This article explains some of the terminology relating to advanced therapy medicinal products (ATMPs), including gene and cell-based therapies, tissue-engineered products, and combined ATMPs. The author explains the key EU regulations and guidance documents for each therapy type, and discusses marketing authorization, accelerated regulatory pathways, and market access. He advises companies navigating this complex regulatory environment to engage with the regulatory agencies early and often during the approval process and to seek external regulatory support if in-house resources are limited. Introduction 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 fairly recently and that present challenges to the regulatory agencies on how to best to regulate them within the existing regulatory framework. Terminology Gene therapies Genes therapies treat disease by replacing, inactivating, or introducing genes into the body (see Figure 1). They deliver a corrected copy of a faulty or missing gene, typically using a vector or carrier molecule. The new gene allows cells to function correctly, alleviating all, or some, disease symptoms and potentially offering a long-term cure. Gene therapy medicinal product refers to a biologic medicinal product, which has the following characteristics:

Cell-based therapies Cell-based therapies involve transplanting substantially manipulated cells or cellular material into a patient. In some cases, the genetic material in the cells may be genetically modified. Cell therapy restores or alters certain cells to deliver therapy. Cells are modified outside of the body first.

Cells may be from the patient (autologous) or from a donor (allogeneic). Autologous therapies are manufactured using cells taken from a patient, which are then re-administered to the same patient. Therefore, each batch is unique and irreplaceable. Allogenic products are those in which batches are manufactured using material from a single donor and administered to different patients. Somatic cell therapy medicinal product refers to a biological medicinal product, which has the following characteristics:

Guidance documents The European Medicines Agency (EMA) provides several guidance documents and operates a specific help desk email address (advancedtherapeis@ema.europe.com):

FIGURE 3 Marketing authorization pathways for ATMPs Reprinted with permission (Detela & Lodge)32 Market access As of May 2020, 10 ATMPs have been granted marketing authorization in Europe seven gene therapies, one cell therapy, and two tissue-engineered products targeting several diseases in different therapeutic areas. An additional five approved ATMPs were removed from the market for commercial reasons. At the time, marketing authorizations for five gene therapies were pending.38 The high number of approved, but not marketed, products can be explained at least partially by the cost of ATMPs. It is, however, important to note the fundamental differences between conventional therapies and ATMPs (see Table 2).

Outlook Navigating this complex regulatory environment means companies need to engage with the regulatory agencies early and often throughout the approval process. Many of these therapies are developed by scientific institutions and medical research groups with typically limited regulatory resources in-house, so it is advisable to seek external regulatory support. This exciting field, with hundreds of products currently tested in clinical trials,39 should yield many new therapies with enormous benefits. Something to look forward to, particularly in a time of a global pandemic crisis. Abbreviations ATMP, advanced therapy medicinal product; CAT, Committee for Advanced Therapies; CHMP, Committee for Medicinal Products for Human Use; EMA, European Medicines Agency; EU, European Union; GMP, good manufacturing practice; SMEs, small- and medium-sized enterprises. References [All references accessed 16 July 2020]

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Advanced therapy medicinal products regulations in the EU - Regulatory Focus