Pharma Focus Europe’s Post

Planning to launch an orphan medicine in European markets? If you’re navigating market access for rare disease treatments, our 4-part series offers valuable insights into key roadblocks: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/eBGh5JRe 2️⃣ Clinical evidence development 👉https://lnkd.in/e8Cqm9F7 3️⃣ Economic evidence development 👉https://lnkd.in/ebpgcMdw 4️⃣ Operational challenges 👉 https://lnkd.in/eVuajumu Planning to launch an orphan medicine in Europe in 2025? Our experts can offer the valuable insights you need. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

Planning to launch an orphan medicine in European markets? If you’re navigating market access for rare disease treatments, our 4-part series offers valuable insights into key roadblocks: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/ermYQZFR 2️⃣ Clinical evidence development 👉https://lnkd.in/e7xzFAqs 3️⃣ Economic evidence development 👉https://lnkd.in/eSF9fHnQ 4️⃣ Operational challenges 👉 https://lnkd.in/ex_2zdWq Planning to launch an orphan medicine in Europe in 2025? Our experts can offer the valuable insights you need. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

!!!New publication!!! We aimed to describe potential prognostic factors for adverse events in patients prescribed colchicine when initiating allopurinol using a retrospective cohort study design in linked UK Clinical Practice Research Datalink and Hospital Episode Statistics datasets. From 01/04/1997–30/11/2016, 13 945 people with gout initiated allopurinol with colchicine prophylaxis (mean age 63.9 (SD 14.7) years, 78.2% male). One quarter (26%, 95% CI 25% to 27%) were prescribed ≥1 potentially interacting medicines, most commonly statins (21%, 95% CI 20% to 22%). Statins were not associated with increased adverse events, although other drugs were associated with some adverse outcomes. Diarrhoea and MI were associated with more comorbidities and more severe CKD. #gout #colchicineprophylaxis #adverseevents #urateloweringtherapy #prognosticfactors #KeeleMSK https://lnkd.in/eRtqMUvE

Planning to launch an orphan medicine in European markets? If you’re navigating market access for rare disease treatments, our 4-part series offers valuable insights into key roadblocks: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/epjwK5jP 2️⃣ Clinical evidence development 👉https://lnkd.in/enWRHfPX 3️⃣ Economic evidence development 👉https://lnkd.in/e5WN8uCs 4️⃣ Operational challenges 👉 https://lnkd.in/eV4UgKVy Planning to launch an orphan medicine in Europe in 2025? Our experts can offer the valuable insights you need. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

Planning to launch an orphan medicine in European markets? If you’re navigating market access for rare disease treatments, our 4-part series offers valuable insights into key roadblocks: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/eQBDJKqg 2️⃣ Clinical evidence development 👉https://lnkd.in/eUT2qS9r 3️⃣ Economic evidence development 👉https://lnkd.in/eCCrHRkr 4️⃣ Operational challenges 👉 https://lnkd.in/e_ZJfkhz Planning to launch an orphan medicine in Europe in 2025? Our experts can offer the valuable insights you need. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

Planning to launch an orphan medicine in European markets? If you’re navigating market access for rare disease treatments, our 4-part series offers valuable insights into key roadblocks: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/eSSwk667 2️⃣ Clinical evidence development 👉https://lnkd.in/e6CQ_pRB 3️⃣ Economic evidence development 👉https://lnkd.in/eTAdY8mj 4️⃣ Operational challenges 👉 https://lnkd.in/e7QxFaGu Planning to launch an orphan medicine in Europe in 2025? Our experts can offer the valuable insights you need. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

Planning to launch an orphan medicine in European markets? If you’re navigating market access for rare disease treatments, our 4-part series offers valuable insights into key roadblocks: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/e548YAe5 2️⃣ Clinical evidence development 👉https://lnkd.in/ev2DedrG 3️⃣ Economic evidence development 👉https://lnkd.in/e88CNGEj 4️⃣ Operational challenges 👉 https://lnkd.in/eqS5nEjV Planning to launch an orphan medicine in Europe in 2025? Our experts can offer the valuable insights you need. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

