FDA Expedited Program

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The FDA Expedited Program is an accelerated approval process for drugs from the American FDA (Food and Drug Administration).

Drug approvals are usually a multi-year process. Accelerated approval procedures (English for expedited program ) of the FDA allow novel drugs for serious or life-threatening diseases to be made available to patients more quickly and thus to avoid delays. It may also allow diseases to be treated more quickly if there is no adequate therapy for them.

The aim of the accelerated approval by the authorities is to make new drugs available to patients more quickly in order to be able to better treat serious and life-threatening diseases. The treatment of diseases for which there is still no adequate therapy can be made available to the patient more quickly with novel drugs. To find out whether a drug can better treat serious diseases, the influence z. B. determined on the survival rate or observed whether the course of the disease worsens, this should remain without therapy. The FDA differentiates between four different types of accelerated approval:

  • Fast track
  • Break Through Therapy
  • Accelerated Approval
  • Priority Review

The pharmaceutical company is filing an application with the FDA to obtain one of the four accelerated approval processes.

Fast track

According to Section 506 (b) of the FD&C Act ( Federal Food, Drug, and Cosmetic Act ; 21 US Code § 356), novel drugs can receive fast-track approval if they have a potential, either as a single drug or in combination with other drugs is used to treat life threatening diseases. Theoretical considerations or explanations of the mechanism of action based on non-clinical data can be used for this purpose in order to obtain fast-track approval. The FDA responds within 60 days.

Break Through Therapy

According to Section 506 (a) of the FD & C Act a new drug status is Breakthrough Therapy , awarded (Engl. For, breakthrough in therapy ') when so serious conditions can be treated and this already in the first preliminary data from clinical studies and over previous standard therapies could be shown. The new drug shows z. B. a significantly improved response rate than the comparator. Examples of drugs with Break Through Therapy status include: a. Atezolizumab from Roche , blinatumomab from Amgen , ceritinib (LDK378) from Novartis , ibrutinib from J & J / Janssen, nintedanib from Boehringer Ingelheim , pembrolizumab (formerly lambrolizumab) from MSD Sharp & Dohme and Sofos Sciencesbuvir from Gilead & Dohme . The FDA will respond within 60 days as to whether the drug meets all the requirements for accelerated approval. If all conditions are met, z. For example, the review of documents is accelerated, meetings with the authorities are increasingly being scheduled or senior managers and very experienced examiners are used to accelerate market approval. Was introduced Breakthrough Therapy Designation in 2012, with obinutuzumab (formerly Afutuzumab) from Roche, the first monoclonal antibody with the status Breakthrough designation was, which was approved by the FDA in-2013.

Accelerated Approval

According to 21 CFR part 314, subpart H, 21 CFR part 601, subpart E and Section 506 (c) of the FD&C Act, a drug can receive accelerated approval if the positive benefit for a drug can possibly only be determined after years, but you can already sees a positive benefit in a surrogate endpoint (e.g. laboratory data). The time until the FDA responds is not specified.

Priority Review

Medicines that are re-submitted after a refuse-to-file (English for 'rejection of a submission') can be given priority review status . Medicines for the treatment or prevention of certain tropical diseases can also be given. The FDA responds within 60 days.

Individual evidence

  1. ↑ a b c d e f g h i j Guidance for Industry Expedited Programs for Serious Conditions - Drugs and Biologics. (pdf) FDA, accessed on July 6, 2014 (English).
  2. ↑ a b c d Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review , FDA website, accessed November 27, 2017
  3. ↑ a b 21 US Code § 356 - Expedited approval of drugs for serious or life-threatening diseases or conditions. Cornell University Law School, accessed July 9, 2014 .
  4. ↑ Amgen Receives FDA Breakthrough Therapy Designation For Investigational BiTE® Antibody Blinatumomab In Acute Lymphoblastic Leukemia. Amgen, accessed July 6, 2014 .
  5. ↑ Breakthrough Therapies. Friends of Cancer Research, accessed July 6, 2014 .
  6. ↑ Roche active ingredient obinutuzumab (GA101) receives accelerated approval process from the FDA for use in previously untreated chronic lymphocytic leukemia (CLL) , Roche PM dated July 3, 2013, accessed on November 27, 2017
  7. ↑ Breakthrough Therapy Approvals FDA, accessed July 24, 2015.