Recordings Available: ARM’s Cell & Gene Webinar Series
This spring, ARM hosted experts on biotech fundraising, market access, value-based therapies, clinical trials amidst the pandemic, and cell therapies to treat COVID-19 as a part of a webinar series on timely sector issues.
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.
Potentially curative cell and gene therapies are changing the face of medicine and providing hope to patients where there was once none. But how will they be paid for? This webinar discusses the implementation of value-based payment models for innovative therapies.
This webinar highlights potential treatments for Acute Respiratory Distress syndrome (ARDS) in COVID-19 patients. Two therapeutic developers, Athersys and Pluristem Therapeutics, presented on their treatment approaches and plans for near term clinical trials.
ARM’s annual State of the Industry briefing provides an update on global financings, clinical development, public policy considerations, and other regenerative medicine sector trends. A recording of the presentation is available here.
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.”