New Real-World Data and Pharmacoeconomic Studies for NPP Products
On 19 January 2026, the Turkish Medicines and Medical Devices Agency announced a new initiative focusing on real-world data and pharmacoeconomic analyses for medicines supplied from abroad within the scope of named patient programs. The initiative applies to medicines obtained under the regulation governing the supply of medicines from outside Türkiye, which are typically used in situations where no authorized or available alternative exists in the domestic market and access is granted through special authorization mechanisms.
While the initial use of such medicines is supported by clinical trial data, the Agency underlined the need for additional evidence derived from routine clinical practice in Türkiye. Within this framework, real-world data will be collected to assess the effectiveness, safety, and patterns of use of imported medicines under everyday healthcare conditions. In parallel, pharmacoeconomic analyses will evaluate the relationship between clinical outcomes and costs, with the aim of determining the overall value of these treatments for the healthcare system. According to the announcement, the results of these studies are expected to support evidence-based decision making in areas such as reimbursement, budget planning, and future access policies, while complying with ethical standards and data protection requirements and being shared publicly.
Recent reimbursement case law has increasingly shaped the way courts formulate their questions in disputes concerning access to and reimbursement for medicines, regardless of whether a product is authorized but not reimbursed, or supplied through named patient programs in the absence of domestic authorization. Rather than treating reimbursement as a formal consequence of regulatory approval or exceptional access, courts now approach each case through a structured evidentiary inquiry. This inquiry focuses on individualized clinical need, comparative effectiveness against reimbursed alternatives, the availability and scope of completed clinical phase data, and the proportionality of public expenditure.
The Agency’s initiative thus not only addresses the evidentiary needs highlighted by recent case law but also lays the groundwork for potential future regulations that could more explicitly define the collection, use, and evaluation of real-world and pharmacoeconomic data for medicines supplied under named patient programs.