Ascenian ’s Post

Interesting analysis by hollandbio on patient access to #OrphanDrugs in the Netherlands. Some key insights: 💊 Of all 242 orphan drugs registered between 2001 and 2023, 61% has a from of reimbursement (patient access) 💊 Over the past years, this percentage has been dropping 💊 Drugs with a conditional EMA approval have a lower rate of reimbursement 💊 Drugs from small and midsize biopharma have a lower rate of reimbursement 👉 Read the full article here: https://lnkd.in/e3ksHZ4K #OrphanDrugs #RareDiseases

Updates from the German AMNOG orphan drug space: 5️⃣ new procedures started, 4️⃣ completed, and 6️⃣ rebates negotiated. Our comprehensive orphan tracker keeps you in the loop. ⬇ Below, you can find an overview of our March and April edition. The complete tracker with all details on new and completed AMNOG orphan drug procedures, new rebates and an analysis about the orphan drug first submissions per year can be downloaded free of charge at https://lnkd.in/eiACGG9N. All analyses are generated by data from SKC‘s proprietary MAIS 🌽 database. #orphandrugs #ATMPs #AMNOG #marketaccess #reimbursement

Caplin Steriles Limited, a Subsidiary Company of Caplin Point Laboratories Limited, has been granted final approval from the United States Food and Drug Administration (USFDA) for its Abbreviated New Drug Application (ANDA) Ephedrine Sulfate injection USP, 50 mg/mL Single Dose vial, a generic therapeutic equivalent version of the Reference Listed Drug (RLD) AKOVAZ. Ephedrine Sulfate injection USP, 50 mg/mL is indicated for the treatment of clinically important hypotension occurring in the setting of anaesthesia. According to IQVIATM (IMS Health), Ephedrine Sulfate injection USP 50 mg/mL had US sales of approximately $36 million for the 12-month period ending May 2024. Click on link for Press Release : https://lnkd.in/gs7BujzD #ANDA #caplinpointlaboratories #CaplinSterilesUSAInc #CSU #caplinpoint #caplinsteriles #caplinone #caplinonelabs #caplinapprovals #sterilemanufacturing #pharmamanufacturing #injectables #formulations #RandD #solidorals #cancertreatment #oncology #ANDAapproval #ephedrinesulfate #ephedrinesulfateinjection #settinganaesthesia #hypotension #singledosevial #AffordableHealthCareForAll

READ NOW: NAVLIN Last Week in Review https://ow.ly/7cRL50T15h0 This week from NAVLIN by EVERSANA's “Last Week in Review,” recapping the latest regulatory changes, pricing & reimbursement updates, drug approvals and more from across the world. Policy: The Biden Administration reveals final prices for the first ten drugs under the IRA, with discounts up to 80%! #HealthcarePolicy Pricing & Reimbursement: Germany’s G-BA mandates early benefit assessments for all reimbursable drugs, even without competitors. #PharmaPricing HTA: Spain’s Ministry of Health drafts a Royal Decree to separate clinical and non-clinical health technology assessments. #HealthTech C&G Therapy: Genprex secures a patent for Reqorsa Immunogene Therapy in Singapore, enhancing cancer treatment options. #GeneTherapy Managed Entry Agreements: AIFA ends managed entry agreements for Eli Lilly’s Cyramza in Italy, effective August 13, 2024. #PharmaAgreements Supply Issues: Novo Nordisk advises against starting new patients on Ozempic and Victoza in Latvia due to supply constraints. #PharmaSupply And so much more...

🔍 Key Highlights from NAVLIN's Weekly Global Pharma Insights: 💡 Policy: The Biden Administration drops final prices for the first 10 drugs under the IRA—discounts up to 80%! #HealthcarePolicy 💡 Pricing & Reimbursement: Germany’s G-BA is shaking things up with mandatory early benefit assessments for all reimbursable drugs, even without competition. #PharmaPricing 💡 HTA: Spain’s Ministry of Health proposes a Royal Decree to separate clinical and non-clinical health technology assessments. #HealthTech 💡 C&G Therapy: Genprex secures a new patent for Reqorsa Immunogene Therapy in Singapore—taking cancer treatment to the next level. #GeneTherapy 💡 Managed Entry Agreements: Italy’s AIFA ends agreements for Eli Lilly’s Cyramza—effective August 13, 2024. #PharmaAgreements 💡 Supply Issues: Novo Nordisk advises caution on starting new patients with Ozempic and Victoza in Latvia due to supply shortages. #PharmaSupply And that’s just the tip of the iceberg… 🚀 👉 Catch the full review here: NAVLIN Last Week in Review Stay informed, stay ahead! #PharmaUpdates #GlobalHealth #RegulatoryNews

