6 months vs. the usual 10-month standard review. The FDA has developed additional incentives, some quite substantial, for rare disease development.2


You can get started earlier than you think

You can request Fast Track and Breakthrough Therapy Designations as early as Phase I. Requests for Accelerated Approval Pathway come a bit later due to the need for efficacy endpoint data. Priority review requests occur with initial marketing application or efficacy supplement. In all cases, discuss timing with the FDA. Note that for all of these except Accelerated Approval, the FDA agrees to respond to the request within 60 days. For a regulatory agency, that is a speedy turnaround time indeed.


Note the caveats

Before applying, make sure you have a sound scientific and medical case and you meet the criteria. Keep in mind that some of these designations are under pressure and may result in regulatory authorities becoming more cautious in granting use of the programs in the future. For example, some drugs approved based on surrogate endpoints under the Accelerated Approval Pathway were subsequently pulled from the market when new studies showed the drug was not efficacious and increased mortality or morbidity. Recently, a major medical journal assessment criticized FDA’s frequent use of expedited programs for drugs that are not as innovative as some believe they should be.3


Professional guidance makes it happen

There are many criteria and nuances to these and other expedited programs at the FDA. Other countries, including the EU, are adding their own acceleration options.4 Talk to your regulatory experts about which one or ones may be applicable to you. If you don’t have an in house regulatory department, Novateur can provide seasoned regulatory affairs professionals to help. Entering an expedited program as early as possible is one of the fastest and most efficient ways to create a positive value inflection.

Highway by Caleb George Morris


1 Data from the New York Times at http://www.nytimes.com/2015/05/02/upshot/speedy-drug-approvals-have-become-the-rule-not-the-exception.html?_r=0

2 FDA guidance on expedited program for serious conditions at http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf. Many other documents are available covering other FDA incentive programs.

3 The most recent notable assessment is from the British Medical Journal found at http://www.bmj.com/content/351/bmj.h4633

4 For European programs, see http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2015/07/news_detail_002381.jsp&mid=WC0b01ac058004d5c1

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