NOVEL's Latest Publication in Nature Biotechnology Analyzes Trends in Breakthrough Therapy Designations Approval Rates, Efficacy, Pricing and Market Access

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Novel Health Strategies announces publication of trends in Breakthrough Therapy Designations (BTDs) in Nature Biotechnology. This study was also selected for peer-reviewed presentations at ISPOR and HTAi annual meetings.

The BTD pathway is potentially a game-changing trend, with the FDA granting these designations to highly promising new products and dedicating more experienced resources to ensure faster approval and earlier access to much-needed therapies.

The US Food and Drug Administration (FDA) created a new expedited pathway called ‘breakthrough therapy designation’ (BTD) to facilitate rapid approval of therapies that have shown substantial activity in early trials. As of March 7, 2014 the FDA had received 155 BTD applications, of which 41 have been granted BTDs and 3 drugs with 4 BTDs have received marketing authorizations. The new pathway promises faster approval and earlier access to therapies for unmet needs. Despite the popularity of this new process, several areas of uncertainty still exist, especially the data requirements for the designation, the definition of “substantial improvement” and “existing therapies,” the scenarios for handling product failures, and the potential impact of the BTD on pricing and reimbursement.

The NOVEL team analyzed the BTDs, assessing the available data on safety and efficacy, mechanisms of action, type of molecule and indications. The analysis also included systematic review of available clinical and marketing data for the three approved products that have received BTDs.

This analysis was published in the April 2014 issue of Nature Biotechnology. The latest full list of breakthrough therapy designations is available at

The article covers the following topics:

  • Review of FDA's expedited pathways for drug approval
  • Trends in approval and denial of breakthrough therapy designations
  • Analysis of mechanism of action, indications and molecule type of granted BTDs
  • Pricing, reimbursement and market access of FDA approved and marketed BTDs
  • Systematic review of clinical, safety and outcomes data for all granted BTDs

This study was also peer-reviewed and selected for presentations at two upcoming conferences:

  • ISPOR 19th Annual International Meeting, May 31- June 4, 2014, Palais des Congrès de Montréal, Montreal, QC, Canada
  • HTAi, 11th Annual Meeting in Washington D.C, June 14-18, 2014

Details of these presentations is available upon request.

For more details about the BTDs or to request discussion with the author, please email at info(at)novelhealthstrategies(dot)com

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Julia Topaloglu, Vice President, NOVEL Health Strategies
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