The 2019 ARM Annual Report & Sector Year in Review provides an overview of the organization activities, initiatives, and successes over the last year. In addition, the report provides an update on the nearly 1,000 regenerative medicine therapeutic developers active worldwide.
Manufacturing Cures: A Roadmap for the Development of Gene Therapies
Senior ARM representatives Janet Lambert, CEO, and Michael Lehmicke, Director, Science & Industry Affairs, provide insights on the upcoming publication of A-Gene, a case-study based guide on applying Quality-by-Design principles to the development of gene therapies.
Report Demonstrates Potential for Cell and Gene Therapies to Provide Cost Savings
Produced by the Marwood Institute and funded by ARM, this report uses a “Transformative Therapy Value Model,” and demonstrates that durable cell or gene therapies for multiple myeloma, sickle cell disease, and hemophilia A could provide cumulative cost-savings of $33B over the next ten years.
Companies Developing Regenerative Medicines for Rare Disease Raise $6.4B in 2019
ARM’s 2019 rare disease report provides an overview of gene therapy, cell therapy, & tissue engineering therapeutic companies active in developing therapies for rare diseases. The report also provides a breakdown of the 647 clinical trials in rare diseases utilizing regenerative medicine technologies.
This webinar features FDA representatives Tejashri Purohit-Sheth and Ilan Irony from the Office of Tissues and Advanced Therapies, CBER, as they discuss the effect of COVID-19 on cell and gene therapy clinical trials. Discussion topics include assuring the safety of trial participants, maintaining compliance with good clinical practice (GCP), and minimizing risks to trial integrity during the COVID-19 pandemic.
Understanding the Coronavirus Emergency Loan Program for Small Businesses
This webinar features speakers Mary Beth Bosco, Nicole Elliott, and Joel Roberson, Partners at Holland & Knight LLP, to discuss the emergency loan program for small businesses established by the Coronavirus Aid, Relief, and Economic Security (CARES) Act. This fiscal stimulus program went live April 3, 2020 and loans will only be available until funds are exhausted, so the government is advising borrowers to apply as soon as possible.
As more regenerative medicine products approach clinical testing and commercialization, seize this opportunity to hear detailed insights and industry best practices from preeminent sector experts. Gain a deeper understanding of the many manufacturing and industrialization hurdles unique to bringing durable and even curative therapies to patients in need.
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.
ARM recommends that the agency issue separate guidance specifically for rare and serious diseases with unmet medical needs and expressed concern “that the draft guidance does not acknowledge the regulatory flexibility for serious conditions.”
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 provided a list of recommendations that include: removal of barriers to the adoption of alternative payment models; improvements to the New Technology Add-On Payment (NTAP) program; greater oversight of unregulated stem cell clinics; increased funding for FDA reviewers; and expedited coverage by state Medicaid programs of newly approved drugs.
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.