RAPS UK Local Networking Group’s Post

The UK Medicines and Healthcare products Regulatory Agency (#MHRA) has approved a product licence for the medicine lecanemab (Leqembi) for use in the early stages of #AlzheimersDisease, following a thorough review of the benefits and risks: https://lnkd.in/emZR7tWY #Alzheimers

Have you considered incorporating RWD/RWE in your integrated evidence strategy? The recent FDA draft guidance on confirmatory evidence paves the way for the inclusion of RWD/RWE to support drug effectiveness studies. Partner with UBC to develop an evidence generation strategy that can improve regulatory review to help bring safe and effective treatments to patients. Download our white paper to learn more: https://hubs.li/Q02xX_920 #PatientsFirst #RWE #RealWorldEvidence #RWD #RealWorldData

🏆 Benefits of incorporating RWE/RWD into your integrated evidence strategy: ✅ Enhanced understanding of treatment effectiveness, ✅ support regulatory approval, ✅ support HTAs and Payer Decision. #RWE #RWD #effectivenessstudies Judy Lytle, PhD, MBEE, PMP Irene Cosmatos Annette Stemhagen Aaron Berger Jay Jean-Mary if want to know more we are at #DIASanDiego 🤝 🧠

A great piece on the use of confirmatory evidence to demonstrate drug effectiveness and expedite the regulatory approval process (as per recent FDA guidelines). Quality and quantity of data sources used to substantiate clinical trials are equally important considerations when developing your evidence generation strategy. Be sure to download our white paper via the link below!

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved leniolisib phosphate (Joenja) as the first treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). #APDSmedicineapproval #APDStreatmentinUK #immunesystemtreatmentUK #Joenjaleniolisibbenefits #leniolisibapprovedforimmunedisorder #leniolisibforAPDS #leniolisibphosphateapproval #MHRAapprovesleniolisib #MHRAleniolisibJoenja #MHRArarediseaseapproval #thxnews

Rare Pediatric Disease Priority Review Vouchers are a well-known FDA regulatory pathway to speed up regulatory approvals. Did you know that there are two others priority review vouchers (PRV) types? Those are (1) the Medical Counter-Measure (MCM) priority review voucher, and (2) the Neglected Tropical Disease priority review voucher. The MCM-PRV most recently came in the news recently after Novartis' approval in December 2023 of FABHALTA (iptacopan) for the treatment for adults with paroxysmal nocturnal hemoglobinuria (see here: https://lnkd.in/geZkBYv2) An excellent ressource to learn more about PRVs: https://lnkd.in/gYWZX3ks I also recommend the (now slightly dated) US Government Accountability Office Report regarding the FDA's PRV program: https://lnkd.in/gYmpME25 #prv #regulatoryaffairs #novartis

Reflecting back on my first BIO International Convention last week, I can say that I left with a much deeper understanding of the potential impact of the #IRA and other major recent US policies like the establishment of #PDABs on not only manufacturers and payers, but also patients. The robust inclusion of patient advocates and patient group representatives in nearly every session that I attended brought a huge amount of value by providing the full picture from the point of view of all key stakeholders. Some of my key takeaways from the sessions that I was able to attend and the people that I was able to chat with included: 1) Patient engagement is key to ensure feedback from this key stakeholder group is accounted for, particularly with these upcoming major policy changes 2) There is the need for more transparency regarding what evidence the state and federal governments will actually value in their assessments, rather than forcing everyone to "wait-and-see" once the details of the first assessments are published 3) Though Health Equity is a key pillar for CMS, there is nothing within the IRA evaluation framework that considers this important, but complex element 4) Even for smaller companies including those that think their assets will not be subject to Medicare price negotiations or PDAB evaluations, it will still be important to prove to investors that you have thought about the potential impact, whether direct or indirect, and have engaged in purposeful decision-making on the topic. Decisions can be made at the clinical development stage to minimize risk because the impact may not be seen 10-15 years down the line but could happen at the time of launch if competitors or analogs are impacted. 5) The IRA exemption for orphan drugs is overly narrow and poses a significant disincentive for manufacturers to develop therapies for rare diseases with multiple potential indications. With the draft guidance for the second cycle of the Medicare Drug Price Negotiation program up for public comment, this appears to be a topic that will likely garner quite a bit of discussion and debate. 6) With the $2,000 Part D out-of-pocket cap starting in 2025, an often overlooked provision of the IRA includes the option of beneficiaries "smoothing" their Part D out-of-pocket costs. This has potential to be transformative for patients with high out-of-pocket drug costs, but its success will hinge on the ability for CMS and other stakeholders to address challenges with designing, promoting, and implementing the program. I will be attending the DRUG INFORMATION ASSOCIATION (DIA) conference next week in San Diego as well. If you would like to speak more about these key policy changes and Evidera's Value and Access Consulting solutions, please come by the PPD booth to say hi or message me over LinkedIn to set up some time for us to chat.

