EU Commission issues four new fact sheets on transborder care for rare, low prevalence and complex diseases, June 8, 2026
Read more at this link on the EU Commission website
Sotarcept has been approved in England and Wales by the National Institute for Health and Care Excellence (NICE), May 14, 2026
Sotatercept has been approved for use in eligible patients in England and Wales. The decision by the National Institute for Health and Care Excellence (NICE), announced on 14th May, means the drug (brand name Winrevair®) can be prescribed alongside existing pulmonary arterial hypertension treatments for patients who meet specific clinical criteria. The drug was approved
The new US Food and Drug Administration (FDA)’s guidance paper on incorporating voluntary patient preference information over the total product cycle, and what It means for patient organisations, March 30, 2026
Jan Geissler, Founder and CEO of Patvocates, a think tank, consultancy and social enterprise on patient advocacy and patient engagement in research, has analysed the Food and Drug Administration (FDA)’s newly issued guidance paper titled “Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle Guidance for Industry, Food and Drug Administration Staff, and
Publication of a new European Medicines Agency (EMA) booklet on medicine safety monitoring
The European Medicines Agency (EMA) has published a new booklet on the safety monitoring of medicines in the European Union. It provides an overview of how the European Medicines Agency and European Union Member States work to ensure that medicines used across Europe remain safe throughout their lifecycle. The booklet is published on a new
US Food and Drug Administration approves YUTREPIA, a treprostinil inhalation powder for pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease
Yutrepia is a dry powder formulation of treprostinil, a prostacyclin analog. It is designed to enhance deep-lung delivery with an easy-to-use device requiring low inspiratory effort. Yutrepia was was tentatively approved by the Food and Drug Administration last year (see our article here), but the agency was waiting for regulatory exclusivity of a competing product
“A wake-up call: key trends and policy asks for cardiovascular health in women”, European Parliament event, March 5, 2025
We were unfortunately unable to attend the European Parliament event which took place on March 5, 2025, but have read the minutes with great interest. Based on these we have put together some key takeaways as this topic is very relevant to pulmonary arterial hypertension patients, who are predominantly women. Opening Member of the European
The US Food and Drug Administration (FDA) has accepted the new drug application resubmission for YUTREPIA™ (treprostinil) inhalation powder, March 28, 2025
Link: Liquidia Corporation announced on March 28, 2025, that the US Food and Drug Administration has accepted its resubmitted new drug application for YUTREPIA (treprostinil) inhalation powder. The treatment targets pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). The FDA had previously granted tentative approval for both indications (see our
Stronger Together: Celebrating the Pulmonary Hypertension Community’s 2024 Milestones
The year 2024 has seen significant progress in the fight against pulmonary hypertension, marked by critical milestones across research, clinical care, advocacy, awareness, and patient engagement, with an unprecedented level of patient involvement in scientific discourse. As we head into 2025 with all that has been achieved we are well positioned in furthering our mission!
Stronger Together: Celebrating the Pulmonary Hypertension Community’s 2024 Milestones Read Post »
Findings of European Patient Forum (EPF) survey on the implementation of EU legislation on medical devices now published
The EU Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR), introduced in 2017, aims to enhance patient safety, transparency, and information through stricter quality standards, performance evaluations, and oversight across the device lifecycle. But not all changes have been fully put into action, and many patients are still waiting to see benefits. To
The US Food and Drug Administration (FDA) issues guidance to medical product sponsors to ensure greater inclusivity in clinical trials, June 26, 2024
The U.S. Food and Drug Administration (FDA) has issued a draft guidance titled “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies.” This guidance is aimed at assisting medical product sponsors in submitting “Diversity Action Plans” for certain clinical studies. These plans are designed to enhance the enrollment of historically
Kick-off meeting of the EU Joint Action JARDIN on the integration of European Reference Networks (ERNs) into national health systems
The European Reference Networks (ERNs) are virtual networks involving healthcare providers across Europe. They aim to facilitate discussion on complex or rare diseases and conditions that require highly specialised treatment, and concentrated knowledge and resources. 24 ERNs are working on a range of thematic issues, including ERN-Lung, the European Reference Center for Rare Lung Diseases, which
The European Medicines Agency (EMA) has issued a call for patient representatives to join the Committee for Orphan Medicinal Products (COMP)
The European Commission has launched a call for expressions of interest to represent patients’ organisations in European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). This is a very important opportunity for patients to have a role in the approval of orphan medicinal products! The call aims to fill three positions nominated by the
Proposed revision of EU pharmaceutical laws, released on April 26, 2023, will contribute to making the EU regulatory framework more patient-centred
On Wednesday April 26, 2023, the European Commission adopted a proposal for a new Directive and a new Regulation, which revise and replace the existing general pharmaceutical legislation. The EU Commission’s proposed revisions include proposals to update the EU’s Regulations on Orphan Medicinal Products and Paediatric Medicines, which are very important to Europe’s population of
“The Added Value of Patient Engagement in Early Dialogue at the European Medicines Agency (EMA): Scientific Advice as a Case Study”, Frontiers in Medicine
ABSTRACT The European Medicines Agency provides Scientific Advice to medicines developers and patient input has been an integral part of this process for many years. As end users of medicines, patients bring their perspectives to many different processes along EMA’s regulatory pathway, complementing the scientific expertise. While the value of including patients has been
The EU Clinical Trials Regulation came into force 31st of January 2022
The European Union (EU) pharmaceutical legislation known as the Clinical Trials Regulation comes into force today, the 31st of January 2022. It aims to ensure the EU offers an attractive and favourable environment for carrying out clinical research on a large scale, with high standards of public transparency and safety for clinical trial participants. Prior to
The EU Clinical Trials Regulation came into force 31st of January 2022 Read Post »