Via Electronic Submission
January 28, 2013
Marilyn Tavenner, Acting Administrator
Centers for Medicare and Medicaid Services
Department of Health and Human Services Baltimore, MD 21244-8016
Re: Coverage with Evidence Development in the context of coverage decisions
Dear Administrator Tavenner:
Bayer Healthcare LLC (“Bayer”) appreciates the opportunity provided by the Centers for Medicare and Medicare Services (“CMS” or “Agency”) to submit comments on the draft guidance regarding Coverage with Evidence Development (“CED”) in the context of coverage decisions (“draft guidance”).
With more than 6,000 healthcare employees across the United States, Bayer aims to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases. We focus our efforts where we can have the most beneficial impact on the lives of those who depend on our nearly 150 years of experience researching and developing new pharmaceuticals and medical devices.
On the basis of this long experience, Bayer wishes to comment on several proposals contained within the draft guidance, which are summarized below:
- The Draft Guidance Changes Long-Standing CMS Policy in a Substantive Manner and, Therefore, Can Only Be Undertaken Through a Legislative Rule: Bayer requests that the Agency not make substantive changes to existing policy, as it attempts to do here, except in a correctly noticed legislative rule.
- Provide Greater Detail Regarding the CED Process: Bayer supports CED as a driver of innovation. However, the current draft guidance is ambiguous and does not provide sufficient detail for active stakeholder participation. Bayer urges CMS to supply greater detail regarding when and how CED will be utilized.
- Re-Incorporate the Eight CED Governing Principles Found in 2006 Guidance: The draft guidance does not contain the eight guiding principles found in the 2006 guidance. These principles provide essential beneficiary protections and should be re-incorporated into the draft guidance. Bayer also requests that the Agency clarify that CED may only occur through the NCD process.
- Exclude from CED Evaluations FDA-Approved Indications, Orphan Drugs, and Drugs in Part D Protected Classes: FDA-approved indications, orphan drugs, and drugs in Part D protected classes have already undergone extensive review. Applying CED to these drugs and uses has the potential to unnecessarily endanger beneficiary health by limiting access to critically important treatments. Therefore, applying CED in these circumstances would only incur unnecessary expense and limit necessary and established treatment options. For these reasons, the Agency should prohibit the application of CED to these drugs and uses.
- Base CED Coverage Decisions on the Statutory Criteria: The Social Security Act requires coverage for items that are reasonably necessary for the treatment or diagnosis of a disease. The Agency’s use of an “outcomes” improvement standard in the draft guidance is not the appropriate standard. Specifically, the draft guidance should clarify that outcomes improvement is not the standard to evaluate diagnostic agents.
- Provide Clear Criteria Describing When CED Will Be Utilized: While the draft guidance does list four quite general factors when CED may be applied, Bayer requests that the Agency provide stakeholders with additional detail regarding when CED will be required.
- Provide Access to CED Services and Items Outside of Clinical Trials: Bayer urges the Agency to provide alternatives to clinical trial participation to allow greater beneficiary access to CED services and items.
- Clarify CED Clinical Trial Requirements: Bayer requests that the Agency clarify how CED trials will be approved, what end-points are required for CED trials, and when and how the CED process will conclude.
- Clarify the Role of the Agency for Healthcare Research and Quality (AHRQ): The draft guidance contemplates the involvement of AHRQ in the CED process. Bayer requests that the Agency provide additional information regarding the role of AHRQ in this process.
- Continue to Allow Medicare Administrative Contractors (“MACs”) to Determine Coverage for Routine Clinical Trial Costs: Bayer strongly supports the Agency’s plan to continue to allow MACs to determine which routine clinical trial costs to cover.
- The Draft Guidance Changes Long-Standing CMS Policy in a Substantive Manner and, Therefore, Can Only Be Undertaken Through a Legislative Rule
The draft guidance makes substantial changes to long-standing CMS policy on CED. As an APA matter, we believe that imposing these new requirements necessitates a more transparent, complete, and formal regulatory process than draft guidance posted to the agency's website without official notice in the Federal Register. Agency guidelines that create new rights and duties are subject to the APA’s requirement of notice-and-comment rulemaking. Further, the Agency must provide sufficient information in its notice to permit interested parties to meaningfully comment and participate in the rulemaking.1
The draft guidance does not provide sufficient detail to allow meaningful comment regarding when CED will be utilized, the standard by which CED evidence will be judged, or how such evidence must be gathered. Bayer believes all stakeholders involved in the CED process would benefit from a more formal review of the CED process and urges the Agency to issue CED regulations using notice and comment rule-making procedures.
