Memo Therapeutics AG’s Post

Success in treating rare disease can be measured—in part—by how well pharma companies integrate feedback from patient advocacy groups into every step of the commercialization journey. As more pharma companies line up to enter this challenging space, we offer some suggestions for how they can draw on the wisdom of patients. #patientadvocacy #healthcareinnovation

Putting the people who are living with rare diseases at the heart of any patient-centered research we do has been one of our aims since day dot (before even the term patient-centricity got floated about!). We appreciate that the pharma and biotech companies that we’re working with have also embraced our innovative approaches to this. 💛💡 It’s seen us bring together PAG communities – in some cases, internationally, to take a study from protocol to publication with the patient voice represented throughout. We’re committed to making a difference in the lives of those facing unique health challenges and highlighting the value of the rare disease community voice for both patients and caregivers. I’m sharing below an example of some of these approaches taken by Vitaccess - bridge building between patient and pharma with the Charcot-Marie-Tooth (CMT) story. Read more below V 💙 #charcotmarietooth #RareDiseaseDay #PatientCenteredResearch

We are live at the World Orphan Drug Congress USA! Come meet with us at booth #413 and talk about how we can tackle rare disease challenges together. We can help find people living with rare disease that are still undiagnosed, often many years before they might eventually be diagnosed following standard clinical diagnostic pathways. And help understand who are most at risk of rapidly progressing towards poor outcomes, thus helping HCPs triage and accelerate care pathways for better outcomes for patients. If you have missed our pre-conference workshop yesterday, the slides and, importantly, survey are now uploaded to the Terrapinn World Orphan Drug Congress USA conference app. See link in comments. And if you are at the event, come listen to our speaker session "How you can deliver consequential projects for patients, using AI in multi-year deployments.". See session link in comments. #inTrigue #inAdvance #inFlow #rarediseaseawareness #rarediseaseresearch With Volv Global SA 💭 Christopher Rudolf and Mike Musson at the Terrapinn World Orphan Drug Congress USA with Justin Franks, Alexandria Edleman , Giovanna Ronzetti and many others.

Did you know that industry-sponsored clinical trial launches in the UK fell by over 40% between 2017-21? We explored how the UK government is changing the way it works with the life sciences sector to reverse this trend.   Industry-sponsored clinical trials are pivotal to the UK’s life sciences sector. In a bid to restore the country’s historic global leadership in such trials, the government commissioned ex-Health Minister Lord O'Shaughnessy to identify key bottlenecks and guide on ways to not only mitigate them, but to also to deliver internationally exemplary services at the benefit of both patients and industry. Since expanding our Axiom Think Tank ‘across the pond’ to the UK in 2022, these improvements to trial set-up are critical to clients engaged with our London team as they seek to launch studies for their innovative oncology and rare disease treatments in the UK. Learn more in the infographic below about key barriers and recommendations outlined in the review, what this means for different stakeholders, and the remarkable progress seen as early as 6 months after – including nearly tripling the average monthly patient recruitment into commercial trials!   #ClinicalTrials #PharmaceuticalIndustry #Biotech #LifeSciences #UKLifeSciences #Healthcare #PatientAdvocacy #NHS #AxiomThinkTank

Exciting news for the myositis community! As the world's leading myositis patient organization, TMA is proud to share that AstraZeneca has begun recruitment for two Phase III trials: LAVENDER for cutaneous lupus erythematosus and JASMINE for idiopathic inflammatory myopathies. TMA's Executive Director, Paula Eichenbrenner, expressed hope and optimism for this potential new treatment option, offering fresh possibilities for those living with myositis. Read the full press release here: https://lnkd.in/eFp9QEXM #Myositis #MyositisWarrior #MyositisAwareness #TheMyositisAssociation #MyositisSupport #RareDisease #MYOMovement #MyositisLife #MyTMA #PatientAdvocacy #TheMyositisAgenda

