ARM advances specific proposals to enable coverage, coding, and payment policies that facilitate the development of and patient access to regenerative medicine and advanced therapy products.
Reimbursement refers to how a healthcare provider is paid by private insurers or government payers, including Centers for Medicare and Medicaid Services (CMS) in the U.S. and the National Healthcare System (NHS) in the U.K., for example. Under the current, decades-old reimbursement system, if a patient has a severe or chronic disease or disability, payers (both private and public) typically expect to pay for each treatment or interaction with a health care provider over the course of many years—potentially throughout the patient’s entire lifetime.
Gene and cell therapies and other regenerative medicine products are different. Many are designed to durably and even permanently address the underlying cause of a disease. They may be given in just a few administrations, or even a single dose. While these therapies can provide significant direct and indirect savings in medical costs over time, their potentially high upfront cost can create a significant burden on existing reimbursement systems. While reimbursement systems have begun to change to accommodate these new treatments, they still have a way to go to catch up with the immense value provided by advanced therapies.
In the United States, CMS typically sets the standard for reimbursement through its decisions regarding coverage and payment through the Medicare program. In the European Union, individual countries regulate their own respective healthcare systems, with the European Commission encouraging coordinated efforts in cross-border healthcare. ARM works with these and other public payers as well as with commercial insurers to promote reimbursement and payment policies that enable innovation in regenerative medicine.
In comments submitted to the European Commission, ARM highlights the potential for the European Health Data Space (EHDS) to advance patient access to cell and gene therapies.
ARM’s letter in response to the Medicaid and CHIP Payment and Access Commission’s (MACPAC) potential recommendation to Congress that there should be differential rebates for drugs approved via the FDA’s accelerated pathway until Phase IV confirmatory studies are completed argues that such an approach could disincentivize the development of cell and gene therapies, negatively impact patient access, and contradict congressional intent.
ARM states that, while the draft report shows the benefits of these innovative therapies, concerns remain that ICER doesn’t properly evaluate curative therapies and that conducting the review prior to FDA approval is premature, potentially harming patient access.
ARM applauds ICER for addressing needed updates regarding patient engagement, clinical trial diversity, and real-world evidence priorities; however, ARM raises critical concerns with ICERs approach for assessing the value of cell and gene therapies, particularly as is relates to timing of their assessments and considerations around health benefit price benchmarks.
ARM and BIO support the creation of International Statistical Classification of Diseases and Related Health Problems (ICD) 10 codes for intravenous infusion of approved CAR-T therapies.
ARM filed comments in response to CMS’ proposal “Medicare Coverage of Innovative Technology and Definition of ‘Reasonable and Necessary.'” Expanding the proposed new pathway to biologics could impede patient access and create coverage confusion.
ARM commends CMS for including lamguage in their draft IPPS rule to establish a separate CRG for CAR-T therapies, and urges that the proposal be finalized and that CMS apply the learnings from the CAR-T experience and expedite reimbursement decisions for future cell and gene therapies as well as evolving the new technology add-on payment (NTAP) program to promote further access to innovative therapies and increase the payment cap.
ARM commends CMS for recognizing the need to address several of the major federal barriers hindering the establishment of value-based payment models for government health programs. ARM recommends that the agency “allow for the greatest amount of flexibility to accommodate the wide range in the types of cell and gene technologies…” and that “manufacturers and payers should be allowed considerable discretion to negotiate the terms of the VBP arrangements.”
ARM and BIO submitted a joint letter to the Centers for Medicare and Medicaid Services (CMS) requesting that the agency issue guidance to hospitals regarding appropriate charges for CAR T-cell therapies. ARM and BIO are concerned that “some hospitals are reluctant to set charges for CAR T-cell therapies that are in line with the CCR (cost-to-charge ratio), and as a result, applying the standard CCR to their charges will underestimate the cost of CAR T therapies.”
ARM recommends that: (1) manufactures should be allowed to pay for travel and lodging expenses for the patient of a Medicaid Health Home and their immediate family; (2) CMS should eliminate reimbursement as an access barrier by requiring states to reimburse providers or facilities 100 percent of the applicable Medicare rate for that facility or provider; and (3) CMS should create one standardized process for all the states to use to simply verify that the patient meets the medically accepted indication of the physician prescribed gene, regenerative or immune therapies and to approve and credential a facility or provider to administer the therapy to out of state patients.
BIO and ARM recommend establishing a DRG for Fiscal Year 2021 for CAR T-cell therapy cases to provide appropriate reimbursement for treatment and protect access to care for Medicare beneficiaries. It’s critical that this be developed for FY 2021 before the new technology add-on payment (NTAP) expiration for currently available CAR T-cell therapies occurs on September 30, 2020, which would result in a significant drop in provider reimbursement nationwide that could impact patient access to CAR T.
The HTA evaluation issues raised in the ICER press release raise critical concerns for ARM members. ARM requests that ICER augment efforts to use Real World Evidence; expand and revise voting structure to capture important potential other benefits and contextual considerations not captured in the cost-effectiveness analysis; and create a new process for re-assessing the emergence of new evidence. ARM also highlighted areas of concern, including the premature timing of review, cost-effectiveness threshold ranges, the discounted approach to cost-effectiveness modeling, imprecise evidence ratings, and the use of the German HTA system prior to an international HTA standard being set.
