Edition of 03 June 2026

The EMA has announced a new pilot programme to support the development of breakthrough medical devices in the EU. This pilot aims to test a new regulatory pathway, which would support patient access to highly innovative technologies while also respecting safety and performance standards.

This pilot provides enhanced regulatory support to manufacturers whose devices are granted ‘breakthrough’ status, including priority scientific advice from EMA’s medical device expert panels. The programme also allows notified bodies to submit specific questions to the expert panels in the context of the conformity assessment and clinical evaluation.

Currently, the pilot is open to class III (high risk) medical devices and class IIb active medical devices intended to administer or remove medicines from the body. Subsequent phases will be open to other types of devices, including in vitro diagnostics. Instructions on how to apply to participate in the pilot can be found on the programme’s webpage.

This pilot builds on the Guidance on Breakthrough Devices published by the European Commission’s Medical Device Coordination Group (MDCG) in December 2025. It represents the first step towards formally implementing a framework for breakthrough medical devices, which is included in the proposed revisions of the Medical Devices Regulation and In Vitro Devices Regulation.

ERDERA has published initial findings from their survey on the rare disease community’s needs when it comes to external consultancy support. This survey aims to help shape an on-demand ERDERA Consultancy Service for the wider rare disease research ecosystem, including stakeholders beyond ERDERA itself.

Of the responses received so far, 84% of respondents have indicated interest in such a service, with 63% saying they would be willing to pay if the conditions are reasonable.

In order to further strengthen the analysis of the survey’s results, ERDERA is inviting further responses, particularly from underrepresented countries and stakeholder groups. If you have not yet completed the survey, we invite you to do so here.

The Second Joint Action Towards the European Health Data Space (TEHDAS2) is seeking input from stakeholders on seven draft guidance documents, aimed at supporting the implementation of the European Health Data Space (EHDS) for secondary use.

The draft documents currently open for consultation are:

• Draft guideline on a framework for collaboration
• Draft guideline for health data access bodies on international and third country access and transfer of electronic health data
• Draft guideline for data enrichment
• Draft guideline for data users navigating the catalogue
• Draft guideline on the linkage of health datasets
• Draft guideline for health data access bodies on informing natural persons about the use of health data – “Citizen Information Point”
• Draft guideline for data users on handling research outcomes

Together, these drafts address a range of governance, operational and transparency-related questions relevant to the secondary use of health data under the EHDS. The consultation is aimed at all stakeholders, including public authorities, data users, and data holders, and will remain open until 28 June 2026. In order to participate, interested stakeholders can submit feedback through the dedicated consultation surveys available on TEHDAS2’s dedicated public consultations page.

In March 2026, the FDA accorded market authorisation to the following medicinal products with an orphan designation:

• Trikafta, for the treatment of cystic fibrosis (CF) in adult and pediatric patients aged 2 years and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein
• Lynavoy, for the treatment of cholestatic pruritus associated with primary biliary cholangitis (PBC) in adult patients
• Kresladi, for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant
• Opdivo, in combination with doxorubicin, vinblastine, and dacarbazine (AVD), for the treatment of adult and pediatric patients 12 years and older with previously untreated, Stage III or IV classical Hodgkin lymphoma (cHL)
• Tecvayli, for the treatment of adult patients with relapsed or refractory multiple myeloma in combination with daratumumab hyaluronidase-fihj in patients who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent
• Avlayah, for the treatment of neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II, MPS II) when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment
• Alyftrek, for the treatment of cystic fibrosis (CF) in adult and pediatric patients 6 years of age and older who have a clinical diagnosis of CF and who have at least one variant in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein