ARM fosters investment, research & development, commercialization, and access to safe, effective, and transformational treatments and cures.
Innovation in Released August 6, ARM’s H1 2020 Global Regenerative Medicine & Advanced Therapies Sector Report provides an in-depth look at trends and metrics in the gene, cell, and tissue-based therapeutic sector in the midst of the pandemic. The sector is poised for record-breaking financings in 2020, with several notable policy wins in the first half. Most importantly, patients are continuing to benefit from these transformative therapies.
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.
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.
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.
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.
The webinar includes discussion around how COVID-19 changes the way we think about healthcare decision making and places intense pressure on financial management at all levels of the health system.
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 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.”
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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.