Will the German Ministry of Health change the orphan drug approval during its EU presidency?
From July until December 2020 Germany will lead the European Union council. Prior to the German presidency, the Federal Ministry of health considers changes affecting the orphan drug approval in Germany. In addition, the bill is also reviewed by the European Medicines Agency (EMA) and the European Commission (APMHE 56398) since a decision is expected to be made during Germany´s EU presidency.
In Germany, approximately 4 million people are affected by rare diseases. The rarity of such diseases as well as economical and medical reasons often prevent an adequate research and treatment of affected patients. The European Regulation on Orphan Drug products (active since 2001) created economic incentives for pharmaceutical companies to produce orphan drugs. 144 orphan drug products have been approved since the EU regulation was introduced in 2001.
The increased number of approvals in recent years resulted in the changes of the legal framework for orphan drugs. Thomas Mueller, head of the department of medicines and medical devices at the Federal Ministry of Health, questions the economic incentives for orphan drugs.
Currently, the additional benefit for orphan drugs is approved in case it is licensed for the German drug market and the G-BA only assesses the extend of the benefit. A new bill on better safety in the drug supply (GSAV) is currently discussed in the German parliament. It will likely take effect in the middle of this year and could lead to considerable changes for the approval of orphans.
The bill introduces new regulations for the calculation of the 50 million Euro threshold for orphan drugs including, beside the outpatient costs, the inpatient costs for the turnover calculation. In case the turnover exceeds the 50 million Euro threshold, the pharmaceutical companies need to prove the additional benefit compared to an appropriate comparator within three months. Therefore, the G-BA is authorized to ask for certain registry data for which the G-BA provides the guidelines. Moreover, Mueller adds that orphan drug approval in the EU is often justified with the argument that patients need them urgently. However, the approval is frequently based on phase II study data that many times fail to fulfill the G-BA´s requirements for the approval of new drugs.
In particular, the new calculation of the 50 million Euro threshold for orphan drugs appears to be problematic, since the inclusion of the inpatient costs in the orphan drug sector will have a significant impact on the orphan drug turnover. This may increase the effort for pharmaceutical companies, while the reimbursement amount may decrease. Based on the register data, the reimbursement amount is supposed to be re-evaluated. The reimbursement may be lowered in case the data do not meet G-BAs criteria .
To conclude, pharmaceutical companies may expect financial losses, which may hamper an adequate research and an appropriate treatment of patients with rare diseases.
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BY Prof. Matthias P. Schönermark, M.D., Ph.D., managing director and Marc Wengler, Dr. rer. nat