In the recently published physician fee schedule (PFS) proposed rule for calendar year (CY) 2014 (Proposed Rule), the Centers for Medicare & Medicaid Services (CMS) proposes to revamp its process for covering items and services in investigational device exemption (IDE) clinical studies. 78 Fed. Reg. 43282, 43342-47, 43523-25 (July 19, 2013). Specifically, CMS proposes to adopt scientific and ethical standards that every IDE study would have to meet in order for costs to be covered. In addition, CMS proposes to centralize the coverage review process. Id. These proposed changes, if adopted, will affect manufacturers of IDE devices sponsoring clinical trials.

Proposed study standards

The Food and Drug Administration (FDA) categorizes IDE devices as either Category A or Category B devices. Category A devices are not covered by Medicare, but Medicare can cover the costs of routine items and services in Category A IDE studies. For Category B devices, Medicare may cover both the IDE device and the costs of routine items and services used in the clinical study of the device. Currently, for both Category A and Category B IDE devices, Medicare contractors have discretion to determine whether to cover IDE studies based on information submitted by study investigators within a contractor’s jurisdiction. As a result, coverage decisions can be inconsistent across contractors.

CMS proposes to change the existing guidance to require all Category A and Category B devices to meet specific criteria for Medicare coverage. CMS indicates that these “minimum standards” are necessary to ensure that “Medicare beneficiaries who volunteer to participate in studies are protected and that the study design is appropriate to answer questions of importance to Medicare and its beneficiaries.” Id. at 43343. Interestingly, the criteria that CMS proposes to apply to IDE studies are substantially the same as the criteria it proposes to use to approve studies under coverage with evidence development (CED) national coverage determinations (NCDs) as set forth in the 2012 proposed CED guidance and that it has recently used in some NCDs with CED. Under a CED NCD, the item or service that is subject of the NCD may be covered only when furnished pursuant to a study that meets those criteria.

The proposed criteria for coverage of an IDE study are the following:

  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 CFR 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 here 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 CFR 312.81(a) and the patient has no other viable treatment options.
  10. The study is registered on the website and/or the Registry of Patient Registries (RoPR) 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 effect 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

Id. at 43344-45, 43524-5. As noted above, CMS proposes that all IDE studies will have to meet these criteria for coverage. Meeting such criteria, however, may not be sufficient for coverage. CMS also proposes to automatically approve an IDE study where the study meets all of the above criteria and the following two additional criteria:

  • the study is a pivotal study
  • the study has a superiority study design

Id. at 43345, 43525. CMS says that the “pivotal study and superiority study design criteria furnish assurances that the study results will be informative for beneficiary choices and medical decision-making in the non-trial settings where most care is actually furnished.” Id. at 43345. It proposes to define pivotal study as a clinical investigation “designed to collect definitive evidence of the safety and effectiveness of a device for a specified intended use, typically in a statistically justified number of subjects,” which “may or may not be preceded by an early and/or a traditional feasibility study or trial.” Id. at 43346, 43524. CMS also proposes to define a superiority study as a study or trial “intended to demonstrate at some pre-specified level of confidence that the effect of an investigational treatment is superior to that of an active control by more than a pre-specified margin.” Id.

Studies that do not meet the pivotal study/superiority study design criteria may be covered but only if attainment of the first thirteen criteria is “sufficient to mitigate the failure” to have pivotal study with a superiority study design. Id. at 43524. Thus even if the first thirteen study criteria are met, CMS may determine that the study is not covered. This creates considerable uncertainty because CMS has given no indication of what factors may influence that determination or what level of “attainment” will be “sufficient to mitigate” the fact that that study is not a pivotal study and/or does not have a superiority study design.

Proposed review process

In addition to establishing standards that all IDE studies must meet, CMS also proposes to centralize the review process such that a single entity would be making the IDE coverage decision. Currently, Medicare contractors are responsible for making IDE coverage decisions. That means that approval must be sought in each jurisdiction in which study participants will be enrolled. Each contractor has a different process to review IDE devices and studies, although these processes are in most cases duplicative. According to CMS, the lack of centralization led to inconsistent IDE coverage across the Medicare contractors. Id. at 43343.

CMS proposes to have all IDE coverage requests submitted to the Director of the Coverage and Analysis Group in the CMS Center for Clinical Standards & Quality. Id. at 43345. This is the same Group in CMS that has jurisdiction over NCDs. CMS proposes that interested parties who seek coverage in an IDE study will be required to send a request letter describing the scope and nature of the IDE study and “discussing each of the 15 standards” in the policy (the 13 study standards plus the pivotal study and superiority study design standards). Id. CMS also proposes to require requests to include the FDA approval letter, IDE study protocol, Institutional Review Board (IRB) approval letter(s) and the identifier. Id. CMS proposes to notify stakeholders of all IDE devices eligible for coverage by posting the IDE study title and registry number on its website and publishing a list in the Federal Register. Id. CMS does not propose any specific timeframe for responding to requests. It indicates that changing the review and decision of IDE coverage to a centralized review process will not affect beneficiary appeal rights. Id. at 43343.

CMS states that it does not believe that a centralized review process would significantly reduce the number of IDE devices currently covered, but specifically requested public comment on that issue. Id. While it may be true that having a centralized process will not significantly reduce the number of covered IDE devices, the rigorous criteria that CMS proposes to require all IDE clinical studies to meet potentially could. At the same time, however, having a single set of criteria that apply to all studies and a centralized review process should address the current problem of inconsistent coverage across contractors.

Opportunity for comment

Comments are due by September 6, 2013. We would be happy to help you consider whether there are any issues on which you would like to comment and to assist you in preparing those comments.