Friends of Cancer Research Issues Public Comment on Centers for Medicare & Medicaid Services Proposed Rule
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Friends' is very supportive of the proposed rule to create a Medicare Coverage of Innovative Technology (MCIT) program and applaud the
1. Broaden the scope of the proposed rule to include, specifically, Medicare Part B-eligible drugs and biologics with a Breakthrough Therapy Designation (BTD).
2. Improve communications between FDA and CMS.
3. Provide guidance on post-market evidence generation that specifically addresses use of real-world evidence.
Expand MCIT to Facilitate Expedited Access and Reimbursement for Breakthrough Therapy Drugs
In recent years, scientific advancements have begun to yield new therapies that are changing the course of previously untreatable diseases. Policies like the BTD by the FDA help to expedite the development of transformative treatments. While this program has accelerated the development of new medicines, and resulted in timelier regulatory review, it doesn't automatically translate into patients having rapid access to these promising new therapies after they are approved.
Following approval by the FDA, a new drug can be widely marketed and distributed across the country, but before a new medicine is paid for by an insurance company, including government programs like Medicare and Medicaid, there is often a complex process to set up the necessary mechanisms for coverage and reimbursement. This is particularly true for entirely novel treatments that involve new mechanisms of action or new technologies altogether. Many products approved with a BTD fit this description. Delays in setting up coverage and reimbursement policies for innovative new treatments can shift immediate burden to clinics or patients themselves and place a significant barrier to access for people who could benefit from these transformative therapies.
For example, tisagenlecleucel and axicabtagene ciloleucel, the first Chimeric Antigen Receptor T (CAR T) Cell Therapies for indications in cancer, both received BTD and FDA approval in 2017, however, the National Coverage Determination for CAR T was not finalized until
To address this, CMS implemented a policy known as the New Technology Add-on Payment (NTAP) to partially compensate hospitals for the use of new therapies that have yet to be fit in existing CMS reimbursement calculations. However, it is not always clear if a new product will qualify for the program and it doesn't accelerate necessary reclassification after a new therapy is approved. In the case of the CAR T Cell Therapies, the additional NTAP rate received was significantly short of providing adequate reimbursement. The resulting lack of coverage clarity and disproportionate reimbursement level placed significant financial burden on hospitals and results in limitations to patient access.
The current processes for coverage determination and establishment of payment rates leads to concerns that the current system will not adequately facilitate patient access to new treatments as new technologies come to market that are increasingly complex and costly.
Indeed, there are currently hundreds of clinical trials for new cellular therapies underway and the FDA has predicted a substantial increase in BLAs in the coming years. The existence of a coverage pathway for BTD treatments to facilitate coverage for a predetermined period of time, during which a NCD, new payment codes, or additional evidence could be generated, will be crucial to provide for a seamless transition from FDA approval to full coverage and prevent patient access barriers to new therapies. We encourage CMS to consider broadening the scope of the MCIT to include drugs and biologics that receive a BTD to reduce access barriers to innovative new therapies while the additional evidence generation and necessary administrative processes can occur.
Implement Earlier Communication Between FDA and CMS
BTD represents the opportunity to identify and enact efficiencies for expediting patient access to potentially transformative therapies. Improved coordination between FDA and government-based insurance programs could help with advanced planning and allow CMS to implement parallel processes that would be operational closer to the time in which a new Breakthrough Therapy is approved.
Advanced coordination between FDA and CMS could drive efficiencies that ultimately result in timelier and more comprehensive patient access to novel therapies. In 2016, FDA approved six new molecular entities that had previously been granted BTD./3
First-in-class products that receive a BTD may represent the greatest opportunity and need for increased collaboration between FDA and CMS. One approach could be that upon designation, FDA could begin to share data with CMS about the products and provide their clinical expertise as to the transformative potential of the product. Such an approach would allow CMS to anticipate timelines for potential approval decisions and begin to implement coverage determinations, where necessary, and payment procedures as appropriate (e.g. development of new codes, resource allocation, potential payment agreements, etc). By establishing earlier communication regarding BTD products, CMS could provide added guidance to developers to inform evidence generation to support coverage and reimbursement earlier in the development process, further enhancing predictability and expediting evidence generation.
