Looking back at the conference "What should the new regulation for orphan, and paediatric medicines look like?"
On Friday, 21 October 2022, the Association of Innovative Pharmaceutical Industry (AIFP), under the auspices of the Czech EU Presidency, organized an expert international conference focusing on the issue of orphan and paediatric medicines. The event is a follow-up to the international conference held in May 2022, which was organized in cooperation with the Health Committee of the Senate of the Czech Parliament under the auspices of MUDr. Roman Kraus, MBA.
The conference was attended by over 80 participants, mainly delegates from the Permanent Representations of the countries to the European Union, European institutions, Czech state administration, health service providers, patients, the medical community, and the innovative pharmaceutical industry. "The aim of the conference was to provide a comprehensive perspective on the issues of orphan and paediatric medicines in the preparation of new medical legislation, including the vision of the Czech Presidency and the commitments of the innovative pharmaceutical industry," says Mgr. David Kolář, Executive Director of AIFP.
A major achievement in the field of orphan drugs was the adoption of the European Orphan Medicines Regulation in 2000. Since then, the European Medicines Agency has approved more than 160 new orphan drugs, an 88% increase from the previous years. The new legislation should build on these achievements, supporting the development of modern medicines and their availability to patients in the EU.
Speakers at the meeting included:
- Lucie Šestáková (Permanent Representation of the Czech Republic to the European Union)
- Yann Le Cam (EURORDIS)
- Milan Macek (ERNs, FN Motol)
- Nathalie Moll (EFPIA)
- Jakub Dvořáček (Ministry of Health of the Czech Republic)
- Karen Facey (University of Edinburgh)
- Victoria Hedley (Newcastle University)
- Pavel Sedláček (AIFP)
- David Kolář (AIFP)
5 main conclusions
- An open discussion between all stakeholders - Member States, the European Commission, patient representatives, experts, and industry - is a prerequisite for legislation that will improve the situation of European patients.
- The discussion should lead to a compromise that does not reduce the attractiveness of the European Union for science and research. At the same time, the results of this research shall be available to patients in the EU.
- The role of patients cannot be overlooked. Their representatives should be involved in discussions and the decision-making processes leading to the reimbursement of new medicines.
- Accessible and high-quality health data is a prerequisite for further development of the healthcare sector.
- All data outside the scope of conventional clinical trials, including patient experience and preferences, should be used.
Early diagnosis and treatment in specialized centers
In order to use the full potential of orphan drugs, it is necessary that patients are diagnosed and treated in a timely manner in centers equipped with the personnel, knowledge, and facilities for the specific therapy emphasized Professor Macek during his presentation.
It is, therefore, appropriate to support the establishment and development of pan-European screening programs for the early diagnosis of rare diseases. The provision of comprehensive care for patients with rare diseases is then possible in the centers of the European Reference Networks (ERNs). ERNs link experts across Europe to improve patient care even in those EU countries where, due to the rarity of the disease, appropriate experts and state-of-the-art technical equipment may be lacking.
Data is the basis
Rare diseases have one limitation - due to the small number of patients, it is often difficult to obtain definitive data on the efficacy and safety of treatments at the time of registration of new drugs. Therefore, it is necessary to develop a system for obtaining valid data beyond registration clinical trials.
As Karen Facey pointed out, Real World Evidence (RWE) data can be a tool for understanding the benefits and risks of treatment. It should be a resource for the evaluation of medicines and their introduction into clinical practice. These data will be of greater value if they are collected from the highest number of patients. Here is another opportunity for European reference networks that are robust enough to assess patient outcomes in all EU countries, Victoria Hedley noted.
The introduction of the European Health Data Space (EHDS) should also help with the availability of quality data, as Jakub Dvořáček pointed out. Karen Facey then emphasized that in addition to data, patients' opinions and preferences are also important for assessing treatment outcomes. Involving patients in the evaluation of treatment, including the adoption of descriptions of their experiences (i.e., evaluating treatment qualitatively), would provide further valuable insights into the effectiveness of treatment.
The use of data from real clinical practice and patient assessments, including qualitative assessments, should become a routine part of the Health Technology Assessment (HTA). Patients should then be involved in the decision-making process for reimbursement of new medicines.
Science and research must also take place in the EU
During the conference, Yann Le Cam introduced specific proposals to make Europe more attractive for early innovation, in particular, to maintain a prevalence threshold so that no disease is left behind. Then, identifying and finding solutions to the unmet needs of patients with rare diseases through early structured dialogue between health partners, targeting tailored incentives for new drug development to this segment, or continuously coordinating new drug research in broader collaboration with experts, patients, and others.
To keep the EU attractive and, in the best case, to increase its attractiveness for research and development of new orphan drugs, it is essential to protect the intellectual property associated with the research and development of new medicines. Incentives in the form of intellectual property and data protection can, of course, be changed to fit the current times, David Kolář pointed out. However, their applicability must be considered, and the limits of European health legislation in relation to Member States must be respected. It should also be borne in mind that research and development of orphan drugs often mean discovering new avenues and options for the treatment of common diseases. Any obstacle to research and development that may appear in the forthcoming legislation on orphan drugs may have a negative impact on research and development for the treatment of common diseases.
Medicines available to patients
Support for R&D through incentives needs to be complemented by mechanisms to ensure that medicines are accessible to patients. This is a common concern of both healthcare organizers (e.g., healthcare payers, HTA agencies), policymakers and the pharmaceutical industry. Nathalie Moll reported that this objective can be achieved by the pharmaceutical industry's existing commitment to launch new orphan products within two years of marketing authorization in all the EU countries where this is possible. Furthermore, by carefully monitoring availability (and reasons for non-availability) in all EU Member States and by introducing a pricing and reimbursement system (including new payment-for-performance models, etc.), which respects the economic possibilities of each Member State.
Conclusion
During the conference, we have once again witnessed that solutions can be found in an open discussion between all stakeholders at the local and European levels. Pavel Sedláček concluded by pointing out that we need to talk together, listen to each other, respect different positions, and trust each other. Only by working together will we be able to prepare legislation that will significantly contribute to improving the situation of European patients with rare diseases.
Complete record of the conference available here.
