Advanced therapy medicinal products (ATMPs), challenges of their regulatory pathways and their global potential in rare diseases

Description: Despite advances getting on the agenda of policy makers and regulatory authority leadership, rare diseases remain an underserved area in diagnosis and available treatment options. Many rare diseases can have devastating consequences for patients, their families and wider social context.
Advanced therapy medicinal products (ATMPs) represent a groundbreaking class of therapies that leverage genetic, cellular, and tissue engineering technologies to treat a variety of diseases, including rare genetic disorders and cancers. These treatments comprise as some of their main groups: gene therapies, which involve the insertion of recombinant genes to achieve therapeutic effects; somatic cell therapies, which utilize manipulated cells or tissues to alter biological functions; and tissue-engineered products designed to repair or regenerate damaged tissues.
The regulatory landscape for ATMPs is complex, governed by stringent guidelines established by e.g. the European Medicines Agency (EMA) and US FDA. These frameworks ensure that ATMPs undergo rigorous evaluation for safety, efficacy, and quality before they can be marketed
Despite their potential, the development and regulatory approval of ATMPs face significant challenges, including limited global regulatory alignment on product type definitions and classifications, high manufacturing costs, the need for robust clinical evidence, and navigating the intricacies of regulatory compliance. As the field evolves, ongoing collaboration between regulatory bodies, industry stakeholders, patient representatives and healthcare providers is essential to address these challenges and optimize the integration of ATMPs into clinical practice, ultimately enhancing patient outcomes and expanding treatment options for unmet medical needs.
The first two presentations of this session will introduce, per region, the EU and US regulatory and development landscape for ATMPs. For the EU, a focus will be, as also illustrated by case studies, on aspects of product classification, use of available interaction pathways with EMA and especially CMC requirements and development aspects.
Gene therapies hold specific potential for rare diseases with early pediatric onset. These diseases are often associated with severe neurological disfunction, a one-time treatment with a gene therapy can be life-saving and holding the promise of a life widely unaffected by the disease. While there are several initiatives of regulatory convergence, EU and US approvals of gene therapies show only limited overlap, with a significant percentage of products approved either at EMA or FDA alone. Even where marketing authorizations were granted in both geographies, differences in approved indications may apply. As part of this session, a focus will be shed on those cases, that were succesful in gaining approval at both, EMA and FDA. It will be examined what differentiates them, in terms of development and regulatory strategy, but also regarding the overall setting of their sponsors. This, especially, as gene therapies for rare disease are a field where non-profit and academic sponsors play an important role, as the traditional commercial pharma model is challenged due to small patient numbers and high manufacturing costs. The session will conclude with a look at possibilities of access to approved gene therapies, for US and EU, but also concerning low and middle income countries.