Director, Payor Policy
National Marrow Donor Program
The American Society for Blood and Marrow Transplantation (ABSMT), the Center for International Blood and Marrow Transplant Research (CIBMTR) and the National Marrow Donor Program (NMDP) appreciate the opportunity to comment on Coverage with Evidence Development (CED). The ASBMT is a national professional association promoting the advancement of blood and bone marrow transplantation. The CIBMTR is an academic research organization that maintains an observational database of more than 350,000 HCT recipients since 1972, and maintains the Stem Cell Therapeutic Outcomes Database for the US government. The NMDP is a non-profit organization that maintains a federally-mandated registry of more 9 million potential hematopoietic cell donors and umbilical cord blood units. Together, we represent the interests of hematopoietic cell transplant (HCT) recipients, donors, researchers, and clinicians.
We commend CMS for soliciting comments on CED. In September of 2009, our organizations submitted a request for a National Coverage Determination (NCD) regarding coverage of allogeneic HCT for Myelodysplastic Syndrome and received a CED decision. In November 2010 the CIBMTR was approved to open an outcome study as part of CED, which continues at the present time. Based on our experiences throughout this process, we would like to respectfully suggest the following issues be considered in future revisions of the program.
Implementation of CED:
1. Timeline: As noted in CMS’s comment solicitation letter, CED has the potential to accelerate Medicare beneficiaries’ access to innovative items and services. However, the actual timeline from NCD request to the initial decision memo to study approval and implementation is several years. This is a deterrent to submitting additional future requests and can significantly delay access to services.
Suggestions: 1) During initial pre-request meeting, indicate if a request will be likely to track to CED or if an NCD will be issued based on current evidence. This would assist requestors in planning appropriately and being able to submit a well-developed study plan as soon as possible. 2) Analyze the process for CMS internal evidence review to minimize the time involved or publish specific guidelines for evidence that the requestors could fulfill and submit to CMS with their initial NCD request.
2. Coverage Gaps: Prior to the NCD request, several regional Medicare contractors had issued LCDs in support of HCT for MDS. Upon issuing a decision memo of CED, the local contractors discontinued coverage pending an approved study. Due to the timeline issues noted previously, many patients were caught in an inadvertent and unexpected coverage gap. We assume that a decision of CED conveys the intent of CMS to cover the item or service at question, particularly if positive LCDs have already been in place.
Suggestion: Consider requiring contractors with local LCDs in place to continue coverage until a national study has been approved and implemented, or establish a mechanism for retroactive billing of cases that meet study requirements but fell into the post-decision memo/pre-study approval time period.
3. Billing Instructions: We appreciate the billing instructions issued by CMS prior to the beginning of the study. However, we have found several circumstances that were beyond the scope of those instructions and have needed to contact CMS for more information. While those situations were new to our organizations, they likely are relatively routine for items or services under CED.
Suggestion: It would be helpful to have additional information related to special circumstances that are common for CED, such as billing procedures when a patient is enrolled in a Medicare Advantage plan; or when a patient carries additional pertinent medical diagnoses, including one that has been previously determined as ineligible for the service at issue.
Potential Impact of CED: We agree with CMS that CED has great potential to accelerate Medicare beneficiaries’ access to new items and services. CMS also notes that many new technologies are developed with insufficient attention to addressing the needs of the Medicare beneficiary population. Requiring specific evidence in a certain age cohort may negate much of CED’s potential for accelerated access. For example, HCT has been developed to successfully treat the disease indications, which have not been shown to differ in their pathology based on age. Allogeneic HCT is a standard of care for many indications and is performed 7,000 times annually in the United States, but is considered unproven based on Medicare NCD criteria. Its demonstrated suitability for Medicare beneficiaries (over and under age 65) for other medical conditions (including, for example, AML and CML) and the lack of any age-specific contraindications suggest that its application to treatment of Medicare beneficiaries for new indications could be guided in part by the well-accepted existing evidence of its effectiveness for numerous other indications.
While the traditional HCT preparatory regimen required modification for older patients, this was due to the dosage and toxicity of the chemotherapy agents, not an age-associated deficiency in the treatment mechanism itself. These modifications have also been widely and appropriately applied in treating patients of any age with various co-morbidities to allow safer application of the therapy. Requiring age specific data is particularly difficult for a low volume treatment like HCT, as Medicare is the most common payor for patients in the beneficiary age cohort. Accruing enough data for a specific indication with a low volume treatment outside of the primary payor in that cohort significantly delays establishing that particular body of evidence and accordingly, access to a potentially curative treatment.
Additionally, CMS appears to be particularly interested in data in patients above the age of 65 while a approximately 16% of beneficiaries are below age 65 and qualify for Medicare coverage based on disability or debilitating illnesses (AARP Public Policy Institute). The illnesses may include hematologic malignancies or other coexisting medical conditions, but do not preclude safe applications of allogeneic HCT. Based on the current process, these beneficiaries are excluded from coverage unless it is expressly indicated in the older beneficiary population despite being within an age cohort where evidence has already been clearly established.
Suggestions: 1.) Unless evidence indicates otherwise or is established in only a very specific cohort, CMS should consider accepting data from the general adult population as the basis for coverage decisions with particular attention to available data on patients over the age of 50 whose experiences may be directly informative to the Medicare beneficiary population. 2.) If age-based evidence is required, consider conditional access for beneficiaries under the age of 65.
Maximizing Benefits of CED: CED requires a significant investment of resources. Small professional societies or clinical organizations may not have the funding or staff resources to build the infrastructure necessary to support an approved study. Alternatively, some organizations may already be tracking outcomes in a way that does not meet the particular design elements of the CMS study requirements, but would satisfy the intent and focus of CMS’s inquiries. Finally, having to request a NCD and create a separate study and database for CED approval for each clinical indication of a particular treatment creates a significant barrier to expanding use of that service even when clinically indicated.
Suggestions: 1) Partner with professional societies or organizations with well-established registries or outcomes databases to utilize their current data tracking mechanisms for items or services of interest to CMS. 2) Allow for the evaluation of multiple indications utilizing the same treatment mechanism and/or allow for sequential addition of indications as the gathering of evidence under CED for the original indication proceeds to conclusion.
In summary, we feel that CMS should adopt measures to increase speed and flexibility in order to realize the potential of CED for the Medicare beneficiary population. We would also like to commend the responsiveness of the Coverage and Analysis Group staff since our study has been opened. Roya Lofti and Leslye Fitterman have been very prompt and thorough in their responses to our procedural questions.
We would welcome the opportunity to further discuss any of the suggestions presented in this comment. Please contact us with any questions or concerns.