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Long-Term Challenges for New FDA Leadership

When Dr. Hamburg arrives, she will face a number of competing priorities, along with a large backlog of policy and enforcement decisions that have waited for permanent leadership. There is a risk that important long-term challenges will go unaddressed…merely because they are not part of an immediate crisis or subject to pressure by the Secretary, the President, or Congress. Two or three years from now, these are the items that everyone will look back upon and say: we should have gotten started sooner.

Here is my list of seven such challenges:

Integrating new science into traditional clinical trials. Constructing real-world clinical trials has never been more difficult. In many clinical areas, we are moving from targeting disease symptomology toward impacting fundamental biological processes. Preventive treatments raise different issues than curative treatments. Two or three generations of products (in trials now and over the next few years) are “at-risk” unless the Critical Path is accelerated and expanded and other “clinical trial improvement” initiatives are developed.

Balancing safety with patient risk and need. Severely-ill patients deserve more balanced and more patient-centric policies with regard to dangerous treatments that have proven to provide great benefit to some patients (NB: “proven” does not mean phase 1 or early phase 2 trials). The Risk Evaluation and Mitigation Strategy (REMS) program helps, but these issues need a broader, more systemic examination.

Sifting valuable information from background noise. FDA is building a Sentinel system for early monitoring of problems, has access to the Medicare database and will be collecting incredibly large amounts of information about safety, food and drug imports, global product supply lines, clinical trials, patient registries, etc. It is a difficult, highly iterative process to distinguish meaningful patterns from background noise…in order to create actionable intelligence.

Managing globalization, rather than just responding to it. This is closely related, but distinct, from food import safety, heparin contamination and related crises. There needs to be a comprehensive, multi-year plan for managing globalization, including a budget for Congress to consider and fund. Tomorrow’s problems need to be identified and addressed before they become even bigger issues. Offices in China and India are necessary, but by no means sufficient.

Resisting the bias toward negative decisions. Up to a point, caution is the appropriate response to uncertainty. Yet uncertainty is inherent in all positive decisions. Taken to the extreme, the whole world of food and drugs could slow down, and then stop. The correct balance is not achieved by a memo or a speech, but by day-to-day actions and enhanced communications. Systemic review of all decisionmaking processes is the ultimate corrective.

Staying focused on priorities. FDA’s responsibilities greatly exceed its resources. Some mission creep is inevitable; some new responsibilities may be needed to benefit society and the public health. But sometimes FDA will need to say “no.” Such moments are difficult. “No” will never be accepted by policymakers or the public unless FDA is clearer in defining, justifying and explaining its priorities. Again, this must be addressed comprehensively.

Keeping the Best and the Brightest. FDA cannot succeed without a high-quality and committed workforce. Public service is an important attraction of working at FDA. It cannot be allowed to go out of style. Increased appropriations provide the opportunity to rationalize workloads, reduce burn-out, and build morale.

Do you have items you would add to this list? If so, post comments with your suggestions.

Please focus on issues and projects that have long-term consequences and take time to develop…but for which there is no immediate crisis or pressure.

We will take the suggestions, compile a list and send it to Drs. Hamburg and Sharfstein.


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One Response to “Long-Term Challenges for New FDA Leadership”

  1. thindin says:

    There is still a need for more clinical trials that take into account the number of medicines being used for children today that never even went through trials. I cannot understand the open neglect on pharma’s part in this area even though they are given an additional amount of time on their drug monopoly. The FDA needs to keep in mind that pediatricians still do not have proper references for dosages on drugs that have never been tested with children in mind, instead, they have to rely on packaging materials. The need for this kind of information only seems to surface when a child has seriously suffered or died from an improper dosage or misuse of a combination of drugs that aren’t necessarily prescription drugs.

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