Making Sense of FDA’s Expedited Drug Approval Pathways and Designations – for the Non-Regulatory Professional

blog header linkedin - FDA Approval v6 blog sizeOne of the fundamental responsibilities of the US Food and Drug Administration (FDA) is to approve effective medicines for people who need them, while upholding high standards for safety. That mission also demands that the FDA work efficiently and not delay approval of life-saving medical advances. Today, the FDA is reviewing applications for approval of new medicines faster than ever, and that’s a welcome change from the status quo 25 years ago. In the era from 1962 (immediately following the thalidomide recall) to the early 1990s, FDA review times for a New Drug Application (NDA) or Biologic License Agreement (BLA) were often measured in years rather than months. In 1993, the standard review time (from NDA or BLA submission to decision) for a new molecular or biologic entity (parlance for a drug not previously approved for any other use) was about 28 months,1 and in some cases, approvals were delayed for many years.

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Implications of ICMJE’s Data Sharing Proposal

ProEd-ICMJE-Data-Sharing-Proposal-PostOn January 20, members of the International Committee of Medical Journal Editors (ICMJE) announced a proposal that would require the authors of clinical trial publications to share the deidentified individual patient data that support their published results within 6 months of publication. Announced in an editorial published simultaneously in multiple medical journals, this proposal is based on the belief that authors have an “ethical obligation to responsibly share data generated by interventional clinical trials.” It also reflects the broader agenda of the ICMJE to foster greater transparency and reduce the potential for bias. This new requirement will likely go into effect in 2016 and will affect any clinical trial that enrolls patients beginning 1 year after ICMJE adopts the requirement.

This proposal makes a lot of sense in the interest of transparency, but what does it mean for clinical investigators involved in research and the companies that sponsor that research? To quote the ICMJE authors, “enabling responsible data sharing is a major endeavor that will affect the fabric of how clinical trials are planned and conducted and how their data are used.” Continue reading

President Obama’s Cancer Moonshot 2020 Initiative: How Big Is the Challenge?

Apollo rocket blasting offWhen President Obama’s White House Task Force meets for the first time this week, it faces a lofty charge: “Let’s make America the country that cures cancer once and for all.” His proposed Cancer Moonshot 2020 initiative will provide much needed funding for research, will encourage collaboration between industry and academia, and will undoubtedly lead to important advances in our understanding of cancer and how to treat it.

Although this initiative is noble and well intentioned, calling for a “cure for cancer” oversimplifies the challenge and is based on an outdated understanding of the disease.

The public needs to understand that cancer is not one disease; it’s hundreds of different diseases, and it’s more complex than anyone imagined back in 1971 when President Nixon first declared war on cancer. Those involved in cancer research know that conquering cancer, in all its varied forms, is truly a global endeavor that will require a sustained commitment for decades to come. While a challenge to be the “country that cures cancer” stirs our patriotic spirit and conviction that American biomedical know-how can render cancer irrelevant in our lifetime, the reality is that meaningful progress will require international collaboration.

Are we, as a nation, prepared to take on that challenge and lead such a charge? The President has taken the first step, appointing Vice President Joe Biden to lead the White House Task Force. But now the difficult work begins. Can we balance competing priorities and sustain both our political will and financial commitment for the many years that such an effort will undoubtedly take? Continue reading