PDA Southeast Chapter’s Post

Part 2 of NORD's FREE online course, Rare Disease Drug Development: What Patients and Advocates need to know is now available on RareEDU at https://lnkd.in/e6brTX2H! Part 2: Navigating Drug Review and Access covers the important topics of expanded access, labeling, FDA's drug review processes, and drug repurposing. And if you haven't yet checked out Part 1: Foundations and Pre-Clinical Research, you can find that on RareEDU as well!

May is IBS Awareness Month! Managing IBS can be challenging, but with the right strategies, relief is within reach. EPSrx is here to support and empower individuals with IBS by providing specialized pharmaceutical solutions tailored to their unique need. Check out our small infographic for some helpful do's and don'ts to navigate the journey with IBS. We are committed to supporting and caring for our community by keeping empathy and patient advocacy at the center of our practice. #IBSAwarenessMonth #ManageIBS #epsrx #chronicillnesssupport

The PPMD Town Hall on Clinical Trials is underway TODAY, March 4th, during PPMD's 2024 Advocacy Conference in Washington, D.C.! This meeting is a platform for families impacted by Duchenne and Becker to share their experiences and insights on past, present, and future clinical trials. The primary goal is to hear directly from families and gather valuable input to shape PPMD's advocacy efforts, especially in interactions with the FDA on drug development.  AGENDA HIGHLIGHTS: - Parent/Patient Panel: Diverse voices at different ages and stages sharing their clinical trial experiences, discussing challenges, and expressing hopes for outcomes. - Professional Panel on Patient Preference and Clinical Trials: Featuring a representative from FDA CDER and clinicians covering FDA updates, issues in clinical trials, and potential solutions.  Following the meeting, a recap will be shared with the community and learnings will be integrated into PPMD's advocacy strategy moving forward as we interact with federal agencies (including FDA, NIH, DOD, and CDC). Together, we're working to enhance the clinical trial landscape for Duchenne and Becker families and contribute to policy strategies for improved outcomes. #PPMDAdvocacy

The Psychedelics Lobby: Over the past two years, psychedelic drug developers and nonprofit advocacy groups have collectively funnelled over $1.5 million into federal lobbying efforts. While that's but a drop in the bucket when compared to the broader pharmaceutical industry's enormous (and enormously controversial) lobbying spend, it's nonetheless a significant increase over prior years. In my latest for Psychedelic Alpha, I take a closer look. Link in the comments.

The World Orphan Drug Congress USA which commenced today in Boston, MA and is attended by our partner Dr. Andreas L.G. Reimann, just couldn't start better: Very insightful workshop by Albert Freedman, Daniel DeFabio and Michelle Davis. Daniel shared his really touching movie covering several parents of children with rare diseases like Menke's disease, STXBP1 and SMA. Albert talked about his experience as a clinical psychologist consulting leaders of patient advocacy groups (PAGs) internationally. Michelle provided insights in her professional life as a PAG executive of the FOP association. The panel identified important challenges of PAG leaders such as "compassion fatigue", the difficulty to manage the transition from the founders generation to the next generation of activists, burn-out and - of course - the lack of financial and human resources. There is clearly a need for professional education and coaching of PAG employees. That effort could be supported by a multi-stakeholder consortium including orphan drug companies. It was pointed out that industry should engage early (e.g. clinical trial design) with PAGs at eye’s level. PAGs are de-risking development for industry as both patients/caregivers and industry have to deal with uncertainty as Albert insightfully eluded to.

IMGs X Rare disease education 🦓 Free Certification 🎓 Have you ever thought about the complex process involved in developing medications for rare diseases? 💬 Course: 'Rare Disease Drug Development: What Patients and Advocates Need to Know.' DID YOU KNOW❓    On average, of 5,000 potential treatments that enter pre-clinical testing, only 5 are tested in humans and only 1 is approved. It takes an average of 12 years for an experimental drug to progress from pre-clinical research to approval. About 14% of drugs entering clinical trials will ultimately be approved. I have a particular interest in rare diseases, and I found this course to be extremely informative. The interviews were interesting and inspiring (no spoilers). I recommend clicking the 'mark complete' button after finishing each section. This will accurately show the percentage of the course you have completed. The course is available free online and can be accessed on mobile. It has been created by the National Organization for Rare Disorders (NORD) in collaboration with FDA's Center For Drug Evaluation and Research (CDER) and the Critical Path Institute (C-Path). 🔗 https://lnkd.in/eXs7f_NM Fun fact: The zebra is the official mascot for rare disease patients. #img #research #raredisease

