Improved orphan drug availability by the EU pharmaceutical package?
Is it realistic?
On April 26, 2023, the EU Commission presented the "Reform of the EU pharmaceutical legislation", commonly referred to in Germany as the EU pharma package. Among other things, this comprehensive reform is intended to replace and simplify the previous pharmaceutical regulations. Its aims include ensuring the supply of affordable medicines to patients and strengthening the security of supply, particularly in indications with a high unmet medical need (e.g. regarding antibiotic resistance).
There is also a particular focus on providing patients with medicines for rare diseases (orphan drugs) and pediatric medicines. Research into diseases that only affect a small group of patients is particularly financially challenging for pharmaceutical companies: high levels of uncertainty make investments difficult. However, research into such rare diseases and the associated development of medicinal products represent an indispensable basis for enabling the treatment of these often severely suffering patients. The development of drugs for rare diseases is also challenging from a scientific and clinical perspective. For example, the planning of robust clinical studies and the selection and recruitment of sufficient and suitable study participants are significantly more difficult.
In order to incentivize the development of orphan drugs, the EU pharma package provides for an extension of the period of market exclusivity, i.e. the period during which no similar drugs for the same indication can come onto the market after a drug has been approved. Until now, this period has been ten years. The EU pharma package (as of February 2024) would initially reduce this period to nine years, although it can be extended to 13 years if certain conditions are met. These conditions include, for example, the introduction of the medicine in all EU member states or meeting a high medical need. Whether these incentives will actually be effective is at least questionable: can a small pharmaceutical company with fewer than 50 employees even meet the above requirements in all 27 EU member states?
In total, there are around 6,000 to 8,000 rare diseases - therapies are currently available for around 200 of these diseases. This means that there is still no approved causal therapy for around 98 % of affected patients. Against this background, the importance of research into rare diseases and the development of drugs for these diseases becomes particularly clear.
The extent to which the changes proposed by the EU pharmaceutical package can have a positive impact on the supply of orphan drugs to patients remains to be seen. In any case, the market players will face changes that need to be prepared for. However, the election of the EU Parliament will have an impact on the original timeline. As in the recent past, there may still be relevant changes to the scope of the EU pharma package.
SKC Beratungsgesellschaft has been advising pharmaceutical, biotechnology and medical technology manufacturers on upcoming legislative changes and their implications for their products for almost 20 years. We incorporate our assessments of the developments and their consequences directly into the advice we provide to our clients. We are the market access special forces.
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