Mindset Change Needed for Re-Engineering Pharmaceutical/Biotech R & D

In the previous post and in the book Pharmaplasia I suggest a need for a new approach to pharmaceutical R & D.  There are two essential elements to this proposal.

The first has to do with scientific and corporate integrity.  If the research scientists and corporate executives are not honest about what you have and what you have seen in drug development (if their priority is to protect the “potential“ of your compounds), you can’t make good decisions about your pipeline products.  Research scientists or corporate executives trying to preserve compound “potential” by strategically navigating around potentially damaging data, half-truth disclosures, or blatantly ignoring negative results and their implications can easily sabotage any corporate effort to produce a more robust pipeline with a higher probability of success.  The second essential element will be meaningless if this first element is ignored.

The second essential element:

Know more about your compounds before they go into Phase III clinical trials.

A more comprehensive basic science understanding of your compounds before they enter the clinic, especially expensive Phase III trials; can increase the probability of success.

For safety, this requires an aggressive exploratory preclinical program that “looks for toxicity” (going well beyond the regulatory requirements) and understanding what you find and what you see … not just being able to explain it away.

From an efficacy and safety perspective, if your compound affects one biologic system, what other systems does it affect? Have you really looked or have you been focused on “getting an indication?”   Do you have the basic science data to support the premise/hypothesis for efficacy (or comparative superiority)?  Have you scientifically challenged the premise with alternative outcome possibilities with data to support the different probabilities?   Or, is it still just a hypothesis you plan to prove in Phase III?  Are you sure you have the right endpoints for your Phase III trials?  Have they been sufficiently validated (high probability statistics) in Phase II studies?  By this I mean, have you done more than just a couple of regulatory required trials?  Have you looked at design alternatives that could affect outcomes for different endpoints options?

This strategy may take longer.

But, rather than how fast can you get how many compounds into patients and to the market, research teams and corporate executives need to shift their mindset to increasing the probability of success.  You can do this with a more comprehensive approach to preclinical research.  Thoroughly understanding how your products are going to perform in Phase III trials (or comparative trials) before the trials commence.  Eliminate the “surprises” with better, more comprehensive science around the products.  Be exhaustive in exploration, honest about the findings, inclusive of interpretations, and have better data to support the “go forward” premise or hypothesis.  This all may seem like a simplification but if you’re interested in a more elaborate discussion, I invite you to read about the need for pharmaceutical R & D change and recommendations for change in Pharmaplasia.

Sure there will still be failures, but they should be fewer.  But perhaps the biggest benefit from this approach is the potential for new discoveries of safer, more effective products.  Better diagnostics, more definitive efficacy benefits (maybe we are looking at the wrong endpoints), and advances in the understanding of human biology await this new Pharmaceutical R & D model.  Not to mention, less contentious regulatory reviews, renewed hope for patients with difficult to treat diseases, and more certainty for the investment community.  mike@pharmareform.com

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