The EHC welcomed the opportunity to contribute to the European Commission’s public consultation on the draft implementing regulation on Joint Scientific Consultations (JSC) for Medicinal Products, for which we issued nine recommendations.

This EU initiative sets out implementing rules detailing how developers can receive early guidance on the evidence and data needed to carry out a joint clinical assessment of their medicinal products.

Read more about this EU Commission’s initiative here.

JCA should not hamper access to transformative ATMPs!

The EHC joins a group of patients, clinicians, academic medical centres, and therapeutic developers and manufacturers in signing a call to action for the successful roll-out of the Joint Clinical Assessment (JCA).

With the adoption of the Implementing Act on the JCA for medicinal products and the HTA Coordination Group’s Methodological and Practical Guidelines on direct and indirect comparison, the implementation of the JCA under the EU HTA Regulation is entering a critical phase. The EHC and other signatories are concerned that European patients with few or no treatment alternatives will experience further delays and limitations in access to potentially life-changing medicines. This would defeat the purpose of the EU HTA and JCA which aspire to increase and accelerate patient access.

Therefore, the joint statement urges the members of the HTA Coordination Group and its relevant subgroups, and JCA assessors to recognise all types of available evidence including single-arm trials and RWE, and to use the JCA report to describe, rather than judge, any resulting uncertainty as to the treatments’ benefits, as called for by the HTA Regulation. A significant portion of outstanding uncertainty can be addressed at the national level during the appraisal phase and through the collection of RWE.

In so doing, the Coordination Group can lead the development of a fit-for-purpose JCA system that efficiently addresses the needs of healthcare systems, without obstructing patient access to transformative therapies.

The EHC joins other leading patient organisations across Europe in releasing a set of ten key recommendations aimed at improving patient involvement in Joint Clinical Assessments (JCAs) under the EU Health Technology Assessment (HTA) Regulation. This collaborative effort highlights the crucial role of patient experiences and insights in shaping effective healthcare assessments and ensuring that the voices of those directly affected by medical innovations are heard and valued. 

Developed with input from a wide range of patient groups and based on extensive consultation and practical experience, the recommendations are designed to foster a more inclusive, transparent, and patient-centered approach to JCAs. The following ten points summarise their input and we highly encourage they are considered to ensure meaningful and practical patient involvement

They’re also a call to action for the HTA Coordination Group to integrate these practices into the guidelines they develop to enhance the quality and relevance of JCAs. By doing so, they can ensure that assessments reflect the real-world experiences of patients, ultimately leading to more patient-centered healthcare solutions.

• Establish a predictable framework for patient involvement in JCAs
• Include input from patients, carers and patient organisations
• Include patient experience data in JCAs
• Streamline patient involvement throughout the process
• Provide plain language summaries of technologies
• Broaden the pool of patients and specify selection criteria
• Provide support to patients
• Make JCA and summary reports available in all EU languages
• Provide feedback to patients
• Adopt a constructive approach to confidentiality and conflict of interest

For the last thirty years, haemophilia A and B patients have been treated using FVIII and FIX replacement concentrates. These therapies are well known to both clinicians and patients. We are now entering a new era in the treatment of haemophilia and rare bleeding disorders. Novel, non-replacement therapies have been approved and are becoming available, which may have a significant impact on the clinical care and quality of life of patients.

The European Medicines Agency (EMA) launched in October 2023 a public consultation on their draft guideline on the clinical requirements for non-replacement therapy in haemophilia A and B. The end of the consultation and deadline for comments was on 30 April 2024.

The European Association for Haemophilia and Allied Disorders (EAHAD), the European Hematology Association (EHA), and the EHC support EMA’s efforts in drafting the guideline which aims to provide both applicants and regulators with much-needed harmonised marketing authorisation requirements for applications of non-replacement therapies for haemophilia A and B.