ARM believes that while there are some positive elements in ICER’s proposal, overall, the value assessment methods are inadequate to fully reflect the long-term value of these transformative therapies.
ARM appreciates that CMS has discussed a few options to improve the current NTAP and overall MS-DRG system with a focus on creating a methodology and a system that balances appropriate access and cost effective care to new and innovative therapies. ARM suggests that CMS adjust its CAR T specific reimbursement policies to utilize actual drug acquisition costs, rather than marked up charges in NTAP and outlier payment calculations for fiscal year (FY) 2020; finalize policies for FY 2020 that create the infrastructure to move toward an appropriately valued CAR T MS-DRG in future fiscal years; recognize certain FDA approval designations for drugs as dispositive for newness and substantial clinical improvement; and establish a more frequent NTAP process. ARM also disagrees with CMS’ proposal to change the severity level designation for 13 ICD-10-CM diagnosis codes from categories I21 and I22 from a MCC to a CC.
While ARM is supportive of the OIG’s efforts to reexamine the applicable regulatory safe harbors currently in place under the anti-kickback statute, we are concerned that the proposed change would serve to also exclude arrangements involving the application of price concessions based on value.
It is critical for CMS to develop and implement policies and programs that support beneficiary access to new technologies when they are deemed most clinically appropriate and stimulate their continued development. This is particularly true for regenerative medicine and other advanced therapies that hold the promise of durably treating and potentially even curing chronic or life-threatening diseases. In light of this goal, ARM asks CMS to ensure Medicare patients who can benefit from CAR T therapies are covered under the NCD, allow CAR T to be administered in certified, trained, experienced facilities and not limit access to hospitals, and certify a registry to be in place by May 17 to ensure continued access to CAR T by Medicare beneficiaries.
It is ARM’s view that traditional HTA frameworks in both U.S. and Europe are not flexible enough to accommodate potential cures and do not allow the ability to capture the full product value due to issues including: the short term time frame for assessing affordability versus the long-term timeframe for assessing value; variability in willingness to pay (and applicability of ICER threshold) based on degree of unmet medical need addressed; and the subjectivity of incorporating contextual considerations such as caregiver and societal impacts into a quantitative framework.
ARM filed comments in response to a Trump Administration proposal which would change the current Medicare Part B reimbursement model, which is based on a manufacturer’s average sale price (ASP) in the United States in combination with an add-on payment, to one that would adopt an International Pricing Index. While commending the effort to align value with reimbursement, ARM believes that the proposal will fall short of this goal and harm patient access to regenerative medicines.
ARM appreciates CMS’ statements related to the similarity between data satisfying FDA designations and data satisfying substantial clinical improvement. This clarity will help future applicants understand which types of data can serve as the foundation for the satisfying the substantial clinical improvement criterion. In addition, ARM urges CMS to change the payment rate for certain classes of NTAP recipients. ARM is concerned that without changes to the current reimbursement methodology for CAR-T therapies and other regenerative and cell therapies that ARM represents, the NTAP will not satisfy the congressional intent of the NTAP program, which is to adequately reflect the estimated average cost of such service or technology.
ARM submitted comments to the Department of Health and Human Services’ Office of Inspector General in response to an RFI concerning the Anti-Kickback Statute and value-based payment models. The current structure of the Anti-Kickback Statute creates regulatory and legal barriers to federal implementation of value-based payment models for innovative, one-time and durable therapies. ARM encourages the OIG to consider the cost of initial assessments, the potentially limited geographic access to regenerative medicine products for patients, and the need for continuing evaluation when developing safe harbors.
ARM provided comments on the CMS requests for information on promoting interoperability and electronic health care information, price transparency, and leveraging authority for the Competitive Acquisition Program (CAP) for Part B drugs and biologicals for a potential CMS innovation center model.
ARM provided comments in response to the House of Representatives’ Health Care Innovation Caucus request for information regarding value-based payment reform and value-based arrangements.
ARM submitted comments on the Department of Health and Human Services (HHS) Request for Information: Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs, encouraging HHS to consider the potential value of cell and gene therapies to patients and society, and the need to enable new pricing and reimbursement approaches that can help make them available to patients.
ARM published its position in response to the proposal for a Regulation of the European Parliament and of the Council on health technology assessment and amending Directive 2011/24/EU (COM(2018) 51 final). ARM’s position statement recognizes the benefit of the proposed regulation, which would require joint clinical assessments to be carried out for many medicinal products undergoing the central marketing authorization. In the statement, ARM also makes three recommendations intended to ensure the success of the regulation in attaining the full impact of those benefits.
ARM submits comments to CMS requesting that the agency rescind the planned NCA on the basis that it was prematurely undertaken and because the NCA may have a significantly chilling effect on access to the approved therapies and curtail clinical and financial investment in these technologies. If CMS continues with the NCA, ARM has identified several issues which we encourage CMS to acknowledge and address.
ARM submits comments in response to the Office of Inspector General’s request regarding the OIG’s “safe harbors,” activities that are deemed in advance to not violate the Anti-Kickback Statute and other laws. These provisions can complicate or limit pay-for-performance arrangements with CMS.
ARM Co-Founder & Senior Policy Counsel Michael Werner delivered comments at the CMS public meeting.
ARM provides policy recommendations to mitigate legislative and regulatory hurdles impeding the adoption of innovative payment models for regenerative medicines.