- Provide Greater Detail Regarding the CED Process
Bayer supports CED as a driver of innovation and as a means of ensuring that Medicare beneficiaries have access to treatments and diagnostic agents that they otherwise may not receive. However, Bayer is concerned about the Agency’s stated intent to use CED more frequently for new products and to re-evaluate existing technologies, which are already in use and on which beneficiaries are dependent.
Bayer believes that CMS should continue its policy of generally allowing local Medicare contractors to make initial coverage decisions. CED should only be considered when new or existing technologies show promising potential, but have not garnered enough evidence to support local coverage. Use of CED in such limited circumstances will truly drive innovation and improve Medicare beneficiaries’ access to treatment and diagnostic agents.
If CMS does intend to use CED more frequently, it must provide clear, detailed guidance that will allow stakeholders to participate in the CED process. CED will only be effective if stakeholders understand when and why CED will be implemented. The current draft guidance does not provide answers to these critical questions. Indeed, without this certainty, investment in innovation will inevitably be stifled and the stated purpose of the proposed rule will be undermined.
Further, like other stakeholders, including patient and provider advocacy groups, Bayer is concerned that the current draft guidance may provide CMS with a means to eliminate coverage for medically necessary services and items based on cost considerations. Bayer requests that, prior to finalizing the draft guidance, CMS clarify that CED will only be used to encourage innovation and improve beneficiary health outcomes by developing additional support for the use of treatments and diagnostics in the Medicare population.
In addition, Bayer urges CMS to clarify that CED will not be triggered or used as a method to make coverage decisions based on comparative effectiveness claims. Section 1182 of the Social Security Act explicitly states that the Secretary may not deny coverage or services solely on the basis of comparative effectiveness research.2 Bayer urges the Agency to clarify that CED will not be used for this purpose. We are concerned that when CMS states that it is considering “cost” it is, as a practical matter, undertaking comparative effectiveness analyses in a fashion inconsistent with the statute.
- Re-Incorporate the Eight CED Governing Principles Found in 2006 Guidance
The draft guidance does not include the eight CED governing principles found in the 2006 guidance. These principles state that: 1) NCDs requiring CED will occur within an NCD process, which is transparent and open to public comment; 2) CED will not be used when other forms of coverage are justified by the available evidence; 3) CED will in general expand access to technologies and treatments for Medicare beneficiaries; 4) CMS will use CED infrequently; 5) CED will lead to the production of evidence complementary to existing medical evidence; 6) CED will not duplicate or replace the FDA’s authority in assuring the safety, efficacy, and security of drugs, biological products, and devices; 7) CED will not assume the NIH’s role in fostering, managing, or prioritizing clinical trials; and 8) any application of CED will be consistent with federal laws, regulations, and patient protections.
Like other stakeholders, including most notably patient and provider groups, Bayer is extremely concerned about the abandonment of these critical safeguards. With these principles absent, the lack of clear guidance regarding the use of CED in the current draft guidance is all the more troubling. Bayer urges CMS to explicitly re-incorporate each of these missing principles that are critical to protecting beneficiary access to essential treatments.
Specifically, Bayer urges CMS to clarify that CED can only occur within the NCD process. In addition to disregarding the principle that CED must occur within the NCD process, the draft guidance deletes sections III and IV from the 2006 guidance that linked CED to the NCD process. However, in several instances, the draft guidance references CED as part of an NCD. For example, on pg. 7, the draft guidance states that “to the extent that the NCD does not identify specific clinical trials…”. Therefore, the draft guidance appears to use the NCD process interchangeably with CED.
The NCD process is an established, transparent process that ensures stakeholder participation. The CED process will only be successful with active participation from providers, patients, researchers, advocacy groups, manufacturers, and all other interested stakeholders, and the NCD process is the most effective approach to enable robust and critical participation from these stakeholders.
- Exclude from CED Evaluations FDA-Approved Indications, Orphan Drugs, and Drugs in Part D Protected Classes
The draft guidance does not specify which treatments, agents, or specific uses will be eligible for CED. Bayer requests that the draft guidance specifically exclude the following categories from the CED process: 1) CED should not be used to limit coverage for FDA-approved indications of drugs and devices; 2) CED should not be used to limit coverage for “medically accepted indications” for treatments that have been appropriately established through the compendia process, given the statutory basis for compendia supported coverage;3 and 3) CED should not be applied to orphan drugs and drugs in Part D protected classes.
Specifically, CMS should look to both the United States Pharmacopeia and the American Hospital Formulary Service for evidence of medically accepted indications. Further, CMS should also look to the peer-reviewed medical literature to determine if sufficient evidence exists for coverage.4 If these sources support the use of the treatment or diagnostic agent, CED should not be utilized.
