Advanced therapy medicinal products include cell therapies, gene therapies, and tissue-engineered products. These highly complex treatments differ from traditional medicines, both in how they are made and administered and in the type of benefits they may provide. These are products for which regulations were established relatively recently and that present challenges for regulatory agencies on how to best they should be regulated within the existing regulatory framework.
The Alliance for Regenerative Medicine (ARM) in Brussels published its response to the EU Pharmaceutical Strategy Roadmap, which is designed to improve and accelerate patients’ access to safe and affordable medicines and to support innovation in the EU pharmaceutical industry. While ARM welcomes the Roadmap’s recognition of the wider industry’s contribution to the EU economy and its focus on ensuring patients’ access to safe, state-of-the-art therapies, the Alliance wants to ensure a continued pathway for the EU to benefit from innovative and life-changing advanced therapy medicinal products.
More focus on value-based pricing, less restrictive GMO designations, and more attention to hospital exemptions are all priorities for the Alliance for Regenerative Medicine, an industry group representing companies working in the gene and cell therapy space. Carlo spoke to Paige Bischoff, senior vice president of public affairs, about what ARM wanted ahead of their upcoming response to the EU’s Pharma Strategy roadmap.
In the midst of a global public health emergency, some businesses are taking advantage of widespread fears by marketing purported stem cell treatments for COVID-19. Such businesses target prospective clients with misleading claims, expose patients to potentially risky stem cell-based products, and undermine efforts to develop evidence-based treatments for COVID-19.
It’s been four years since the first CAR T cell therapies were approved and inadequate reimbursement is still a barrier to access in the U.S. A new payment rule proposed by CMS aims to change that, and its adoption could provide a road map to faster implementation of payment models for the next new technologies.
Beyond the value of ATMPs for healthcare and considering the total cost for a single patient, it is important to consider healthcare-related costs as well as non-healthcare and indirect costs to perform an appropriate evaluation. It is necessary to have a holistic view on expenses for the government, especially in the case of therapies with high up-front costs.
The excessive inflammatory response seen in the most serious cases of Covid-19, along with shortages of ventilators, has caused a healthcare crisis in many countries around the world. Could cell therapy provide an answer to this dilemma?
Gene therapy is defined as the transfer of genetic information to a patient for treatment of a disease. Clinical investigation of such therapies began in 1990 with a treatment for a rare immunodeficiency disorder and since has expanded to almost 1,000 clinical studies in 2019. In its most straightforward incarnation, the goal of gene therapy for genetic diseases is long-term expression of a transferred gene at levels that are high enough to be therapeutic.
