The Michael J. Fox Foundation for Parkinson's Research’s Post

We are excited and looking forward to the upcoming Patients As Partners in Clinical Research Community News conference. Don't miss our presentation on March 20, Implementing a Patient-Focused Drug Development Framework for Enhanced Patient-Centric Strategies. The session, featuring Angela Wheeler, will share how to implement a Patient Focused drug development framework, integrating patient engagement into the clinical development process—a versatile approach applicable across diverse organizational systems. Key discussion points include: - Assisting biopharma companies in identifying their specific requirements and desires from patients at different stages - Understanding the untapped benefits of patient involvement, extending beyond protocol design to encompass crucial aspects like determining endpoints - Navigating the initial steps of implementing patient-focused strategies, providing insights on essential considerations before embarking on this transformative process - Delving into the essential do’s and don’ts of developing a patient-focused drug development process, drawing from practical experiences and best practices And be sure to stop by our exhibit booth to connect with our staff on-site in Philadelphia! #patientengagement #patientempowerment #patientadvocacy #patientsaspartners #PAP2024 #clinicalresearch #drugdevelopment

We are excited and looking forward to the upcoming Patients As Partners in Clinical Research Community News conference. Don't miss our presentation on March 20, Implementing a Patient-Focused Drug Development Framework for Enhanced Patient-Centric Strategies. The session, featuring Angela Wheeler, will share how to implement a Patient Focused drug development framework, integrating patient engagement into the clinical development process—a versatile approach applicable across diverse organizational systems. Key discussion points include: - Assisting biopharma companies in identifying their specific requirements and desires from patients at different stages - Understanding the untapped benefits of patient involvement, extending beyond protocol design to encompass crucial aspects like determining endpoints - Navigating the initial steps of implementing patient-focused strategies, providing insights on essential considerations before embarking on this transformative process - Delving into the essential do’s and don’ts of developing a patient-focused drug development process, drawing from practical experiences and best practices And be sure to stop by our exhibit booth to connect with our staff on-site in Philadelphia! #patientengagement #patientempowerment #patientadvocacy #patientsaspartners #PAP2024 #clinicalresearch #drugdevelopment

Accelerated approval for rare disease therapies holds so much promise for the entire rare disease community, and particularly for the Sanfilippo community as promising therapies edge towards the regulatory approval phase. Please register ASAP for this important event to be held Wednesday, 21 February from 10am-4pm (EST).  This day-long hybrid public workshop will explore primary disease activity biomarkers in rare genetic diseases using heparan sulfate in neuronopathic mucopolysaccharidoses (MPS) as a case study for a biomarker to support accelerated approval. Perspectives from FDA representatives, patient advocates, researchers, and representatives from regulated industry will be presented and there is the option for in-person or remote participation. #sanfilipposyndrome #raredisease #patientadvocacy #FDA

We are excited and looking forward to the upcoming Patients As Partners in Clinical Research Community News conference. Don't miss our presentation on March 20, Implementing a Patient-Focused Drug Development Framework for Enhanced Patient-Centric Strategies. The session, featuring Angela Wheeler, will share how to implement a Patient Focused drug development framework, integrating patient engagement into the clinical development process—a versatile approach applicable across diverse organizational systems. Key discussion points include: - Assisting biopharma companies in identifying their specific requirements and desires from patients at different stages - Understanding the untapped benefits of patient involvement, extending beyond protocol design to encompass crucial aspects like determining endpoints - Navigating the initial steps of implementing patient-focused strategies, providing insights on essential considerations before embarking on this transformative process - Delving into the essential dos and don’ts of developing a patient-focused drug development process, drawing from practical experiences and best practices And be sure to stop by our exhibit booth to connect with our staff on-site in Philadelphia! #patientengagement #patientempowerment #patientadvocacy #patientsaspartners #PAP2024 #clinicalresearch #drugdevelopment

Early Phase clinical trials present a slew of unique challenges. Biospecimens are increasing in importance across the board, so what challenges do sponsors face in the early phase setting? Join us to find out! https://lnkd.in/dkGfHQkC

