Greg Rossi’s Post

🌟 Advancing Rare Disease Research and Innovation with NORD 🌟 With more than 90% of rare diseases lacking an FDA-approved treatment, the need for progress has never been more urgent. NORD, founded by passionate advocates, played a pivotal role in passing the Orphan Drug Act. This groundbreaking legislation provides essential incentives for developing treatments for rare diseases. It's crucial that we continue to support a research and regulatory environment that addresses ongoing barriers and fosters a successful bench-to-bedside pathway for rare disease treatments. NORD's advocacy focuses on several key policies: 🔹 Protecting the Orphan Drug Act 🔹 Ensuring adequate funding for research and innovation at the NIH, FDA, and other agencies 🔹 Promoting patient-focused drug development 🔹 Improving clinical trials to meet the unique needs of patients with rare disorders In a recent publication in The Milbank Quarterly, NORD President Peter L. Saltonstall, Vice President of Policy and Regulatory Affairs Heidi Ross, MPH, and former Henry Waxman staffer Paul Kim highlighted the renaissance in rare disease drug development spurred by the Orphan Drug Act. They also addressed critiques of prescription drug pricing following the introduction of "blockbuster" orphan drugs. Together, we can drive progress and hope for millions of patients with rare diseases. 💪🌍 #RareDisease #ResearchInnovation #NORD #OrphanDrugAct #PatientAdvocacy #Healthcare #SfRD #SustainableDevelopment #MedicalResearch Referrals https://lnkd.in/dYA9TjWX

It's time to take action!⌛ To mark #RareDiseaseDay last month, an article by Justine Ra in Pharmaceutical-Technology discusses the FDAs drive to promote rare disease drug development.🦓 Access to digital health records and companies that support creation of registries of rare diseases is a big part of research. At Greenlight, we are proud to be working with clients who are active in the rare disease research space. Read now to learn more about the key challenges in rare disease drug development and the efforts being made to address real-world patient needs: https://lnkd.in/e54PY9Hf #RareDisease #FDA #GreenlightHealth

The FDA’s Center for Drug Evaluation and Research (CDER) is making strides in addressing rare diseases through various initiatives. With over 7,000 rare diseases affecting millions, CDER's Accelerating Rare disease Cures (ARC) Program is central to these efforts. Key initiatives include enhancing collaboration across FDA centers, developing the Rare Disease Innovation Hub, and implementing expedited review processes. These efforts aim to overcome the challenges of drug development for rare diseases, ensuring faster access to effective therapies for patients. #raredisease #FDA #CBER #healthcare #innovation Explore the full article: https://lnkd.in/dTacjaRM.

Rare diseases, such as sickle cell disease and thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. Our new report provides recommendations for enhancing and promoting rare disease drug development in the U.S. and the European Union by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between the FDA and the European Medicines Agency. "While rare diseases by definition have small patient populations, together, they afflict hundreds of millions of people — meaning very large numbers of people across the world have no drug designed to treat their condition," said Jeffrey Kahn, chair of the committee that wrote the report. Learn more at https://ow.ly/6ME850Tm7wx. #RareDisease #RareDiseases #DrugDevelopment #DrugDiscovery

Did you know? 2023 was the 40-year anniversary year of the Orphan Drug Act (ODA), and the RDCC is committed to protecting and strengthening ODA policies that support continued innovation for rare disease patients. Before the ODA was enacted in 1983, only a handful of rare diseases had FDA-approved treatments, leaving patients diagnosed with these conditions feeling alone and forgotten. But today, more than 650 medicines have been approved for orphan conditions. The ODA makes it possible for innovative biotech companies to invest in life-saving therapies, and with at least 95% of rare diseases still without treatment options, more research is critical. ODA incentives enable RDCC member companies to ODA pioneer over 200 new treatments that have the potential to change the landscape of rare disease. Learn more: https://buff.ly/3jnFlID #OneRareVoice #ODAat40 #OrphanDrugs #RareDiseases

Another important step completed - with some valuable experience of the new(ish) CTIS clinical trial approval system gained along the way!

There are fewer than 150 individuals living with #BarthSyndrome in the United States - and this #ultrarare disease is devastating. Fewer than 15% of affected individuals survive their fifth birthday, and there’s currently no FDA-approved treatment on the market.   But Stealth BioTherapeutics is a company of firsts when it comes to Barth Syndrome. They were the first to introduce the disease to the FDA; the first to conduct a clinical trial; the first to show potential improvements in how affected individuals feel and function; and the first to submit a new drug application. They recently resubmitted that application with additional supportive data, and hope FDA will, for the first time, review the application to evaluate a first potential therapy for this devastating disease.   Stealth’s story of innovation and frustration is all too common in #rare and #ultrarare drug development, where inconsistency in FDA review can compound challenges and risks already inherent to rare disease drug development.   When it comes to #BarthSyndrome and countless other rare diseases, time is of the essence. The FDA must ensure appropriate application of regulatory flexibility to all #ultrarare disease drug reviews to ensure patients have timely access to therapies.   #OneRareVoice #LifeScience #Healthcare #RareDiseaseWeek

Below are some key details regarding the Phase 3 Study conducted by AstraZeneca and Amgen for severe uncontrolled asthma. This study, conducted across multiple centers, employs a randomized, double-blind, placebo-controlled, parallel-group design to evaluate the effectiveness and safety of tezepelumab in both adults and adolescents. Let’s explore the clinical results and observe how tezepelumab compares to the standard of care for patients. #ClinicalResearch #Astrazeneca #tezepelumabtrials #Asthma #ClinicalTrials #ResearchUpdate 

This is a good summary of the diversity of participants in clinical trials that led to CDER drug approvals in 2023. It would be useful if the report included information on prevalence of the disease within each the racial and ethnic subgroups reported. Prevalence information could further enhance interpretation of the racial and ethnic composition of the study populations. #FDA #CDER #diversity #clinicaltrials

Since our September 2023 RWE4Decisions roundtable where they presented their work reviewing whether national and international rare disease registries are fit for HTA/Payer purposes, we have been following the great work of the Zorginstituut Nederland. Their final report was published in November 2023. Read the reports below. 👇🏻 One of the registries they reviewed was for a neurogenerative disorder, where highly innovative medicines often come to HTA with many uncertainties that registries could help resolve. They’ve shared this work with academics, patient experts, HTA bodies, EMA and industry to create an interesting new practical framework to guide the creation and implementation of rare disease registries to support use in research, drug development and policy taking as seen in this new publication. ⚕️The new article Framework for Multistakeholder Patient Registries in the Field of Rare Diseases Report: https://lnkd.in/eGFGHuW3. 📃Find the report to the RWE4Decisions September 2023 Roundtable: https://lnkd.in/da5uvPW5. 👉🏻 Read the final report of the Zorginstituut Nederland November 2023: https://lnkd.in/dmRhCzxJ. #RWE #RareDisease #ResearchPaper