OBJECTIVES: ; Advanced Therapy Medicinal Product (ATMPs) hold great promise as treatments for previously incurable diseases. While expectations are high and full pipelines are observed, few ATMPs are currently on the EU market. This study aimed to identify root-causes of challenges experienced in achieving successful market access (MA) for commercially developed ATMPs in Europe.

METHODS: ; We completed a qualitative case study by conducting 10 semi-structured interviews amongst ATMP company representatives. MA success was defined as receiving reimbursement for an authorized product in at least one Member State. Company MA actions, strategies, experiences and know-how were questioned, as well as how was dealt with encountered challenges. Developers in the study sample differed in: ATMP type (cell and gene therapies), development stage, (Phase I-II, Phase III, regulatory application, Phase IV, discontinued) and company size (Small-Medium-sized Enterprises (SMEs) and large companies). Interviews were recorded, transcribed and coded. Recurring themes were classified via thematic content analysis.

RESULTS: ; Large companies had more experience developing non-ATMP products, giving them an advantage over SMEs in creating value dossiers and HTA authority contact. Large companies also experienced more financial stability. Therefore, they were able to look ahead, while SMEs were also occupied funding their current development stage. Developers who early on involved MA expertise (in-house or external) and had a detailed business plan from the start, seemed more successful. Respondents mentioned a business plan should at least include: short- and long-term goals, market access strategy and return on investment predictions. Some SMEs partnered with large companies to solve funding issues and utilize expertise. No differences between cell and gene therapies were found.

CONCLUSIONS: Obtaining market access for ATMP development requires more planning and specialised skills compared to more traditional pharmaceutical development. A MA strategy and business plan should be drafted as soon as possible in ATMP development.