EMA’s Head of the Stakeholders…, A new Head of the Veterinary…, Ivo Claassen joined EMA in 2018. Among many career highlights, Ivo and his team worked with the veterinary medicines network on the successful implementation of the 2019…

Original title: “New leadership team appointments”

EMA’s Head of the Stakeholders…, A new Head of the Veterinary…, Ivo Claassen joined EMA in 2018. Among many career highlights, Ivo and his team worked with the veterinary medicines network on the successful implementation of the 2019…

Original title: “EMA appoints new Deputy Executive Director and new Head of Veterinary Medicines”

Then $75 per month. Complete digital access to quality FT journalism on any device. Cancel anytime during your trial · Essential digital access to quality FT journalism on any device. Pay a year upfront and save 20% Then $75 per month. Complete digital access to quality FT journalism on any device. Cancel anytime during your trial. From Sam Barrell, CEO of LifeArc, London WC1H, UK

Original title: “Letter: UK can lead way on rare disease treatments”

The clinical trials industry has never had more tools, data, or innovation aimed at accelerating timelines. In fact, if you walk the floor of any industry event The clinical trials industry has never had more tools, data, or innovation aimed at accelerating timelines. In fact, if you walk the floor of any industry event you’ll see “acceleration” everywhere. It’s in the messaging, the booths, the product demos, and the promises. But in 2003, when my two babies were diagnosed with a rare disease, I was forced to navigate a system that dismissed my concerns while my children suffered. That experience changed everything. I learned the true power of patient voices — how advocacy, storytelling, and compassion can transform healthcare. It also shaped my purpose: to ensure sponsors, CROs, and sites embrace patient-centricity in every clinical trial. In fact, better alignment with patient reality often strengthens both, by improving adherence, data quality, and overall trial execution. This complexity extends beyond patients to caregivers, who are often essential to participation, particularly in rare disease, oncology, and chronic conditions. Caregivers coordinate schedules, manage logistics, and provide emotional and physical support, yet their burden is rarely accounted for in trial design. This omission matters, especially when more than 40% of patients cite logistical challenges such as travel, time commitment, and coordination as key barriers to participation (CISCRP). Incorporating caregiver perspectives through surveys, advisory panels, and qualitative research can surface constraints that would otherwise go unseen and unaddressed. When endpoints miss the patient Clinical endpoints are designed to satisfy regulatory and scientific requirements, but they do not always reflect outcomes that patients value, such as quality of life or day-to-day symptom relief.

Original title: “If Everyone Claims to Accelerate Clinical Trials, Why Are We Still Falling Behind?”

Where the future of workplace wellbeing takes centre stage

Original title: “MAD World Summit 2026”

Clinical trial access remains the biggest barrier in sarcoma care, with travel and insurance limits keeping patients from open studies, says Vicki Keedy, MD. sarcoma, where many subtypes fail to respond to standard therapies and oncologists often reach for investigational options early, according to Vicki Keedy, MD, MSCI, clinical director of the Sarcoma Program at · Vanderbilt-Ingram Cancer Center. The greatest obstacle, Keedy explains, is simply reaching a trial. Because sarcoma is rare, research funding is limited, and the sites running any given study are often scattered across the country. Keedy notes that insurance network rules compound the problem, as some Medicaid programs will not cover out-of-state travel, and patients without a sarcoma center in their state can struggle to access expert care at all. Expanding the number of trial sites and enlisting foundations to offset travel costs, she argues, would meaningfully widen access. She also pushes back on a persistent assumption that rare disease trials cannot enroll. With a growing menu of therapies and no head-to-head trial data, treatment becomes highly individualized.

Original title: “Expanding Clinical Trial Access and Individualizing Care: Vicki Keedy, MD | AJMC”

The success of these local meetings ... of rare disease advocacy is hybrid. We don’t need to choose between local intimacy and global reach. In fact, they complement each other beautifully. Local meetings can serve as incubators for ideas that are later shared at larger European forums. A patient who finds ... The success of these local meetings suggests that the future of rare disease advocacy is hybrid. We don’t need to choose between local intimacy and global reach. In fact, they complement each other beautifully. Local meetings can serve as incubators for ideas that are later shared at larger European forums. A patient who finds their voice at a local gathering might feel confident enough to present their story at a national conference. You sign up for a massive virtual conference, log in from your kitchen table, and spend the next three days clicking through breakout rooms while half-heartedly eating lunch. It’s convenient, sure, but let’s be honest: it lacks soul. That is exactly why the recent wave of local meetings for European rare disease patients has been such a breath of fresh air. If a doctor in your local clinic recommends a specific type of physical therapy, the person next to you probably knows the best center to go to. They might know which hospital administrator is the most patient or which pharmacy stocks the expensive medication on discount days. This hyper-local knowledge is gold for rare disease patients who often feel lost in the system. You get perspectives from rural areas as well as cities, from full-time workers and students alike. This diversity enriches the discussions and ensures that the community represents the real spectrum of the rare disease population. It proves that advocacy doesn’t have to be a luxury experience.

Original title: “popular rare 2011 rare disease conference reviews regional orphan disease conference directory - orphan disease conference”

Ardena has appointed Ariane de Ganck as Chief Scientific Officer

Original title: “Ariane de Ganck”

Emily Curran, MD, University of Cincinnati, calls for decentralized trials and stronger community-academic ties to widen ALL patient access. Although trials are essential for advancing new treatments, Curran noted that strict eligibility criteria, put in place for safety, often exclude patients with impaired kidney function, even when a treatment is not renally cleared or in prior lines of therapy. In practice, this leaves clinicians making treatment decisions for real-world patients without the benefit of trial data to guide them. Practical nuances, such as when to hold or dose-reduce a drug, rarely make it into published manuscripts. Compounding this, the pace of change in the field means that by the time findings are published, treatment approaches may have already evolved, leaving published literature outdated almost as soon as it appears. Looking ahead, Curran pointed to decentralized clinical trials as a promising solution, allowing patients to receive care locally through their own oncologists, supplemented by virtual or occasional in-person visits rather than requiring travel to a central site. Patients who enroll tend to be those willing and able to participate, and mistrust of clinical research means some communities remain · underrepresented. Geography compounds the problem: patients living hours from a trial site may be unable to commit to visits spanning multiple consecutive days, even when institutions offer support such as discounted hotel stays. Part 1: What Now Predicts Outcomes in Older Adults With ALL: Emily K. Curran, MD · Part 2: Rethinking ALL Treatment for Older Adults: Emily K. rare disease cases, helping extend expert-guided care to patients regardless of where they live.

Original title: “Clinical Trials Leave Many ALL Patients Behind: Emily K. Curran, MD | AJMC”

spinal muscular atrophy all babies in england to be screened for rare neuromuscular disease

Original title: “Spinal muscular atrophy: all babies in England to be screened for rare neuromuscular disease.”

B. Braun has appointed Sophie Spencer as Social Value & ESG Specialist

Original title: “Sophie Spencer”

Get the full story behind the latest Rare Barometer survey findings and their implications The post From Evidence to Action: Closing the Mental Health Gap in Rare Disease Care appeared first on EURORDIS-Rare Diseases Europe.

Original title: “From Evidence to Action: Closing the Mental Health Gap in Rare Disease Care”

Brussels, 14 July 2026Check against deliveryDear Ms Jerkovic, dear honourable members, thank you for the invitation today and for this opportunity to report on EMA’s…

Original title: “Executive Director Emer Cooke's speech at the European Parliament's Committee on Public Health”