Having just returned from meetings with regulatory authorities in Kenya, Jordan, and Saudi Arabia, I am energized that higher levels of pharmaceutical quality and pharmacovigilance are possible.
But it won’t be easy.
Enhanced levels of excellence will require, if not global harmonization, more aggressive partnerships between agencies around the world.
In other words, it’s time to plan and execute a regulatory Marshall Plan to help build, nation-by-nation, global systems for both quality and safety. Working together to raise the regulatory performance of all nations will help all nations (even the 20% deemed “capable” by the WHO) to create sound foundations to address a multitude of quality and safety dilemmas such the manufacturing of biosimilars, the control of API and excipient quality, pharmacovigilance and, yes, even counterfeiting.
But drug regulation has to go beyond safety and quality and pharmacovigilance. It’s got to embrace innovation. What we need here at home and around the world is a hunger for entrepreneurial regulation.
Entrepreneurial Regulation is a philosophy that allows agencies such as the FDA to be both regulator of and colleague to industry. Expedited pathways are Entrepreneurial Regulation. Special Medical Use is Entrepreneurial Regulation. REMS are Entrepreneurial Regulation. The exercise of enforcement discretion is Entrepreneurial Regulation. More aggressive use of the Reagan/Udall Foundation is Entrepreneurial Regulation. A more central role for the patient voice is Entrepreneurial Regulation.
Entrepreneurial Regulation makes bodies such as the FDA enablers rather than roadblocks to innovation.
One of the key conundrums of Entrepreneurial Regulation is that there is an inherent tension between predictability and innovation.
The foundational principle of PDUFA is predictability – not speed. And that’s been the focus of the conversation: ambiguity vs. predictability. But, when it comes to innovation, ambiguity is inherent. The pathways to innovation are always ambiguous. Innovation is risky – and not only to investors.
And Entrepreneurial Regulation is likewise risky. But as with all matters regulatory, risk must be viewed alongside benefit – to the public health.
Another level of tension is the relationship between research (R) and development (D). Specifically, the lack of respect between the two. Beyond the disproportionate levels of government funding (when’s the last time you heard anyone talk about “doubling” the FDA budget), nascent relationships between academia (“R”) and industry (“D”) are struggling.
The issue of out-sourcing basic research isn’t new – but it’s mighty contentious. And it’s the new reality of drug development. But, if we are to learn any lesson from the CRO experience, it’s that while we say “partnership,” the danger is that it devolves into a vendor-like relationship. It’s the Golden Rule. He who has the gold makes the rules. Will that be acceptable to high-level, big ego Ivy Hall-ers?
And then there’s the issue of academic priorities, specifically tenure. Does industry funding carry the same weight as NIH grants when it comes to advancing a university career? Not at present. That will have to change.
Need drives change. Just as CROs are finally really partnering with pharma to drive the development of personalized medicine, so too must academe and industry collaborate on the continued evolution of pharmaceutical innovation. It will take discipline and focus. It will be risky. And it will take will. There is a confluence of interest.
When it comes to global safety, quality, and pharmacovigilance standards, there’s a general consensus that a high tide floats all boats. When it comes to Entrepreneurial Regulation demands that we honestly address a tough but fundamental question, how can regulatory agencies around the world (FDA-led by both word and deed) focus on what they can do to facilitate collaboration that results in innovation?
Step One is focus.
In the words of entrepreneur extraordinaire Steve Jobs, “I'm convinced that about half of what seperates the successful entrepreneurs from the non-successful ones is pure perserverance."
And, in the case of Entrepreneurial Regulation, failure is not an option.