CMS Issues Proposed Rule That Will Update Payment Policies/Rates Under the Medicare Physician Fee Schedule

This entry was written by guest blogger Isabelle Chivoret, a student at the Paris Bar School. Isabelle holds a Ph.D. in private law, in the field of pharmaceutical law.

The Centers for Medicare & Medicaid Services (CMS) issued a proposed rule that will update payment policies and rates under the Medicare Physician Fee Schedule (MPFS) for calendar year (CY) 2014 on July 6, 2013.

CMS proposes to approve an IDE study automatically when:

• The study meets the 13 following criteria.

1. The principal purpose of the study is to test whether the item or service meaningfully improves health outcomes of patients who are represented by the Medicare-enrolled subjects.
2. The rationale for the study is well supported by available scientific and medical information, or it is intended to clarify or establish the health outcomes of interventions already in common clinical use.
3. The study results are not anticipated to unjustifiably duplicate existing knowledge.
4. The study design is methodologically appropriate, and the anticipated number of enrolled subjects is appropriate to answer the research question(s) being asked in the study.
5. The study is sponsored by an organization or individual capable of completing it successfully.
6. The study is in compliance with all applicable federal regulations concerning the protection of human subjects found at 45 C.F.R. Part 46.
7. All aspects of the study are conducted according to appropriate standards of scientific integrity set by the International Committee of Medical Journal Editors.
8. The study has a written protocol that clearly demonstrates adherence to the standards listed as Medicare requirements.
9. Where appropriate, the clinical research study is not designed to exclusively test toxicity or disease pathophysiology in healthy individuals. Trials of all medical technologies measuring therapeutic outcomes as one of the objectives may be exempt from this standard only if the disease or condition being studied is life threatening as defined in 21 C.F.R. § 312.81(a), and the patient has no other viable treatment options.
10. The study is registered on the ClinicalTrials.gov website and/or the Registry of Patient Registries by the principal sponsor/investigator prior to the enrollment of the first study subject.
11. The study protocol specifies the method and timing of public release of results on all pre-specified outcomes, including release of negative outcomes. The release should be hastened if the study is terminated early. The results must be made public within 24 months of the end of data collection. If a report is planned to be published in a peer-reviewed journal, then that initial release may be an abstract that meets the requirements of the International Committee of Medical Journal Editors. However, a full report of the outcomes must be made public no later than three years after the end of data collection.
12. The study protocol explicitly discusses subpopulations affected by the item or service under investigation, particularly traditionally underrepresented groups in clinical studies, how the inclusion and exclusion criteria affect enrollment of these populations and a plan for the retention and reporting of said populations in the study. If the inclusion and exclusion criteria are expected to have a negative effect on the recruitment or retention of underrepresented populations, the protocol must discuss why these criteria are necessary.
13. The study protocol explicitly discusses how the results are or are not expected to be generalizable to subsections of the Medicare population to infer whether Medicare patients may benefit from the intervention. Separate discussions in the protocol may be necessary for populations eligible for Medicare due to age, disability or Medicaid eligibility.

• The study is a pivotal study.
• The study has a superiority study design. (CMS believes superiority trial designs provide important advantages that are not completely addressed by non-inferiority design; for instance, superiority trial designs allow beneficiaries to compare their options and determine which one is superior for them.)

However, the Medical Device Manufacturers Association (MDMA) is concerned about the proposed coverage criterion. MDMA thinks that the requirement that a device in an IDE study must have a superiority study design is critical and prefers the non-inferiority study design (i.e., the investigated device is not substantially worse than the comparator device ["at least as good as" criterion]).

According to MDMA, superiority study design would:

• Be inconsistent with the FDA’s policies on IDEs and create conflicts between the FDA’s data collection requirements and CMS’s coverage.
• Medical device trials generally are not structured as superiority trials, and non-inferiority trials typically are considered to be the most appropriate trial designs for IDE trials.
• Create an incredibly high barrier for smaller companies that are targeting specific and sometimes underserved patient populations, yet have similar clinical characteristics to existing technologies. These trials require larger patient populations and longer duration than non-inferiority trials. If Medicare’s coverage requirements become more stringent, manufacturers will be discouraged from conducting these trials and including Medicare beneficiaries in the studies. We believe that after nearly 20 years of encouraging manufacturers to include Medicare beneficiaries in their trials and expanding access to innovative devices, the proposed policy would be a misguided change.
• Discourage sponsors from including Medicare beneficiaries in trials for medical technologies, and thus cause access problems for Medicare beneficiaries. CMS states that for studies that do not meet the superiority design study/pivotal study criteria, coverage is possible if a sponsor attains the 13 newly established criteria in a manner that is “sufficient to mitigate the failure” to have a pivotal study with a superiority design, but does not clearly explain what “sufficient to mitigate the failure” means.
• Be particularly inappropriate for diagnostics as diagnostic information, by itself, does not show health outcomes improvements.

For this reason, among others, MDMA does not support CMS proposed revision to the coverage process for medical devices in IDE clinical trials, and recommend that CMS withdraw its proposal.