A new approach to new drug approvals?

Few topics inspire more heated discussion among drug developers and pharmaceutical industry watchers than the regulation of new products.  For those unfamiliar with the debate, the two sides of the argument can be summarized as follows.

Industry veterans feel that excessively conservative regulators squelch innovation in a desire to cover their own behinds, while industry critics contend that regulators aren’t strict enough, and that pharmaceutical companies should be held to an even higher standard and warrant even greater supervision.

In the meantime, patients wonder why modern science hasn’t produced the medicines they so desperately need.

Now it seems there may be an answer that could satisfy everyone.  Susan Desmond-Hellmann, Chancellor of the University of California, San Francisco (UCSF) and former Genentech executive, suggests turning drug approvals from a discrete yes or no variable into a continuous moving target.

The fundamental problem with the current system, Desmond-Hellmann observes, is that regulators have only two options, when in fact, it might make a lot more sense for them to have a range of choices. She proposes that agencies, such as the FDA, could approve a drug for limited use, or use with limited promotion, following the submission of initial acceptable evidence of safety and efficacy. Permission for broader use, and less restrictive promotion, could be given after additional data are obtained.

This solution appears to make sense on many levels. It acknowledges:

  • the long time it takes to become familiar with a drug and learn some of it’s more subtle effects – good, as well as bad
  • the importance of accelerating the time it takes to get potentially useful drugs into the hands of patients who might need them
  • the technologies now available to track and assess the performance of new drugs

FDA Approval Process

One advantage of such a system would be the emphasis it would place on durable results, as full approval would likely require not just success in a clinical trial, but continued demonstration of success under “real world” conditions.  Such a requirement would almost certainly stimulate the development of integrated “health solutions,” a bundled assortment of patient- and physician-support programs accompanying the drug.

Although a graduated regulatory approval process would not be perfect; it is almost certainly a significant step in the right direction.

Patient and physician educational and adherence programs make sense, but many companies have been reluctant to invest in them.  In the context of an offering that would need to succeed in order for a drug to be fully approved, the incentives are considerably more apparent, and would almost certainly stimulate interest in companies striving to deliver the most effective patient support programs.

However, the big questions still remain.  Are regulators ready for such change? Is the Industry ready to take on the responsibility and cost of ensuring appropriate use of their products and long-term pharmacovigilance? Would patients and consumer watchdog organizations accept a tiered approach?

What do you think?  SRxA’s Word on Health would love to know.

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