FDA Rapid Approval Pathways

VIA IDEA Pharma by Partha Ghosh

The Food & Drug Administration (FDA) published new draft guidance on expedited programmes for serious conditions in June 2013, intended to facilitate and expedite development and review of new drugs in serious/life-threatening conditions with unmet medical need. The four available programmes are described, compared and the application processes outlined.

The designations can only be applied to drugs used to treat serious conditions, defined as those “…with morbidity that has substantial impact on day-to-day functioning and where there is an unmet need…” i.e. there is no treatment, there are serious outcomes despite approved therapy, there are benefits for those unable to tolerate/resistant to approved treatment, or better overall benefit/risk. The FDA has also stated that it would generally like to see a choice of treatments in serious conditions, so more than one treatment for a condition may attract a form of expedited review and/or approval.

A comparison of the programmes available is given in the table below (Click for larger version):


Fast Track Designation

  • FDA provides provision for additional meetings to discuss study design, safety data requirements, dose-response biomarker suitability.
  • Products may subsequently be granted priority review.
  • Rolling review allows FDA to review sections of the marketing application before all sections are completed.

Breakthrough Therapy Designation

  • There is clinical data to support a treatment effect, representing a potentially substantial improvement over available therapy in a credible number of patients.
  • FDA would prefer to see preliminary comparative data if other therapies are available (placebo, otherwise)
  • FDA will interact with the sponsor to design an efficient clinical development programme (smaller studies, more efficient design, reduction in patient exposure to less efficacious treatments) although there must still be adequate efficacy & safety data overall.
  • FDA will involve reviewers intensively and assign a cross-disciplinary project lead.
  • FDA can suggest application for breakthrough therapy status if the sponsor has not yet requested this.

Accelerated Approval

  • This is usually granted contingent on the sponsor agreeing to conduct additional post-approval studies to verify the clinical benefits of the drug. Such studies should be part of the overall development plan and can commence prior to accelerated approval being granted.
  • There is a lack of alternative treatments.
  • Most relevant to chronic conditions where an extended period of time would be required to show the intended clinical benefit, often in oncology.
  • The endpoint can either be a surrogate endpoint likely to predict clinical benefit or a clinical endpoint (that in itself is not of clinical benefit, or provides a modest or short-term benefit) that can be measured earlier than a relevant irreversible outcome (morbidity or mortality based).
  • FDA can withdraw approval if post-approval results fail to confirm the clinical benefits; the drug is shown subsequently to not be safe or effective; post-approval commitments are not fulfilled.

Priority Review

  • This may require an expedited manufacturing development programme to meet the expedited timelines. There may also need to be modified nonclinical study and clinical trial inspection programmes.

The programmes give FDA and sponsors distinct routes to bring therapies that bring significant new patient benefit to market ahead of the standard timeline. There is a need to balance making new therapies available earlier versus the potential for safety signals to be missed and the early promise of a new drug not being maintained. The increased pace and richness of interaction with FDA reviewers should be welcomed by sponsors but may place a heavy burden on staff, both at the FDA and in applicants’ regulatory departments. However, if patients with serious, life-threatening diseases have access to significant, new treatments then this should improve many lives.

Original post here. Thank you, IDEA Pharma, for contributing!Image


Official blog of: INTERPHEX New York - www.interphex.com
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