In addition, Medicare beneficiaries suffering from orphan diseases or requiring drugs found in protected classes have limited options for effective treatment. Therefore, it is critical that the Agency not utilize CED for these essential therapies. CED in these contexts would have a devastating impact on the most vulnerable Medicare populations.
Excluding these categories from potential CED decisions will ensure that the CED process does not negatively impact access to validated, essential treatments. A failure to exclude these categories would also be fundamentally inconsistent with CMS’s stated goal of encouraging innovation. If these basic areas of coverage support are eroded through the draft guidance, innovation and the investment necessary to sustain it will inevitably suffer.
- Base CED Coverage Decisions on the Statutory Criteria
The draft guidance repeatedly references “outcomes” improvement as the standard for Medicare coverage.5 Bayer is concerned about the use of this standard for CED decisions, which is not consistent with the statute. The focus on outcomes improvement is not the correct standard to use when determining what treatments and diagnostic agents will receive coverage.
The Social Security Act provides for coverage of enumerated types of services and items so long as those services and items are reasonably necessary for the diagnosis or treatment of a disease or to improve the functioning of a malformed member.6 There is no authority to limit coverage to the least costly item or the one that may, according to one study or another, have the most promise for affecting outcomes. Such an approach would fail to appreciate the unique circumstances of a particular Medicare patient, would not provide flexibility to account for differences in patient populations, would dictate a single approach to treatment and diagnosis, would make impossible the use of combination treatment approaches that in oncology and other areas are a driver for innovative and personalized treatment, and would be utterly inconsistent with the desire to encourage innovation in health care.
Medicare beneficiaries are statutorily entitled to access to important diagnostic agents. Although diagnostic agents often lead to beneficial outcomes, by allowing disease to be accurately managed, they are covered, as a matter of statute, even if there is no effect on the ultimate outcome of a disease process, where they produce a diagnosis. This is why the statute refers to coverage for “diagnosis or treatment.”
Similarly, many treatments, such as pain management, are not designed to improve outcomes, but to address symptoms. The management of the symptom is beneficial regardless of any outcome improvement.7 The Medicare statute nowhere refers to an “outcomes” test for coverage; it does not exist.
Accordingly, Bayer asks that the agency strike its “outcome” references in the draft guidance, as they are inconsistent with the statute.
- Provide Clear Criteria Describing When CED Will Be Utilized
Bayer is concerned that the draft guidance does not provide sufficiently clear criteria regarding when CED will be utilized. The draft guidance lists four general situations in which CED will be considered. These include instances: 1) when the relevance to the Medicare population is uncertain; 2) when available evidence is not representative of the Medicare population; 3) when evolution and reevaluation of evidence on older products occurs; and 4) when evidence was not developed in a setting that represents the typical Medicare community care setting. It will not increase innovation for the Medicare program to be vague as to the circumstances under which coverage will not be provided; by injecting uncertainty into the development process, it will diminish innovation.
Bayer requests additional information regarding these circumstances. The CED MEDCAC meeting held in May specifically identified the need for clear factors establishing when CED would be utilized for different product types. In the absence of greater specificity, the criteria listed in the draft guidance could apply to practically every new and existing technology available and leaves many important questions unanswered. For example, will the same factors and criteria be used for both drugs and devices? How will CMS determine when a reevaluation of past evidence is appropriate? The MEDCAC meeting clearly identified the need for clear answers to these questions in any draft guidance.
- Provide Access to CED Technologies Outside of Clinical Trials
Per the 2006 guidance, beneficiaries could receive coverage for CED technologies either through a Coverage with Appropriateness Determination (“CAD”) or through Coverage with Study Participation (“CSP”). The draft guidance no longer includes the CAD approach and limits coverage to beneficiaries participating in a clinical trial. Bayer is extremely concerned about this significant restriction in access.
Limiting coverage for CED to clinical trial participation is clearly inconsistent with the stated goal of encouraging innovation. Bayer, along with many patient and provider stakeholders, is concerned that the true intent of the change in policy may be to restrict coverage to innovative items and services. Only by providing adequate access options will CED be able to accomplish its goals of both collecting sufficient information for CMS coverage and driving innovation.
Therefore, Bayer urges CMS to develop flexible, alternative procedures to allow Medicare beneficiaries to access CED items or services while evidence is gathered during clinical trials. This is especially important for beneficiaries who are ineligible for available CED clinical trials. Medicare should not establish a “two tiered” system of access.