🌍Reflecting on an Inspiring Day at the World Orphan Drug Congress 2024 in Barcelona! Leaders from across the globe gathered to discuss the future of orphan drugs. The theme of this year’s congress, “Strategy, Advocacy, and Partnering for the Global Orphan Drug Industry,” truly resonated. Here are a few of my key takeaways: 💡Strategic Innovation: It’s clear that the future of orphan drugs lies in strategic innovation and cross-sector collaboration. Developing therapies for rare diseases requires creative problem-solving and breaking through traditional barriers. 💡Advocacy at the Forefront: Advocacy continues to be a driving force behind progress in this field. The commitment to patient-centered approaches is not just a nice-to-have; it’s essential for ensuring that patients’ voices are heard, and their needs are met. 💡Building Partnerships: Partnerships—between industry, patient organizations, regulators, and academic researchers—are crucial to advancing new therapies. The congress highlighted how collaboration can accelerate the journey from lab to life-changing treatment. Looking forward to continuing conversations and being part of this journey towards a healthier future for all! 💙 Tamsyn F. Francisco Harrison Naomi Krikman #idearegulatory #syneos #alexion #chiesi #centogene #WorldOrphanDrugCongress #WODC2024 #OrphanDrugs #RareDiseases #Innovation #DigitalHealth #Advocacy #Collaboration

**Study update: Sanofi's AMETHIST trial for GM2 gangliosidosis discontinued** Today it was announced by Sanofi that the AMETHIST phase 3 study of venglustat for the treatment of GM2 gangliosidosis was discontinued based on the absence of positive trends on clinical endpoints. This is incredibly disappointing news as we hoped it would have potentially provided a much needed treatment option for our community. We are working with our advocacy partners Cure & Action for Tay-Sachs (CATS) Foundation and the Cure GM1 Foundation to explore next steps and we will provide more details in the coming weeks when it is available. The full press release can be read here: http://bit.ly/44imRfX. Please know that this disappointment will not stop the work we are all doing to support our communities and we will continue to explore potential future treatments. If you have any questions please reach out to us. #dontgiveup #wewontgiveup #resiliency #community #advocacy #taysachs #sandhoff #gm1 #gm2 Daniel Lewi Kathleen Flynn Diana Jussila Christine Waggoner

Dont miss “Leveraging Patient Advocacy Functions and Organizations to Provide Better Patient Experience” with B.J. Viau, Director Patient Advocacy, Rare Disease, Amgen, Alicia Barron, LGSW, Executive Director, Infusion Access Foundation, & Chrissie Jenkins, LISW-CP, Patient Advocate, Palmetto Infusion at #NICA2024 June 21-22 at the MGM Grand, Las Vegas. Sign up at bit.ly/NICA2024 Join us for a dialogue on how pharmaceutical companies, sites of care, and nonprofit organizations can work together to ensure that patients are receiving the information and resources available and vital to them to best manage their disease and their care. All of these groups have important pieces of information to share, but a more coordinated effort must be made to disseminate information and engage patients and providers. Patients are often unaware of services that they could access that would improve their quality of life, quality of care, and their financial well-being. In this session, the group will discuss ideas on how to better collaborate and will dialogue with the audience on ideas they may have or efforts already underway in their centers or companies. #infusionprovider #medicalprovider #infusion #infusioncenter #infusionnurse #infusiondeliverychannel #medicalinfusionprovider

In the intricate landscape of MPN research and development, the patient voice is not just valuable — it's indispensable. Sharing personal journeys empowers those affected by MPNs by fostering a sense of community. At the same time, it equips researchers, clinicians, and biopharmaceutical professionals with the nuanced insights necessary to tailor discovery and development efforts to meet genuine patient needs.  The era of underrepresentation of MPN patient voices is at an endpoint. We’re marking the dawn of a collaborative, insight-driven approach in our field. Stories like those from Ignacio and countless others are pivotal, enriching our collective endeavors and driving innovation in therapeutic solutions and treatment methodologies.  Join MPN Research Foundation in championing this collaborative ethos. Together, we can enhance the synergy between patient experiences and scientific expertise, accelerating our understanding and treatment of MPNs.   Stay informed and engaged with the latest advancements and insights in MPN research by joining our community today: