Quality Assured Use of Pharmaceuticals: Does German Gba Follow Ema?

• Published in: Value in health Vol. 20; no. 9; p. A699
• Main Authors: Menzler, Jzzz, Minartz, C, Thienel, P, Neubauer, AS
• Format: Journal Article
• Language: English
• Published: Lawrenceville Elsevier Science Ltd 01-10-2017
• Subjects: Cancer; Drug status; FDA approval; Health authorities; Market entry; Medical personnel; Metabolic diseases; Metabolic disorders; Pharmaceuticals; Physicians; Prescription drugs; Rare diseases
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2017.08.1805

OBJECTIVES: After receiving the EMA approval for Europe, it is mandatory for pharmaceuticals in Germany to complete the AMNOG process to receive reimbursement. Finally the responsible G-BA (joint federal committee) publishes a decision not only on the added benefit of the drug, but also on any specific requirements for the quality assured use. These requirements may differ from the EPAR and SmPC specifications for quality assured use of the drug. METHODS: For all 110 AMNOG applications started and finished between January 2015 and June 2017 we compared the specifications of the AMNOG decision for quality assured use with the respective EPAR and SmPC documents. We categorized differences and determined its relative amounts. RESULTS: The G-BA decisions were clustered into three groups: [A] Complete agreement with the EPAR/SmPC documents (52%), [B] Concretization of the EPAR/SmPC requirements (41%) and [C] divergent requirements by G-BA (7%). In cases of category [BJ the G-BA named the type of specialized physicians indicated by the EMA phrasing "...a physician experienced with...". All products of category [C] receiving divergent specifications (7%, n=8) were orphan drugs and two of them had an oncologic indication while the others were for metabolic diseases. While EPAR and SmPC mentioned "health care professionals" or had no specific assignation, the G-BA assigned the application of the drug to specific disciplines. The G-BA gives no explicit reasons for this decision, but the orphan drug status appears to be an important driver. CONCLUSIONS: In case of rare diseases German Health authorities request supervision by a specialized and experienced physician, also in cases where the EMA does not. This should be taken into concern when the market entry of an orphan drug is projected, especially for critical indications like cancer or severe neurologic and metabolic diseases.