As someone who I personally know, is affected by muscular dystrophy—a rare and debilitating disease—I want to share my thoughts on the recent discussions surrounding the FDA’s Rare Disease Innovation Hub and the call for more collaboration and regulatory flexibility. The FDA’s new initiative is a step in the right direction for accelerating treatments for rare diseases. But as many stakeholders have pointed out, we need greater collaboration both within the agency and with external stakeholders. This includes stronger ties with federal agencies like CMS (Medicare & Medicaid Services), SSA (Social Security Administration), and NIH (National Institutes of Health), as well as better engagement with payors who often hold the keys to treatment accessibility. For rare diseases like muscular dystrophy, small clinical trials and alternative study designs are often the only way to gather meaningful data. Unfortunately, FDA reviews can sometimes lack consistency and flexibility, which leads to delays and uncertainty in getting treatments to market. Given the progressive nature of muscular dystrophy, these delays are not just inconveniences—they’re life-altering. Every moment matters. Regulatory flexibility is crucial, but regulatory nimbleness—defined as the ability to adapt without compromising the integrity of the process—may be even more important. There needs to be clearer guidelines that allow for the rapid approval of therapies that can make a meaningful impact on rare disease communities. We can’t afford to wait years for treatments that could change lives. Patients, like my father and his sister, who are battling rare diseases need hope—and they need it now. Let’s advocate for a system that allows for faster, more efficient approval of rare disease therapies, and ensures that collaboration, both inside and outside the FDA, becomes the norm rather than the exception. #RareDiseases #MuscularDystrophy #FDA #RegulatoryFlexibility #Collaboration #InnovationHub #RareDiseaseCommunity #HealthPolicy

Planning to launch an orphan medicine in European markets? If you’re navigating market access for rare disease treatments, our 4-part series offers valuable insights into key roadblocks: 1️⃣ Disease knowledge and awareness 👉https://lnkd.in/eMwjhMfQ 2️⃣ Clinical evidence development 👉https://lnkd.in/en3yfcNs 3️⃣ Economic evidence development 👉https://lnkd.in/eaztfaf8 4️⃣ Operational challenges 👉 https://lnkd.in/eMB4mXKq Planning to launch an orphan medicine in Europe in 2025? Our experts can offer the valuable insights you need. Get in touch at info@mtechaccess.co.uk #raredisease #orphanmedicine #marketaccess #pharma #orphandrug

Testing the effectiveness of a drug on a disease can be quite challenging, especially when the disease's natural history involves many patients improving on their own. For example, in conditions like COVID where the majority of people recover without intervention, it's difficult to determine whether a patient's improvement is due to the medication or just the natural course of the illness. On the other hand, with a disease like Castleman, where no one improves without treatment, it's much easier to attribute a patient's recovery directly to the drug. This contrast highlights the complexities of clinical trials—where sometimes, a drug's true impact might be masked by the body's natural healing process. Determining whether a medication genuinely works requires careful analysis and often, comparing results to placebos, which can sometimes show a surprisingly high rate of recovery on their own. At Every Cure, we aim to navigate these complexities by employing innovative approaches to drug repurposing and clinical trials. Our goal is to identify existing medications that might be effective against diseases with unclear treatment paths. By leveraging artificial intelligence and comprehensive data analysis, we can better predict which drugs have the highest potential to make a difference. This allows us to prioritize clinical trials for drugs that could offer real benefits to patients, even in cases where the disease's natural history makes it challenging to assess a drug's effectiveness. Ultimately, we strive to bring life-saving treatments to patients faster, ensuring no opportunity for healing is overlooked. #DrugRepurposing #EveryCure #RareDisease #Pharma #HealthcareInnovation