𝗢𝗿𝗽𝗵𝗮𝗻 𝗱𝗿𝘂𝗴𝘀 𝗣𝗗𝗨𝗙𝗔 𝗱𝗮𝘁𝗲𝘀 𝗮𝗻𝗱 𝗙𝗗𝗔 𝗿𝗲𝘃𝗶𝗲𝘄𝘀 𝗳𝗼𝗿 𝗤𝟯/𝟮𝟬𝟮𝟰 🚀 The US FDA advances rare disease treatments through scientific progress, despite challenges like workloads and complex science. Since the Orphan Drug Act 40 years ago, over 600 drugs for 1,200 indications have been approved. The Prescription Drug User Fee Act (PDUFA) sets review deadlines of 10 months, or 6 months if the drug is given a priority review designation. Meeting these deadlines is challenging due to factors like drug complexity, data quality, and review team workload. We fully appreciate the effort required throughout the commercialisation process, not least of which - the approval processes. Our team is always considering ways to accelerate outcomes through data-driven decision making and automation of processes to free up time for practitioners. Vinod Bombale and Wayne Speechly are always available to explore various insights we’re seeing across our 200m+ data points and welcome what you’re seeing in the market. #regulatoryapprovals #orphandrugs #fda #pdufa #drugcommercialisation

🧠 Biogen and Eisai’s Alzheimer's drug is rejected by the EU regulator. Here is my summary: ❌ The European Medicines Agency (EMA) has recommended against granting market authorization for Leqembi, Biogen and Eisai’s Alzheimer’s drug. The EMA's decision is based on concerns that the risk of serious brain swelling associated with the drug outweighs its modest benefit in slowing cognitive decline. 🌍 Despite having received approval from six regulatory authorities, including the U.S. and Japan, this setback is significant for the drug-makers. Leqembi has had a sluggish launch in the U.S., partly due to the requirement for additional diagnostic tests, bi-monthly infusions, and brain scans at clinics. 👀 This regulatory rejection of Leqembi casts a shadow over the EU prospects for Eli Lilly’s Alzheimer’s drug, donanemab, which was recently approved by the FDA. To avoid facing a similar fate as Leqembi, donanemab will need to demonstrate a more favorable benefit-risk profile in its European review. #Biogen #Eisai #Alzheimers #amyloid #leqembi

What does that mean for drug development in the rare disease space? We saw a 40% increase in the number of novel orphan drugs approved compared to the 20 approved the previous year. It was a reversal of a two-year decline in novel rare disease drug approvals. Read more about drug approvals in the 2024 NEXT Report: Rewriting the Rules. Download your copy: https://lnkd.in/eqgPQcHq #NEXTReport #NEXTReport2024 #RareDrugDevelopement #DrugApprovals #RareDisease #RareDiseaseCommunity #RareDiseaseFact #CareAboutRare

I am proud of the work accomplished by the Regulatory Submission teams under the leadership of Steve Sibley at Certara. You will be hard-pressed to find a service partner with the depth and breadth of rare disease experience similar to Certara. Please contact me to learn how we can accelerate your regulatory submission. #regulatorysubmissions #regulatorywriting #CoAuthor #raredisease

What does that mean for drug development in the rare disease space? We saw a 40% increase in the number of novel orphan drugs approved compared to the 20 approved the previous year. It was a reversal of a two-year decline in novel rare disease drug approvals. Read more about Drug Approvals in 2023 in the 2024 NEXT Report: Rewriting the Rules. Download your copy: #NEXTReport #NEXTReport2024 #RareDisease #RareDiseaseCommunity #RareDiseaseFact #CareAboutRare