📣 Exciting news from Rallybio! Today at #ASBMR2024 with our partners Exscientia and Millán Lab, we debuted promising nonclinical data supporting ENPP1 inhibition as a therapeutic approach for hypophosphatasia (HPP) for the first time. Thanks to Dr. José Luis Millán for presenting these findings - With the significant unmet patient need, especially among adults, our findings bring hope for a potential new treatment that could improve the lives of those battling this devastating rare disease.     We're encouraged to see this data as we continue to work with Exscientia on an ENPP1 inhibitor with improved properties compared with REV101 as a differentiated therapy to address the unmet need in patients with HPP. We expect to have more to share on our inhibitor development candidate later this year - for now, download our poster here to take a closer look: https://lnkd.in/eesRGgsr   #RareDisease #HPP #ASBMR2024 

A new IHPI policy brief by Kassem Faraj, Vahakn Shahinian, and colleagues examines the association between hospital participation in the 340B Drug Pricing Program and the use of oral specialty drugs for advanced prostate cancer. The team found treatment initiation was the same whether men received care at a 340B- or non-340B participating hospital. However, men from communities with high social and economic vulnerability were less likely to start treatment than men from communities with low vulnerability. Men taking oral specialty drugs had similar overall adherence whether they were treated at a 340B- or non-340B hospital, but patients from highly vulnerable communities were more likely to continue treatment if they received their care at a 340B-participating hospital. In the brief, Faraj and Shahinian discuss the implications of their findings for health policy and practice, including the importance of learning about and implementing strategies and programs that improve patient outcomes. Read more: https://lnkd.in/eS3ZvFxR

I’ve been a terrible rare disease patient. That’s a hard thing to write. But it’s true. And I can’t avoid the truth if it. Or the sting of it. I was reading a piece yesterday about the challenges faced by rare disease organizations. The author said that one of the common obstacles to successful rare disease efforts—whether enrollment in clinical trials, advocacy efforts, or pushes for coverage—is “the detached patient”. The detached patient, the piece said, gets the diagnosis, puts their head down, and does their best to ignore the disease. I saw myself in that mirror. It’s embarrassing. But that was me. I never participated in a registry, I did not enroll in a study, and I didn’t do fundraising. I just put my head down and tried to ignore it. It’s particularly embarrassing because most of my professional life was devoted to rare disease issues. I helped launch almost 200 rare disease products, I developed a similar number of value-based pricing and contracting offers, I developed compliance programs for dozens of rare disease hubs, I defended rare disease drug manufacturers and patient charities in federal investigations, I secured advisory opinions from OIG that related to rare disease products, and I worked on the design of gene testing programs. But never for my disease. I never picked up the phone or sent an email asking “hey, is there something I can do to help?” Until three months ago, a few days after I told my law firm that, after 35 years of practicing law, it was time to do something new. I should have taken that step 30 years ago. But at least I’ve taken it now. #rarediseases #raredisease #fshd #orphandrugs

Our latest poster presentation, "A Paradigm Shift in Collecting RWD for GM2 gangliosidosis," introduces a game-changing method for gathering real-world data (RWD) on GM2 gangliosidosis. We're overcoming historical challenges by aligning with FDA guidelines and fostering collaboration across stakeholders. Our approach integrates insights from Key Opinion Leaders and patient advocacy leaders, prioritizes transparency, and aims to accelerate progress in rare disease research. Read more about our approach here: https://lnkd.in/eUstpt9S #WorldSymposium #RareDiseases #LysosomalDisease #CareAboutRare

Patient centricity has become the norm in biopharma, at least it should be, right? 🤷♀️ Well.. a report on HCPs attitudes to biopharma companies only 45% of them feel they embody patient centricity at a high level. #Patientcentricity is now more than just patient research and usability testing. Its: 1. engaging the community as contributors in the drug development  2. co-creation in the design of end to end clinical trials 3. leveraging the communities that surround patients to understand the true patient experience AND A LOT MORE! Avalere Health have developed an article touching on how rare disease teams may approach the ultimate goal of patient co-creation. Why not check it out? https://bit.ly/3SRVGDA Other helpful information on Rare Disease patient co-creation: - National Organization for Rare Disorders (NORD) at NCSU have developed on Education Series to Advance Patient Involvement in Rare Disease Drug Development - Rare Disease Moonshot- an organisation working with patient groups to optimize translational research, modernize clinical trials, and develop the infrastructure to shorten the path to diagnosis and treatment. #raredisease #patientcentricty #biopharma #patientexperience #drugdevelopment #thoughtleadership