FDA, CMS facilitate continuity between approval of, payment for medical products

Fragmentation in the process of approving and clearing drugs for marketing by the FDA and then clearing drugs for coverage by CMS has led to questions regarding whether FDA approval necessarily results in approval for coverage and payment under Medicare and Medicaid. In a Journal of the American Medical Association (JAMA) perspective written by CMS Acting Administrator Andy Slavitt, FDA Commissioner Robert Califf, and FDA Deputy Commissioner for Medical Products and Tobacco Rachel Sherman, the authors posit that, despite such challenges, changes in the organization of health care and the larger technology landscape should allow the FDA and CMS to move toward the use of shared sources of evidence while still applying the most appropriate criteria to decision making.

In product approval and clearance for marketing by the FDA and in coverage and payment determinations by CMS, the agencies use scientific evidence to make determinations. The use of shared sources of evidence would help to reduce gaps in information that could lead to uncertainty in the approval or clearance of new therapies, as well as their subsequent use in medical treatment. Shared information would also increase efficiency in medical product development and ensure the use of high-quality evidence.

It is standard practice to use in the approval and clearance for marketing process research examining the effects of therapeutics in narrow, strictly delineated populations that may not reflect the clinical practice settings in which the products will be used. The authors stressed that when a product demonstrates promise in this narrow setting, developers should pivot quickly toward evaluating the product, using “evidence about the risks and benefits of tangible health outcomes in clinical settings and among patients representative of those who will actually use these products.” The expansion of the scope of research used in the approval and clearance process will help show how a product is likely to perform and how to utilize the product in treatment.  Garnering early involvement of health systems and payers will help the agencies to determine what kinds of evidence are needed to incorporate the product in practice, where the product fits in formularies and device inventories, and whether or how much to pay for its use.

The FDA and CMS are focusing on the following to ensure that adequate evidence is available to guide patients, clinicians, and payers in their choices:

  • clarifying the need for including diverse populations and measuring relevant clinical outcomes within the trials conducted for regulatory approval and to inform labeling;
  • collaborating with other federal agencies to build functional links across a range of systems to make the best use of existing digital information captured in the course of health care delivery, such as electronic health records, insurance claims, and data within clinical registries; and
  • ensuring broad collaboration across public and private sectors.

Seems like smooth sailing for Califf’s FDA nomination

Dr. Robert Califf, President Obama’s nominee to be the Commissioner of the Food and Drug Administration (FDA), coasted through questions at a confirmation hearing Tuesday in front of the Senate Health, Education, Labor and Pensions (HELP) Committee. Although many thought the process may hit a few bumps due to his ties with the pharmaceutical industry, Republicans and Democrats alike seem to support his candidacy and the nomination seems expected.

Califf’s background

A cardiologist and clinical trial expert from Duke University, Dr. Califf has been a consultant to drug companies and ran a research institute that received a majority of its funding from the industry. Dr. Califf said in his prepared statement that we need “an unbiased FDA that can work with industry to advance critical technologies while still making independent determinations to ensure that scientific potential is translated into safe and effective products. To advance, we must find common ground with industry and academia on the science without compromising this fundamental role of the FDA.”

Califf’s ties to the industry raised concerns among some public health groups and some Democrats say that he is too close to the industry he is being called on to regulate. Many medical experts dispute that, saying that industry is a principal funder of research in the United States and that working with companies does not present an inherent conflict.

Although essentially friendly, the hearing was punctuated with a few skeptical questions from Senators Elizabeth Warren (D-Mass) and Bernie Sanders (I-VT), who is running for the Democratic presidential nomination. In response to questions from Warren, Califf told the committee that the clinical trials he conducted at Duke, if funded by a drug company, had “ironclad” contracts giving the investigators the final rights to publication. Sanders said aid that the FDA needs a candidate who can stand up to an industry that has been “ripping off” the American people by charging “outrageous” prices for medicines.

Vote

The HELP committee will vote on whether to approve Califf’s nomination, which must then be approved by the full Senate. Patient groups and medical associations are hoping that Califf will help to speed new drugs to market and have publicly supported Califf.