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FDA, CMS Seek Significant Amendments to Data Collection


The FDA commissioner and the acting administrator of CMS have published an opinion piece in JAMA that recommends changes with trial design, data collection, and the need for collaboration across public and private sectors.

The FDA commissioner and the acting administrator of CMS have published an opinion piece in JAMA that recommends changes to trial design and data collection, and discusses the need for collaboration across public and private sectors—all in an effort to fortify the evidence on which regulatory and coverage decisions are made.

The piecemeal manner in which data is collected and maintained in disconnected databases has contributed to questions on whether FDA marketing clearance also means the product will be reimbursed. Shared resources are key, the authors wrote, although approval criteria would remain distinct. While the FDA approval process seeks substantial evidence from at least 1 “well-conducted” clinical trial, CMS establishes the standard for coverage as “reasonable and necessary.”

Evaluating a product being developed in the intended population who will use it post launch is vital, the authors pointed out, and they recommended including this population in early phases of a clinical trial. They also recommended involvement of health systems and health plans earlier in the process “to understand and address the kinds of evidence needed to incorporate the new product into practice, place it on formulary or device inventory, and decide whether and how much to pay for its use, thereby facilitating implementation and adoption.”

With this end goal, the FDA and CMS have developed the following recommendations for researchers and institutions to improve on the existing practice of clinical evidence generation:

  1. Selecting the right patient population. FDA and CMS would like to see more diversity among clinical trial enrollees, especially for products that gain biomarker-based accelerated approval.
  2. Linking databases. Capitalizing on existing digital information is key, and the authors recommend connecting information collected during healthcare delivery that is trapped in electronic health records, insurance claims, and clinical registries.
  3. Establishing public-private partnerships. Approaches such as the Patient-Centered Outcomes Research Institute are ideal, the article claimed, with broad stakeholder participation.

These changes will allow both the FDA and CMS to measure more patient-centered health outcomes, the authors wrote, and also pave the path for a more personalized approach to medicine.


Califf RM, Sherman RE, Slavitt A. Knowing when and how to use medical products a shared responsibility for the FDA and CMS [published online November 7, 2016]. JAMA. doi:10.1001/jama.2016.16734.

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