🩺 Are you looking to improve your understanding and ability to start a #biologic treatment, follow up with the #patient, and evaluate the success of treatment? EUFOREA hosted its first edition of the European Biologics Training Course on 1 March 2024 at the Académie royale de Médecine de Belgique in #brussels with more than 250 attendees worldwide. A hybrid training course designed to increase engagement and understanding of #biologics among healthcare professionals (ENT specialists, pulmonologists, and allergologists) treating patients with uncontrolled type 2 diseases including #asthma, Chronic Rhinosinusitis with Nasal Polyps (CRSwNP) and Atopic Dermatitis (AD). Watch the European Biologics Training Course now and earn your #CME accreditation! 🔗 https://lnkd.in/ejmBkbn4 #eubiologics24 #health #healthcare #healthprofessionals #patients #patientcare #airwaydiseases #chronicdiseases #physicians #trainingcourse #trainingprogram #events2024 #nasalpolyps #asthmaawareness #treatment #ent #nonprofit #euforea #belgium #clinicians #pulmonologist #allergologist #atopicdermatitis #cmecredits

Are We Rare Disease Advocates Not Doing Enough in Addressing the Threat of the IRA? I’ve reread the AstraZeneca decision over and over, and I’m haunted by “what if?” questions. By and large, rare disease advocates, like me, have sat on the litigation sidelines. Should we be filing amicus briefs in the manufacturer cases? Could it have made a difference? Certainly, many rare disease organizations and advocates have been active in addressing the threats created by the IRA. Many have, like me, helped file comments on the terribly flawed “guidance” CMS issued, engaged with CMS to express concerns that the unintended consequences of the IRA will inevitably be to delay or prevent the introduction of orphan products, and highlighted for Congress the very real need to revise the statute to save orphan drug innovation. But the rare disease community has not played a meaningful role in the litigation challenges. I read and reread the opinion. Each time I grew more concerned as the impact of the court’s holding, rationale, and language cut deeper. It’s a decision that contains one gut punch after another. It dismissed the threats to drug innovation as “speculative”, brushed aside the impacts on clinical trial initiation as insufficiently “concrete”, and failed to get to the merits of the risk to innovation posed by the agency taking a “single source” drug price control system and enlarging it to wholly different drugs approved under different applications. That baseless rewriting of the statute risks swallowing up orphan drug development programs whole. I don’t know that rare disease voices expressed through amicus briefs would have changed the outcome, but I do know that, in a fight this important, our voices should be heard—directly. If you are at a rare disease organization, and you think the same, I’d like to help. #astrazeneca #raredisease #pricecontrols #drugpricing #drugdevelopment #clinicaltrials #clinicalresearch #orphandrugs

🔊 The FDA recently published guidance regarding general principles on planning, designing, and analyzing Pharmacoepidemiological Studies that utilize real-world data for medicine safety assessment. This is exciting news since conducting post-market pharmacoepidemiological studies is crucial for assessing the benefits and risks of 💊 medicines in children, especially since they are often left out of pre-approval clinical studies. 𝑳𝒆𝒕'𝒔 𝒄𝒐𝒏𝒕𝒊𝒏𝒖𝒆 𝒕𝒐 𝒑𝒓𝒊𝒐𝒓𝒊𝒕𝒊𝒛𝒆 𝒐𝒖𝒓 𝒄𝒉𝒊𝒍𝒅𝒓𝒆𝒏'𝒔 𝒉𝒆𝒂𝒍𝒕𝒉 𝒂𝒏𝒅 𝒘𝒆𝒍𝒍-𝒃𝒆𝒊𝒏𝒈. 𝐡𝐭𝐭𝐩𝐬://𝐰𝐰𝐰.𝐟𝐝𝐚.𝐠𝐨𝐯/𝐫𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲-𝐢𝐧𝐟𝐨𝐫𝐦𝐚𝐭𝐢𝐨𝐧/𝐬𝐞𝐚𝐫𝐜𝐡-𝐟𝐝𝐚-𝐠𝐮𝐢𝐝𝐚𝐧𝐜𝐞-𝐝𝐨𝐜𝐮𝐦𝐞𝐧𝐭𝐬/𝐦14-𝐠𝐞𝐧𝐞𝐫𝐚𝐥-𝐩𝐫𝐢𝐧𝐜𝐢𝐩𝐥𝐞𝐬-𝐩𝐥𝐚𝐧-𝐝𝐞𝐬𝐢𝐠𝐧-𝐚𝐧𝐝-𝐚𝐧𝐚𝐥𝐲𝐬𝐢𝐬-𝐩𝐡𝐚𝐫𝐦𝐚𝐜𝐨𝐞𝐩𝐢𝐝𝐞𝐦𝐢𝐨𝐥𝐨𝐠𝐢𝐜𝐚𝐥-𝐬𝐭𝐮𝐝𝐢𝐞𝐬-𝐮𝐭𝐢𝐥𝐢𝐳𝐞-𝐫𝐞𝐚𝐥-𝐰𝐨𝐫𝐥𝐝 #𝐏𝐞𝐝𝐢𝐚𝐭𝐫𝐢𝐜𝐑𝐞𝐬𝐞𝐚𝐫𝐜𝐡 #𝐏𝐡𝐚𝐫𝐦𝐚𝐜𝐨𝐞𝐩𝐢𝐝𝐞𝐦𝐢𝐨𝐥𝐨𝐠𝐲 #𝐁𝐞𝐜𝐚𝐮𝐬𝐞𝐂𝐡𝐢𝐥𝐝𝐫𝐞𝐧𝐂𝐚𝐧𝐭𝐖𝐚𝐢𝐭