An expert advisory board focused on innovative therapies could streamline coverage decisions.
Given the rate of change in the external research community, it would be difficult for any agency or organization to effectively respond without regular interactions with experts from varying perspectives. We recommend the establishment of an advisory committee comprised of relevant stakeholders including patients, manufacturers, and physicians to review transformative technologies upon awarding of breakthrough designation and throughout development to provide recommendations to CMS pertaining to coverage and reimbursement.
This committee would be similar to the
Complement Expedited Coverage with Real-world Evidence Generation
The goal of rapid reimbursement processes of potentially transformative new therapies is to facilitate timely patient access to innovative new medicines. Such an approach should be incentivized with an expedited process for coverage and reimbursement yet recognize the need for long term demonstration of value. Given the potential of Breakthrough products to address unmet medical needs, it would be unfavorable to require long-term information about the new treatment prior to establishing initial payment policy. In many cases, a Breakthrough-designated product would represent a novel technology or class of drugs.
Therefore, these advancements may require additional evidence regarding their benefit over time in order to further support the initial efforts to enable access to the product.
Real-world evidence should be developed to aid coverage decisions for Breakthrough Therapies. We note that FDA, in response to 21st Century Cures Act and PDUFA VI, has created the Framework for Real-world Evidence and issued guidance for advancing the use of real-world evidence for regulatory use. However, we would emphasize that RWE can also be leveraged in the post-market setting to support coverage and reimbursement. This approach could allow for subsequent studies, including studies required as part of FDA post-approval commitments, or real-world evidence to be used as part of a longer-term value assessment to support early and expedited reimbursement mechanisms designed to ensure rapid patient access and could include a post-market evidence review at a set time period to assess the value that the product provides.
We recommend that the proposed rule include guidance regarding mechanisms for post-market benefit assessment of products that utilize a rapid reimbursement pathway. This would include:
* Creating a framework to identify breakthrough designated therapies that represent novel technologies and may encounter coverage or reimbursement barriers and provide a rapid reimbursement pathway while additional evidence is generated,
* Issuing guidance for sponsors of breakthrough therapies regarding the type of evidence that may be possible to develop in the post-market setting, timelines for post-market evaluation of evidence, and inform real-world evidence that could be used to support determinations of coverage, and
* Working with stakeholders to develop a list of real-world outcomes that are important to patients and that be used to support coverage determinations.
For therapies that demonstrate early indicators of substantial improvement, a shift toward post-market evidence collection may be necessary to balance the need for adequate supportive evidence with appropriate patient access. However, while considerations to shift development of evidence in the post-market space will be an important step to advancing access to breakthrough therapies, this will not be effective without parallel policies to support coverage and reimbursement decisions through improved communication between FDA and CMS. In addition to avoiding redundancy in analyses performed by both agencies, such an approach would allow CMS to anticipate timelines for potential approval decisions and begin to implement payment procedures, to be applied at the point of approval by FDA. It should be noted that the vast majority of BTD products ultimately achieve FDA approval. Therefore, earlier planning of payment policies and procedures needed for these drugs would represent a shift in timing of resource allocation that otherwise might be spread over a longer period of time.
Conclusion
Friends is appreciative of the opportunity to provide input on the MCIT Proposed Rule and look forward to the opportunity to discuss our comments with you.
Sincerely,
President & CEO
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Footnotes:
1/ The CAR T Cell Therapy NCD was the first ever NCD to be issued late.
2/ Hernandez J, Machacz SF, Robinson JC. US hospital payment adjustments for innovative technology lag behind those in
3/ OMPT FDA, as of 12/16/16: https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/UCM533192.pdf
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The proposed rule can be viewed at: https://www.regulations.gov/document?D=CMS-2020-0098-0002
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