Critical Path Institute (C-Path)’s Post

Hello from Amsterdam! 👋 At the #ERICA and #EJPRD Joint Conference, Magda Chlebus (EFPIA - European Federation of Pharmaceutical Industries and Associations) emphasized that "The industry is very much looking to work with ERNs! It is not always easy, there are many real and perceived barriers, but only by engaging together, can we develop public-private partnerships to improve patient outcomes". 👉 Read Together4RD's Position Statement in the Orphanet Journal for Rare Diseases here, to learn more about the barriers to ERN-industry collaboration and how to overcome them: https://lnkd.in/epgN5UZr

Explore our case study and learn how we helped a leading global pharmaceutical company successfully conduct two paediatric studies in Rare Endocrine-Metabolic Diseases.  With a track record of 90+ Rare Disease clinical 𝐭𝐫𝐢𝐚𝐥𝐬 𝐢𝐧 𝐩𝐡𝐚𝐬𝐞𝐬 𝐈-𝐈𝐕 in over over 45 indications and 99 𝐡𝐢𝐠𝐡𝐥𝐲 𝐞𝐝𝐮𝐜𝐚𝐭𝐞𝐝 𝐭𝐞𝐚𝐦 𝐦𝐞𝐦𝐛𝐞𝐫𝐬 with rare disease 𝐞𝐱𝐩𝐞𝐫𝐢𝐞𝐧𝐜𝐞, #teamOptimapharm is helping you expedite the development of new medicines, therapies and treatments that advance health globally. Reach out to us at contact@optimapharm.eu, and let's discuss how we can work together to overcome the challenges in orphan drug development and ensure patients have access to life-changing treatments.  #clinicaltrials #clinicalresearch #contractresearchorganization #CRO #raredisease #orphandrugs

Choosing predictive and clinically relevant endpoints is a key aspect of drug development in the regulatory space. By assessing orphan drug development for sickle cell disease (SCD) over the past two decades, we observed that failures in clinical development were mainly found in phase 3 due to lack of efficacy on vaso-occlusive crises as the primary study endpoint. This was likely related to variable definitions and heterogeneity of pain scoring and treatment. If you are interested in improving the debate on how fostering pharmaceutical R&D on SCD (and other rare diseases), you may wish to read our last work on this. https://lnkd.in/difgpBep #sicklecell #orphandrugs

In case you missed it, we released a special podcast episode in honor of Rare Disease Day. Angela Wheeler, President of Insight US at Lumanity and Dr. Karin Hoelzer, Director of Policy and Regulatory Affairs at the National Organization for Rare Disorders (NORD), discussed opportunities for pharmaceutical companies to work with and support rare disease communities. Discussion highlights included the importance of: - continued support for the development of rare disease treatments - making a push towards more equitable clinical trial populations - focusing on how we can reduce the barriers to access - bringing in the patient perspective and patient voice in every step of the drug development process Listen to the episode at #rarediseases #raredisorders #patientengagement #patientadvocacy #RareDiseaseDay #WeAreRare

[🔔 📣 The Cancer Drug Development Forum (CDDF) Engages at the Accelerating Global Health Equity Annual Summit]   Dr. Catarina Edfjäll, representing the CDDF, participated in a panel discussion on EU Pharmaceutical Legislation at the Accelerating Global Health Equity Annual Summit, held as a World Economic Forum (WEF) side event in Davos on January 18, 2024.   Dr. Edfjäll joined the panel of experts to share diverse perspectives on the revision of the EU Pharmaceutical Legislation. The CDDF's key takeaways from the summit are summarized as follows:   🔴 CDDF welcomes several of the reforms in the pharmaceutical legislation discussed at the Accelerating Global Health Equity (AGHE) Annual Summit.   🔴 CDDF is aligned with the presented view that increasing #patient input to clinical research and development as well as decision making (e.g. by introducing CHMP patient representatives) is a step in the right direction. The importance of increasing the #PatientVoice in the EU was also recently highlighted in CDDF's white paper on Empowering Involvement and Engagement of Patients with Cancer in Oncology Drug Development (link to the paper: https://lnkd.in/ezeY_9U2)    🔴 In general, CDDF supports measures that will streamline regulatory procedures, reduce complexity and speed up #DrugDevelopment and thus ultimately improve #PatientAccess in the EU. Harmonising systems to ensure timely and equitable access for patients across all EU member states is equally important. 💡 Special thanks to Partners for Patients for organizing the great panel discussion. #oncology #patientcentric #EUPharmaLegistration #worldeconomicforum

On the heels of the end of the European Parliament’s legislative mandate with the last plenary session held last month, the Children’s Tumor Foundation Europe reflects on the progress made in the EU's regulatory regime for medicines. CEO Annette Bakker applauds the ambition on market exclusivity for orphan drugs. A historic opportunity to advance treatments for patients worldwide. Read the full statement at the link below. #EndNF #EUpharma #raredisease https://lnkd.in/edMUyCXU

In case you missed it, we released a special podcast episode in honor of Rare Disease Day. Angela Wheeler, President of Insight US at Lumanity and Dr. Karin Hoelzer, Director of Policy and Regulatory Affairs at the National Organization for Rare Disorders (NORD), discussed opportunities for pharmaceutical companies to work with and support rare disease communities. Discussion highlights included the importance of: - continued support for the development of rare disease treatments - making a push towards more equitable clinical trial populations - focusing on how we can reduce the barriers to access - bringing in the patient perspective and patient voice in every step of the drug development process Listen to the episode at #rarediseases #raredisorders #patientengagement #patientadvocacy #RareDiseaseDay #WeAreRare

#RareDiseaseDay is around the corner! Celebrated each year, it’s a globally coordinated movement that works towards equity in social opportunity, #healthcare, and access to diagnosis and therapies for people living with a #raredisease. In 2022, over half of the novel drugs approved by the FDA were orphan drugs. However, experts say that regulatory incentives for drug reimbursement and support for innovative trial designs is needed for this significant progress to continue. As we stand on the precipice of Rare Disease Day, we acknowledge that advancements in #raredisease clinical research demand innovative approaches and creative trial designs. By fostering collaboration, we can continue to accelerate the development of treatments for patients with rare diseases. Learn more about what is on the horizon for rare diseases and orphan drug trials in this Pharmaceutical-Technology article, sharing insights from the Clinical Trials in Rare Diseases conference: https://lnkd.in/eGwCdAR9

Today, 29 February 2024, is Rare Disease Day. Avance Clinical is proud to have completed over 60 clinical trials for rare diseases in the past 5 years. In late April, the North American team will attend the World Orphan Drug Congress USA in Boston. The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. The conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies. Book a meeting with our team of clinical trial experts 👇 https://lnkd.in/eQ5s_fw #AvanceClinical #CRO #clinicaltrials #drugdevelopment #raredisease #worldorphandrugcongress #Boston #USA #Australia #RareDiseaseDay #awareness #biotech #clinicalresearch

If you are attending #DIAglobal, be sure to check out this panel!