Named Patient Programs as an Access Pathway in Germany, France, and the UK

OBJECTIVES: Patient access to rare disease treatments is often delayed due to their lengthy approval and reimbursement process. This has triggered increasing interest in innovative access pathways, including Named Patient Programs (NPPs). With NPPs, patients receive treatments on a case-by-case basis which are not approved in their country but are elsewhere. The prescribing physician, under their direct responsibility, makes the request for the individual patient. This research aims to explore NPPs in three key European markets and when NPPs should be considered.

METHODS: Secondary research was conducted, targeted at NPPs in Germany, France and UK. Identified country-specific terms for NPP expanded the search.

RESULTS: In Germany, “Individueller Heilversuch”, the NPP equivalent, is justified by the emergency regulation of §34 of Criminal Code. It is appropriate only if all other treatment options are exhausted and benefit is assumed for the specific patient based on scientific evidence.

In France, NPP is known as Autorisation d’Accès Compassionnel (AAC) and is granted by ANSM. AAC is valid for a renewable period of one year. The prescriber certifies all granting criteria are met or justifies the request if one/more granting criteria are not met. A hospital pharmacist checks the request and, if AAC is granted, orders/imports and dispenses the medicine.

In UK, NPP is covered by “Specials”, unlicensed medicinal products specially manufactured or imported in response to an unsolicited order by a UK-registered healthcare professional, to address the patient’s clinical needs. Manufacturers must hold a Manufacturer’s “Specials” (MS) Licence, while importers must hold an MS Licence or Wholesale Dealer’s Licence, depending on exporting country.

CONCLUSIONS: NPPs may bridge the problematic “access gap” in a drug’s lifecycle, when there is demand but no accessibility (yet). Pharmaceutical companies should consider NPP as a viable alternative access pathway particularly for ultra-orphan drugs and seek to understand NPP-related country-specific procedures.