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.
Developing principles of an ARM-endorsed value framework, which will be used to evaluate and influence those of key organizations and payers.
Developing and executing strategies to remove or mitigate new payment model barriers in Medicare and Medicaid, including “Best Price” requirements and application of the Anti-Kickback Statute
Working with CMS to reform Medicare’s program to cover new technologies in the Medicare Inpatient Prospective Payment System, enabling patient access to advanced therapy products, including CAR T-cell therapies.
Defining the key challenges and opportunities for advanced therapy medicinal product (ATMP) access and uptake in the five major EU countries (France, Germany, Italy, Spain, UK), developing consensus recommendations by country to improve access to ATMPs.
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.
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.