Zevra Therapeutics’ Post

The impact of EU policy and advocacy on progress with treatments for rare disease https://lnkd.in/edEm4Yea Despite the increase in awareness, drug development, and regulatory improvement, the rare disease market continues to face issues such as patient access and affordability. This update explains how EU policy and advocacy play a key role in overcoming these challenges. https://lnkd.in/edEm4Yea #MarketAccess #HEOR #RareDiseaseDay #WeAreAvalereHealth

We are standing together with the European Hematology Association (EHA) and Members of the EHA Patient Advocacy Committee to advocate for the protection of the #HospitalExemption in the ongoing EU #PharmaceuticalLegislation update. Through collaborative efforts, backed by numerous #patientgroups, including the Thalassaemia International Federation (TIF) and supporters of medicine accessibility, we stress the crucial role of hospital exemption in allowing #patientaccess to lifesaving Advanced Therapy Medicinal Products (#ATMPs). #TIF is actively contributing to this movement for preserving and enhancing the Hospital Exemption as part of this critical reform, as therapies should be available for every patient that needs them. This rule is more than legislation; it's a lifeline for patients with #rarediseases or those without effective #treatment options, granting them hope and possibility through innovative therapies across #EU Member States. Read the Statement at: https://lnkd.in/dyD8YaHt

As a CLL/SLL patient advocate I am excited the FDA has approved a new medicine, Japirca (pirtobrutinib), from Eli Lilly and Company. It gives more confidence to patients that there are more options for them to live a longer, fuller life. Congrats to the people who have worked hard to bring us this new option. See you at #ash23 . My brief video explains where this new med fits in. #cll #cancerresearch #cancerpatients

As a CLL/SLL patient advocate I am excited the FDA has approved a new medicine, Japirca (pirtobrutinib), from Eli Lilly and Company. It gives more confidence to patients that there are more options for them to live a longer, fuller life. Congrats to the people who have worked hard to bring us this new option. See you at #ash23 . My brief video explains where this new med fits in. #cll #cancerresearch #cancerpatients

Thoughts on this? >> Rare pediatric PRV reauthorization faces uncertain fate as summer recess approaches >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #productmarketing #pharma #biotech #pharmaceutical #healthcare

Massachusetts lawmakers have moved forward a proposal to legalize medical, therapeutic, and spiritual use of psilocybin, with a focus on supervised sessions and an inclusive licensing framework for facilitators. https://lnkd.in/g2Mj4UrV #psychedelics #psilocybin #psychedelicsattorney

🌟 Don't let patient feedback become a last-minute hurdle! Join Tanja Hoffman, Consultant at TNJ Life Sciences Consultancy for her session "The Devil is in the Details: Embracing Patient Engagement in Drug Development." Honest question: Are you worried that involving patients in your clinical protocol decision making may render your clinical trial infeasible with demanding ideas and comments? 🤔 It doesn’t have to be that way! • Discover how embracing the opportunity to engage patients from the start can reduce patient burden and improve trial outcomes through a compelling case study in paediatric rare disease investigation. • Understand how asking the right questions, truly listening to patients and keeping an open mind can lead to smoother, more efficient trials. • Learn practical strategies to integrate patient feedback without jeopardizing your timelines and budgets. 👩⚕️👨🔬 Join us on 10-11 September 2024 in Amsterdam or virtually and turn potential obstacles into powerful opportunities. Let's pave the way for more effective and patient-centred drug development together. Download the full agenda to learn more! 📅 Download agenda: https://bit.ly/3X36fY5 👉 Register today: https://bit.ly/3Kwp6Dr #PatientEngagement #ClinicalTrials #DrugDevelopment #PatientCentered #MedicalResearch #Pharma #PatientInvolvement #ClinicalResearch

Thoughts on this? >> Rare pediatric PRV reauthorization faces uncertain fate as summer recess approaches >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #productmarketing #healthcare #pharma #pharmaceutical #biotech

Today marks World MS Day, a global initiative established to raise awareness about Multiple Sclerosis and support those affected by it. 🌍Originating in 2009, the day was created by the MS International Federation to unite individuals, organizations, and communities worldwide in the fight against this neurological condition. 💡In the realm of medical research, cannabis has emerged as a potential ally in alleviating symptoms associated with Multiple Sclerosis. From managing pain to reducing muscle stiffness and spasticity, cannabis-based medicines show promise in enhancing the quality of life for MS patients. 🌿As we delve into cannabis-based treatments, Sativex emerges as a notable contender. Legal in various countries across Europe, Canada, and elsewhere, Sativex offers relief for MS symptoms such as spasticity. However, its availability remains limited due to legal restrictions, notably in the United States, hindering access for many in need. 🔬Despite advancements, more comprehensive research is essential to unlock the full potential of cannabis medicines in treating MS. Advocating for a shift in regulatory frameworks, particularly the reclassification of cannabis to Schedule III, holds promise in fostering widespread development and accessibility of such treatments globally. #worldMSday #cannabiseducation #cannabispolicy #cannabislaw #cannabisregulations #cannabismedicine #cannabisismedicine