Market Access Today’s Post

Spinal Muscular Atrophy Treatment Expanded: Medicinrådet Recommends Risdiplam for Patients Over 25 Following New Pricing Agreement The Danish Medicines Council (Medicinrådet) has expanded its recommendation for the SMA drug risdiplam, marketed by Roche as Evrysdi, to include patients over 25 years old, following a new pricing agreement with Roche that balances cost with expected treatment benefits. This expansion marks a significant advancement in SMA treatment, offering a non-invasive, oral therapy option for all ages. While patient advocacy groups have welcomed this broader access, the decision also underscores the ongoing challenges of ensuring equitable and sustainable access to expensive therapies for rare diseases. The new pricing model reflects the varying levels of clinical evidence for different age groups, ensuring that the treatment remains accessible while protecting healthcare budgets. For more details please click the link! https://lnkd.in/dkNSaG8u #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

Synergy Healthcare is thrilled to share groundbreaking developments in the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The Food and Drug Administration (FDA) is on the verge of approving the first-ever medicine specifically designed to combat this serious liver disease. Developed by Madrigal Pharmaceuticals, the pill, known as resmetirom, targets a protein in the liver, effectively reducing liver fat, inflammation, and scarring associated with MASH. If approved, resmetirom will mark a significant milestone in healthcare, offering hope to patients with advanced stages of MASH where current treatments have shown limited effectiveness. At Synergy Healthcare, we pride ourselves on being at the forefront of clinical research, especially in metabolic and neurological diseases. The potential approval of resmetirom represents a significant step forward in addressing the unmet medical needs of patients suffering from MASH. The addressable market for drugs to treat advanced MASH is substantial, with an estimated 315,000 individuals in need of effective treatment. Resmetirom's innovative approach and promising clinical trial results position it as a potential game-changer in the field of liver disease treatment. We commend the dedication and perseverance of researchers and scientists, including those at Madrigal Pharmaceuticals, for their tireless efforts in bringing new therapeutic options to patients in need. Stay tuned for more updates as we eagerly await the FDA's decision on resmetirom, and let's continue our collective efforts to advance healthcare and improve patient outcomes! 💊💪 #SynergyHealthcare #ClinicalResearch #MASHTreatment #Resmetirom #HealthcareInnovation #LiverDiseaseTreatment #FDAApproval #MedicalBreakthroughs 🔗 https://lnkd.in/ekjzn4uy

📢 Exciting News for CIDP Patients! I'm happy to hear the news that offers a new treatment option for those living with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Working with PTCE, I've learned so much about CIDP by collaborating with expert clinicians, understanding medication challenges from pharmacists and nurses, and even had the privilege of talking to a couple fantastic patient advocates. Their stories highlight the need for effective treatments. On Friday, argenx announced the FDA approval of Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), a neonatal Fc receptor (FcRn) blocker. This is the first new treatment mechanism in over 30 years, offering hope and a new option for patients. Efgartigimod alfa and hyaluronidase-qvfc showed significant improvements in mobility, function, and strength in clinical trials. Let's celebrate this monumental step forward in CIDP treatment! #CIDP #PatientCare #NewTreatment #FDAApproval #efgartigimod #argenx #HealthcareInnovation #PTCE

FDA is alerting patients and health care professionals about the voluntary withdrawal of Oxbryta from the market due to safety concerns FDA is alerting patients, caregivers, and health care professionals that Oxbryta (voxelotor) is being voluntarily withdrawn from the market by the manufacturer due to safety concerns. Health care professionals should stop prescribing Oxbryta. Patients and caregivers should contact their health care professional about stopping Oxbryta and starting another treatment option. Pfizer Inc., the manufacturer of Oxbryta, announced it is voluntarily withdrawing the medication from the market, ceasing distribution, and discontinuing all active clinical trials and expanded access programs for Oxbryta because recent data indicate the benefit of Oxbryta does not outweigh the risks for the sickle cell patient population. #fda #drug #recall https://lnkd.in/gpwA_ZFQ

Pharmaceutical Industry Faces Challenges as Biden Administration Implements Bold Medicare Drug Price Cuts The Biden Administration has announced significant price reductions for ten Medicare-covered drugs under the Medicare price negotiation program established by the Inflation Reduction Act of 2022, with expected savings of $6 billion by 2026 and a projected $100 billion over ten years. Despite the potential financial relief for Medicare beneficiaries, the pharmaceutical industry has strongly opposed the initiative, citing concerns over its impact on innovation and drug development, and has filed numerous lawsuits, all of which have been dismissed by federal judges. The program's future, including potential expansions, will likely be influenced by the upcoming 2024 elections and the evolving political landscape. For more details please click the link! https://lnkd.in/dUNnBHD8 #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

🚨 Exciting News in Pharma! 🚨 Bayer's Kerendia has just delivered promising results in the Phase III FINEARTS-HF trial! 🌟 This breakthrough treatment for #KidneyDisease and #HeartFailure reduced the risk of cardiovascular death and heart failure by an impressive 16% over 32 months. With these results, Bayer is on track to expand Kerendia's market reach and achieve a projected $3 billion in peak sales. 💰 The trial included 6,000 patients across 37 countries, and the findings were presented at #ESCCongress and published in the New England Journal of Medicine. This is a significant milestone for patients with heart failure, offering hope for better outcomes. 💪❤️ #Pharma #ClinicalTrials #CardiovascularHealth #Innovation #Healthcare

Ensure Adequate Titration of β-Blockers for Optimal Clinical Outcomes in Heart Failure: Higher doses of β-blockers are associated with better clinical outcomes in patient with heart failure. #finance #pharmacy #lifesciences

IVIG, Plasmapheresis Comparable in Long-Term Efficacy for Treatment of Myasthenic Crisis: Though both treatments come with positives and negatives, ultimately, individual patient clinical presentation and accessibility will determine the proper treatment for myasthenic crisis. #finance #pharmacy #lifesciences

Advancements in Myelofibrosis Care: Pharmacists' Perspectives: Pharmacists discuss emerging therapies, challenges, and pharmacists’ vital role in myelofibrosis patient care, emphasizing disease-modifying agents and care team collaboration. #finance #pharmacy #lifesciences

The REDUCED-1 study of SoniVie Ltd's TIVUS renal denervation system for hypertension treatment—an FDA investigational device exemption (IDE)-approved pilot study—has recently completed enrolment. SoniVie's CMO, Christian Spaulding, has stated that the the “initial results from the ongoing REDUCED-1 study are encouraging," and CEO Tomaso Zambelli also stated that their next step will be to “clinically validate the TIVUS system in a global pivotal trial”. To learn more about this, click the link below to read the full article! #renaldenervation #hypertension