Off-label prescriptions in dermatology: A prospective, multicentre and supraregional study of applications and decisions on the reimbursement of costs in Germany

• Applicant: Dr. Ricardo Niklas Werner
• Subject Area: Public Health, Healthcare Research, Social and Occupational Medicine ; Dermatology
• Term: from 2019 to 2021
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 417609812

"Off-label use" is defined as the prescription of pharmaceutical products or interventions outside their approved indications. Rare diseases frequently lack "in-label" treatment options. Given the large amount of disease entities with a low prevalence such as pemphigus vulgaris or systemic scleroderma, the field of dermatology is particularly affected by problems arising with off-label use. Cost coverage of off-label prescriptions by the statutory health insurance is only warranted under specific circumstances. In order to avoid reimbursement claims, physicians in Germany can - on a case-by-case basis - file an application for cost coverage by the health insurance prior to the prescription of off-label therapies (off-label application, OL-A). Due to processing times and possible rejections, this may be time consuming and lead to a delay of the initiation of relevant treatments for patients who have rare diseases. Intention of the planned healthcare research project is to assess and analyze data concerning the frequency of OL-A, decisions on the reimbursement of costs, time consumption and implications for patient care. OL-A and the consecutive correspondence will be collected consecutively from German university hospitals offering a dermatology outpatient clinic. Apart from these data derived from routine patient care, further aspects (e.g., duration between decision for and initiation of an off-label treatment) will be assessed. Outcomes of the project will be the frequency and relevance of off-label use, the generation of a list of frequently approved interventions for specific indications and the quantification of the delay in the initiation of patient treatment. Furthermore, we will investigate whether approval rates and duration between the decision for and initiation of an off-label treatment are determined by any of the following factors: health insurer, region, quality of the OL-A, costs of the requested treatment, or patient age.The study aims at investigating whether the current legal circumstances restrict an optimal patient care and what amount of time expenditure is attributable to the elaboration of OL-A. Determinants of approval rates will be revealed and regional disparities or differences between health insurers assessed. The generation of a list of frequently approved interventions for specific indications may serve as a basis for future research to establish standardized criteria for approvals of OL-A. Moreover, the list of frequently approved off-label prescriptions may stimulate the expert groups of the German regulatory institution (Bundesinstitut für Arzneimittel und Medizinprodukte) to include further positive recommendations into the list of approved off-label use in Germany (“Arzneimittelrichtlinie”). In the context of health care policy, the study results may encourage a discussion of the situation of patients who have rare diseases and possibilities to improve medical care processes.

• DFG Programme: Research Grants
• Co-Investigator: Professor Dr. Alexander Nast