In this regard, we note that, at present, the draft guidance only contemplates potential observational studies as a method to provide coverage at the conclusion of all prospective clinical trials. Such observational trials should be designed in tandem with prospective studies to provide access for those beneficiaries ineligible for prospective clinical trials.
In addition, CMS should also develop alternative measures to allow beneficiaries to access CED technologies while evidence is being developed. Specifically, the draft guidance should require all CED decisions to establish coverage for beneficiaries if their care will be provided in a fashion consistent with trial requirements, even if they are not in a clinical trial. Beneficiaries should not be discriminated against based on whether they can gain access to a trial.
Without such alternative measures, beneficiaries will be denied access for significant periods of time given the length of most clinical trials and the subsequent time required for Agency review of clinical trial results. Alternative evidence collection (such as a registry) would ensure that beneficiaries have access to needed technologies, while also allowing CMS to gather additional evidence to support coverage.
- Clarify CED Clinical Trial Requirements
The draft guidance lists 13 factors that CED trials should meet. However, the draft guidance does not require the Agency to state what evidence a trial sponsor must provide to the Agency at the conclusion of the CED process. Bayer requests that the draft guidance clearly require any CED decision to include specific questions from the Agency to assist in the design of a meaningful CED clinical trial. Only by clearly describing what evidence is missing will trial sponsors be able to design clinical trials capable of meeting the Agency’s identified evidence gap.
The draft guidance also defines the following three situations in which CED coverage may conclude. Clearly defining the end of CED is an important issue, as stakeholders are concerned that CEDs will linger unnecessarily, limiting coverage in circumstances where such limitations cannot be justified. CED trials will conclude if any of the following three criteria are satisfied: 1) No CED trials are approved; 2) no CED trials are completed in the timeframe required in the CED opinion; or 3) CED trials are completed.
The first criteria implies that all clinical trials must be approved. However, the draft guidance does not clarify who must approve these trials, how long a sponsor has to seek approval, or what factors the trial must meet to gain such approval. Bayer requests that CMS clarify who will be responsible for approving such trials and how the process will work.
Bayer urges the Agency to also provide additional information regarding the conclusion of CED trials. All CED decisions should include clearly defined end-points for each CED trial. This clarification will allow sponsors to develop effective trials and also will provide clear timelines to ensure that the CED process does not continue indefinitely.
Further, the draft guidance allows for a potential period of non-coverage while CMS reviews the results of completed CED trials. Bayer is extremely concerned about this total restriction on CED technologies during the time period during which the Agency reviews the results of CED trials. Since coverage for CED technologies is limited to participation in a clinical trial, once all trials have been completed, CMS’ contractors should be permitted to provide coverage pending action by the Agency. At the very least, CMS should be required to act within 90 days of the conclusion of the data collection, the timeline that applies for various NCD actions.
- Clarify the Role of the Agency for Healthcare Research and Quality (AHRQ)
The draft guidance discusses the potential role for AHRQ in the development of CED. However, the draft guidance contains little detail regarding the role AHRQ will play in the development of CED trials. The draft guidance does generally describe three potential areas of AHRQ involvement: 1) assisting with the design of clinical trials; 2) form partnerships to fund CED clinical trials; and 3) provide confidentiality protections for CED data. Bayer requests that the Agency provide additional information regarding AHRQ’s role in the CED process. It is critical that all stakeholders understand the role of AHRQ to ensure the successful operation of the CED process.
- Continue to Allow MACs to Determine Coverage for Routine Clinical Trial Costs
Bayer supports, with great enthusiasm, CMS’ proposal to continue to allow MACs to determine what routine items and services are covered outside of the CED clinical trial.
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Bayer appreciates the opportunity to comment on the draft guidance and looks forward to working with CMS in the future to improve access to quality, affordable healthcare coverage.
1 5 U.S.C. § 553(c).
2 See Social Security Act § 1182
3 42 U.S.C. § 1396r–8
4 See Medicare Benefits Policy Manual. Ch. 15 – Covered Medical and Other Health Services.
5 CED Draft Guidance: Section V.
6 42 U.S.C. § 1395y(a)(1)(A)
7 In addition, even if the agency continues to refer to “outcomes” analysis as part of the a CED process, Bayer asks that CMS clarify what is meant by “outcomes” improvement, which the guidance completely fails to do, at present. How will this standard be evaluated? Conducting rigorous research on outcomes improvement, without using comparative effectiveness claims, can be extremely challenging. If CMS plans to consider outcomes improvement when making coverage decisions, Bayer strongly requests that the Agency provide greater detail regarding how this standard will be met.