On 17th May 2022, the Czech Parliament organised a conference in cooperation with the Association of Innovative Pharmaceutical Industry (AIFP) on the revision of the EU legislation on medicines for children and rare diseases (OMP and Paediatric Regulations). The conference focused on the state of play of the revision and on challenges that will need to be addressed through the legislative framework. The panelists participating in the workshops were high-level representatives of research organisations, patient associations and the pharmaceutical industry.
As Czech Republic will take over the Presidency of the Council of the European Union in July 2022, this conference was particularly relevant to anticipate Czech Republic’s priorities on this topic. In addition, the timing of the conference was highly relevant given that the European Commission is currently preparing a legislative proposal for the revision of the OMP and Paediatric Regulations, which are due for publication in Q4 2022.
RCE’s Steering Committee Chair, Prof. Paolo G. Casali, participated in the workshop dedicated to discussing “Incentives for Innovation, Research, Expedited Regulatory Pathways and Faster Market Access”. During this session, Prof. Casali highlighted the peculiarities of rare cancers as compared to rare diseases (i.e., rare non-neoplastic diseases). A point he raised was that despite the fact that some treatments for common cancers would be also effective for some rare cancers, and would be thus an opportunity for some rare cancer patients, they may not be developed in the rare, or ultra-rare, indications due to the perceived high uncertainty on the outcome of the regulatory and reimbursement processes. Thus, any effort to improve regulatory certainty about the rules for such rare and ultra-rare indications would be welcome. This also raises the issue of possible lack of awareness by regulators and reimbursement bodies about specific rare and ultra-rare cancers.
To help address the regulatory issues, Prof. Casali pointed out that the joint clinical assessment, under the Regulation on Health Technology Assessment, should be a useful tool to harmonize access to drugs for rare cancers and reduce delays in time to their availability throughout the EU. Furthermore, Prof. Casali shed the light on the role that European Reference Networks (ERNs), in collaboration with the upcoming European Health Data Space (EHDS), could play in testing innovative tools to facilitate regulatory processes. For example, innovative tools like “adaptive licensing” could be implemented in these networks to support the generation of new evidence through ought the early lifecycle of a medicine in a highly flexible fashion. However, the success of ERNs, as emphasised by Prof. Casali, heavily depends on their integration with national healthcare systems by Member States. Indeed, he explained during his presentation that effective national rare cancer networks would facilitate the functioning of EU-level research and data analysis.
Regarding incentives, Irena Storová from the Czech Institute for Drug Control stated that the extension of the IP protection period, which is currently being discussed in the context of the revision of the OMP and Paediatric Regulations, can only be considered as long as it is well balanced and comes with an obligation to launch medicines in all EU Member States. She highlighted the need to better regulate the entry in the market of generics or biosimilars as well as the conditions under which a drug is transferred from the research organization to the commercial entity. To complement this point, Prof. Casali, stressed that collaboration is needed amongst all stakeholders. He mentioned, for example, that a kind of scientific advice provided by regulatory and reimbursement bodies to disease-based communities on criteria to develop new agents on single rare ane ultra-rare cancers would be exceedingly useful. This because the disease-based communities could bring their expertise to regulators and third payers as a means to improve their decision-making process, valuing the peculiarities of single rare and ultra-rare cancers.