Collin G. Johnson, Ph.D.’s Post

Commercial success in rare disease can be measured—in part—by how well pharma companies partner and collaborate with patients & PAGs. As more pharma companies line up to enter this challenging space, the ZS Rare Disease Practice shares some suggestions for how they can draw on the wisdom of patients to address commercial barriers. #rarediseaseday2024 #raredisease #zs

Here's a round-up of the February industry news covered by The Evidence Base®, including news about xCures, Centers for Medicare & Medicaid Services, LANGaware, Premier Inc., PINC AI™, Verisense Health, Datavant, The Innovation and Value Initiative (IVI), EveryLife Foundation for Rare Diseases, PhRMA, National Infusion Center Association (NICA), Global Colon Cancer Association, Alliance for Aging Research, Leavitt Partners, National Pharmaceutical Council, and the European Medicines Agency. #inflationreductionact #rwe #rwd #realworlddata #realworldevidence #medicare #ai #articialintelligence #machinelearning #regulation #regulatoryaffairs #hta #healthtechnologyassessment #cellandgenetherapy #healthoutcomes #healthequity #neurodegenerative #behavioralhealth #digitalhealth #rarediseases #patientengagement #ehds

Yesterday was #RareDiseaseDay. The annual observance highlights how far the #biopharma industry has come and serves as a wake-up call for how far it must still go to meet the needs of #raredisease patients. Despite recent investment and breakthroughs for #rarediseases, experts told BioSpace that helping a much larger patient population will require cross-sector support and desperately needed funding. We looked at some of the investments to date, as well as the biopharma industry’s promising pipelines and what is required to bring more groundbreaking therapies to patients with unmet medical needs. #Patientadvocacy groups have been critical to the development of the first marketed drugs for certain rare diseases, including progeria and Friedreich's ataxia. We spoke with leaders of these groups formed by families coping with rare diseases, providing case studies on their partnerships with biopharma companies. https://lnkd.in/g7R6hU23 #RareDiseaseDay2024 #genetherapy #genetherapies #duchenne #duchennemusculardystrophy #biopharmaceuticals

Huge congratulations to AIRNA for raising an additional $60 million in their Series A round, totaling $90 million! 🎉 This funding will push forward the company's groundbreaking RNA editing treatment for alpha-1 antitrypsin deficiency (AATD), a rare but serious genetic disorder that can affect the liver or lungs. In recent years, biopharmaceutical companies have ramped up their R&D investments in rare diseases. Why? Perhaps because the average direct cost to conduct a Phase III trial for rare disease treatments is about half that of non-rare diseases ($103 million vs. $193 million), according to a 2019 study by Tufts Center for the Study of Drug Development (Tufts CSDD). This lower cost, coupled with fewer competitors and strong patient advocacy, makes rare disease research a promising area for innovation. With 30-40 million people in the U.S. living with rare diseases and nearly 300 million living with rare diseases worldwide, it's time to prioritize rare disease research and to work harder to address these unmet needs. #HealthcareInnovation #RareDiseases 

Yesterday, the FDA announced plans to launch a Rare Disease Innovation Hub that will utilise new and collaborative approaches to expedite the development and approval of safe and effective drugs and biologics that treat rare diseases. One out of every 10 people in the US are affected by a rare disease and around half of these people are children. Many rare conditions are life-threatening, and most do not have approved treatments. The new Hub will work across rare diseases with a focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. It will serve as a single point of connection and engagement with the rare disease community; enhance agency collaboration to address common scientific, clinical and policy issues related to rare disease product development; and advance regulatory science. Having supported organisations across the globe with the development of medicines for rare diseases, Boyds warmly welcomes this promising initiative which ultimately aims to improve outcomes for people living with rare diseases by providing new therapeutic options. Find out more: https://lnkd.in/egJKYwvs #rarediseases #fda #innovationhub #medicine

Today is Rare Disease Day - the official international awareness raising campaign for the 300 million people currently living with one of the 6,000+ rare diseases worldwide. The primary goal of the campaign is to increase awareness amongst the general public and decision-makers about rare diseases and their influence on the lives of families living with these conditions. In the article below, BAP Pharma's Group Director of Medicines Access and UK General Manager - Rebecca Bibby - highlights our Medicines Access team's commitment to bridge the gap between patients and companies developing advanced medical treatments, providing access to combat rare diseases across the globe. https://lnkd.in/ej5q4BwU For more information on how BAP Pharma can support your medicines access needs, please contact us at bd@bappharma.com or visit our website at www.bappharma.com #rarediseaseday #raredisease #medicinesaccess #marketaccess #clinicaltrials #cellandgenetherapy #celltherapy #generaltherapy #biotech #medicine #pharmaceuticals #oncology #patientexperience #pharmacy #pharmacists #orphandrugs #biotechnology #nhs

Imagine going to Uni with someone who has done a billion dollar deal with Eli Lilly and Company. Couldn't be more proud of what he has achieved, well done mate! Samir Ounzain Spent this morning at the 50th DMDg open meeting discussing the importance of new modalities and the unique DMPK challenges they pose. Big pharma are investing heavily in their future which will ultimately make our future brighter. #darkgenome, #Oligos, #DMDg #HAYATherapeutics

🇺🇸The FDA is launching a Rare Disease Innovation Hub to enhance the development and approval of treatments for rare diseases. Connecting with patient communities, fostering intercenter collaboration, and advancing regulatory science will be the focus. This initiative co-led by the directors of CDER and CBER is expected to significantly improve outcomes for patients with rare diseases by streamlining the development of new therapies and leveraging cross-agency expertise and existing programs. 🇬🇧 The Medicines and Healthcare products Regulatory Agency (MHRA) has initiatives similar to the FDA's Rare Disease Innovation Hub. One notable initiative is the Rare Therapies Launch Pad (RTLP) announced in the UK’s 2023 Autumn Statement. This program aims to develop a new pathway for the development and access to novel medicines, particularly targeting children with rare and life-threatening conditions. It involves collaboration among Genomics England, the MHRA, Oxford-Harrington Rare Disease Centre, the Association of the British Pharmaceutical Industry (ABPI), and Mila’s Miracle Foundation. Additionally, the UK government has established the Rare Diseases Action Plan, which aligns with the UK Rare Diseases Framework. This plan focuses on improving diagnosis, increasing awareness among healthcare professionals, coordinating care, and enhancing access to specialist treatments. It includes actions like enhancing genomic testing communication, implementing networked models of care, and addressing health inequalities for those with rare conditions. However, the continuity of the UK's Rare Disease Action Plan is a topic of concern with the new Labour government which has not specifically mentioned rare diseases in their manifesto but have committed to advancing health innovation, such as genomics, which is crucial for rare disease diagnostics and treatments.

$1-Billion-dollar deals: A new era for rare disease treatments? In a landscape where only 5% of rare diseases have approved treatments, recent acquisitions signal a tidal shift. With companies like Ipsen, Chiesi Farmaceutici, and Amgen investing billions, the economics of rare disease treatments are evolving. Read my latest opinion piece in the link and comment here what your thoughts are on the current landscape for rare disease treatments. https://lnkd.in/ea5Da82b #BiotechInnovation #HealthcareInnovation #RareDisease #AmbroseHealthcare

Follow the link below to read some great insights from Toby Wilson Waterworth on deals associated with #rarediseases