Reach the Market Smarter and Faster: Breakthrough Therapies, Orphan Drug Designation, Fast Track ... and the Right Strategy

by Diane Seimetz

“Breakthrough” and “Fast Track” are frequently heard buzzwords in the pharmaceutical arena. Looking at development times of up to 17 years from early R&D to product approval with further two years for pricing and reimbursement, it appears that the biopharmaceutical industry is rather on a “Slow Track”. Despite significant advances in medical science, there are still numerous patients without appropriate treatment options.

The US FDA has responded to this challenge already in 1997 by implementing the Fast Track provisions whereby important drugs can get to patients more quickly. In 2012 the FDA launched the Breakthrough Pathway to facilitate and expedite the development and review of new drugs for serious or life-threatening conditions. Following FDA’s designation of a product in a defined indication as breakthrough, the sponsor benefits from (1) frequent meetings, (2) intensive guidance on efficient drug development, (3) organizational commitment of senior managers, (4) opportunity for rolling review, and (5) priority review.

In the EU the EMA responded in 2016 by implementing its own breakthrough concept, named PRIME. PRIME stands for PRIority MEdicines and is intended to support the development of medicines addressing unmet medical needs. Sponsors of PRIME designated products benefit from early and enhanced dialogue with regulators at EU level and accelerated assessment of marketing authorization applications. As this program is still young, it is premature to conclude on the acceleration potential.

In the US the approval time of products that benefited from expedited pathways (2013 to 2015) was as low as 150 days compared to 370 days for products that were registered through the standard approval pathway. However, when looking at the median development times, there was no difference between products following an expedited pathway and products following the standard pathway.

This shows that expediting regulatory tools on its own have marginal effects on the overall development and approval time. It takes much more to reach the market smarter and faster. We at Biopharma Excellence have identified several important rules for biopharmaceutical companies to be smarter and faster. To name a few of them:

  1. Start with the end in mind,
  2. Understand where you can speed up and where you can’t,
  3. Benefit from the power of partnering,
  4. Understand why others failed and make it right the first time,
  5. Integrate your program,
  6. Learn from others who are fast (…)

If you would like to learn more about how YOU can be faster and smarter with your programs, please get in contact with us.

Diane Seimetz was invited by Max-Planck Innovation and Ascenion to present on this topic during the 11th Biotech NetWorkshop in Munich on 28-29 June 2017.