Monday, October 4

 

Evidence Led Decision Making for Clinical Proof-of-Concepts

8:30 am Morning Coffee and Short Course Registration *

9:30 - 11:45 Short Course

Instructors:

  • John Arrowsmith, Ph.D., Science Director, CMR International
  • Philip Miller, Ph.D., Product Strategy Manager CMR International

The R&D based Pharmaceutical Industry is currently faced with major issues around revenue generation and productivity. Consequently it has been actively adopting a variety of strategies, aimed at maintaining its long term viability as the provider of new breakthrough medicines to address unmet medical needs. Such strategies include managing cost by downsizing and outsourcing, increasing access to new drugs and technologies through partnering, acquisitions and mergers, bringing a greater internal focus on productivity through re-organizations into Business Units and Research Units combined with a narrowing of scope to fewer therapeutic areas and finally, to extract the maximum value from existing assets through investing in emerging markets, in drug repositioning, in generics/biosimilars and in diversifying the portfolio to encompass biologics/vaccines, consumer health, animal health and diagnostics.

These strategies are being adopted as means to maintain revenue streams and to mitigate some risk, but fundamentally they are being driven by the low output of new medicines from the R&D divisions of Pharma. Later in this paper some science led approaches to cracking this R&D productivity issue will be discussed.

Despite the huge increases in the investments made in Pharma R&D over the last decade or so, productivity, as measured by the NDA approval of new molecular entities, has remained fairly constant at around 20+ per annum. This level of productivity is not sufficient to sustain an R&D based pharmaceutical industry that relies heavily on NME’s to drive future revenue streams. Phase 2 survival is the Achilles Heel of R&D productivity since it has always been the phase with the lowest survival rate. Only about 1 in 4 drugs entered into a Phase 2 proof of concept study (POC) will successfully transition into Phase 3; and of these only about 50% become an approved drug. Companies that claims “improved or high productivity” by bolstering the size of it’s portfolio in Phases 1 and 2, have not addressed the real issues involved in delivering a positive POC and progressing those successes to Phase 3. The short course will discuss not only the key criteria that underpin quality PoC decisions, but also strategies that should be considered in achieving a PoC in a timely and cost effective manner.

*Separate Registration Required