Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

New Requirements and Legal Framework for Orphan Drug Medicinal Products with Conditional Marketing Authorization for Data Generation according to the "Gesetz für mehr Sicherheit in der Arzneimittelversorgung" (GSAV – Law for more Security in Drug Supply) in Germany

Uwe Faude (Abschlußjahr: 2020)

Summary
Language: English
As of January 2020, 132 drugs are approved in rare indications within the scope of the European Union. This must be recognized as a great success of the European regulatory framework for drugs with orphan drug status and often makes a significant contribution to improving the quality of life for affected patients. The often limited data basis of the clinical trial program, which is used as the basis for the European approval procedure and thus also has legal force for the area of application in Germany, has been controversially discussed by various stakeholders for some time. Examples of approved drugs in rare indications in recent years support this and are listed. To improve the data basis for the evaluation of the clinical efficacy of orphan drugs, this topic was introduced into a legislative process on a national basis (Germany) and implemented in 2020.
As a result of a broad discussion in expert circles and interest groups, which are listed, regulations were included as part of the law for more safety in drug supply and implemented in the legislation in the Social Code Book V. The resulting consequences (application accompanying data collection) for pharmaceutical entrepreneurs and marketing authorization holders are listed and critically discussed.  In addition, study designs and clinical trial strategies with the corresponding recommendations and minimum requirements are presented in the context of the legislative process.
With reference to a current new approval of a drug in the (rare) indication spinal muscular atrophy and taking into account current medical-scientific and statistical bases, presented by the Institute for Quality and Efficiency in Health Care and other institutions and professional societies, effects are discussed and supported by own proposals on how the data collection required by the legislator in Germany can be implemented. Limitations of the data collection accompanying the application are also listed and explained, especially with regard to statistical requirements and the interpretation of the results to the basic population.
Using the indication spinal muscular atrophy, it is shown how a selection of suitable clinical variables and surrogate parameters could contribute to the improvement of the validity of clinical data from application-accompanying data collection, if, as is currently foreseeable, data collection after the approval of drugs in rare indications will take place in the context of non-controlled (non-randomized) register studies.
Pages: 71