Last week, Science 37 announced the final enrollment figures from its 20-month engagement with a clinical-stage drug development company in a Phase 2 rare disease clinical trial for patients with Cholestatic Pruritus. The study protocol mandated a highly specific patient population, requiring participants to undergo evaluation against rigorous inclusion and exclusion criteria. Science 37 surpassed its contracted enrollment goal, achieving an average enrollment rate of 1 patient per month, compared to the 0.1 patients per month rate at each of the 51 participating traditional brick-and-mortar sites. “Traditional recruitment methods are geographically limited, whereas Science 37 can recruit and enroll patients located anywhere, not just within a certain travel radius of a trial site. This significantly broadens the pool of qualified participants, which is vital for rare disease research,” explained Erica Prowisor, SVP of Patient and Provider Networks at Science 37. Read the press release: https://lnkd.in/e383UtCH #clinicalresearch #clinicaltrials #clinicalstudies #patientrecruitment #virtualsite #pressrelease

📣 Exciting News from SYNGAP1 Foundation! 🌟 Our Voice of the Patient & Caregiver Report, a result of the EL-PFDD Meeting, is now available for download. 📑 This report captures valuable insights from patients with SYNGAP1-related disorders. By sharing their experiences, we aim to drive advancements in drug development and support the community. 🌐 The SYNGAP1 Foundation, dedicated to advocating for those affected by SYNGAP1-related disorders, is thrilled to present this comprehensive summary. The report was carefully crafted to reflect the voices heard during the virtual meeting held on November 19, 2020. ➡️ To access the report, simply enter your information in the provided form. Once submitted, you'll receive an email with a link to the PDF, ensuring you can refer to it at any time. 🤝 Your participation matters! The insights shared by patients have been compiled into this final report, which will not only benefit our community but will also be shared with researchers embarking on drug trials for SYNGAP1-related Disorders. We believe in the power of collective voices to make a lasting impact. 🔗 Additionally, the Food & Drug Administration has recognized the significance of this report by hosting a link back to our page on their External Resources and Information Related to Patient Experience webpage. 🙏 Thank you for being a crucial part of this journey. Together, we're making strides towards a brighter future for those affected by SYNGAP1-related disorders. Download the report, share your thoughts, and let's continue to amplify the voice of the SYNGAP1 community! 💙 #SYNGAP1Foundation #PatientVoice #DrugDevelopment #Advocacy #CommunityUnity 🌈 https://lnkd.in/g6EZjtEp

💡 Breaking Barriers in Rare Disease Research - From development planning to regulatory submissions, ERGOMED partners with you to design and execute innovative, patient-centric clinical research programs for patients and families living with the challenge of a rare disease. Ready to learn more? Meet our team at Booth #437 at World Orphan Drug Congress USA 2024 next week! Together, let's make a difference. #RareDisease #PatientCentricity #Innovation #ClinicalResearch

Just about a week to go and counting! I am excited to see everyone in San Diego at #DIA2024. What a treat to discuss "Sustainability of Rare Disease Drug Development" with an esteemed panel of FDA experts including Julie Tierney, Patroula Smpokou, MD, and Katherine Donigan - of course expertly moderated by Darcy Frear, PhD. Current incentives for #raredisease #drugdevelopment including the #orphandrugact #ODA have changed to world for many #raredisease patients and families. We went from fewer than 40 FDA approved therapies in 1983 before the #ODA to #orphandrugs consistently making up more than 50% of new drug approvals (#NMEs) at #FDA. But more than 95% of rare diseases still lack any FDA-approved therapies. So how do we sustainably improve rare disease drug development and bring life-changing therapies to those communities that have for too long been left behind? There are so many promising approaches, pilots, and policy priorities to make this dream a reality, and I can't wait to discuss them with this esteemed panel of experts!

🎙 Last year, OVID’s Deputy Head of Public Affairs, Rachael Stewart, sat down with John James OBE, CEO of the Sickle Cell Society and judge for the 2023 edition of the Patient Partnership Index, to discuss innovative approaches in patient partnerships as part of our OVIDcast series. During the discussion, they delved into the importance of involving patients and families in the measurement of partnership impact. With entries *officially open* for this year’s Patient Partnership Index, make sure you give the episode a listen to learn why high-quality patient partnerships are growing ever more vital. 🎧 https://lnkd.in/g9HFnqYj 🎧 🌟 As a reminder, if you’re a patient organisation, pharmaceutical or biotech company with a piece of patient partnership work you’re particularly proud of, make sure you register today or contact a member of the team at info@patientpartnershipindex.com for more information. The 2024 Patient Partnership Index will close to entries on Friday 26th April just before midnight! #innovation #advocacy #OVIDcast #patientpartnerships #changemakers