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.
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.
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.”