TRANSFORM Alliance’s Post

2025 promises to be an exciting year for the TRANSFORM Alliance. With new European legislative bodies in place, we can finally resume the much-needed advocacy work for facilitating the development and uptake of #ATMPs. Watch this space and follow the TRANSFORM Alliance page to stay abreast of our work. In 2025, we are also thrilled to launch a new series profiling TRANSFORM members. We kick off with an interview with TRANSFORM Steering Group Member Giovanni Migliaccio from EPTRI. EPTRI connects academic research units with biotech actors to advance research and development in ATMPs. Giovanni higlights the key importance of partnerships between public and private organisations to further the development of ATMPs. '

Stories making headlines today in the biopharma industry: This past week has been pivotal for the biotech and pharmaceutical sectors, marked by significant advancements and strategic movements. Here’s a brief rundown of the notable developments: 1. The FDA has extended the use of Sarepta Therapeutics’ gene therapy, Elevidys, for Duchenne muscular dystrophy to include patients aged four and older. Originally approved for 4-5-year-old boys, this $3.2 million treatment now offers accelerated approval for wheelchair-dependent patients and full approval for others, providing new hope for families. 2. Lexicon Pharmaceuticals, Inc. resubmitted its type 1 diabetes medication to the FDA, nearly five years after it was initially rejected due to safety and efficacy concerns. This renewed application aims to address previous regulatory issues, potentially marking a milestone in diabetes treatment innovation. 3. Ginkgo Bioworks, Inc. and Xellia Pharmaceuticals have announced workforce layoffs, while Melodia Therapeutics has struck a $275 million partnership with Alivexis, Inc to enhance their research and development endeavors. 4. In an industry rarity, Tectonic Therapeutic, Inc. made its public market debut through a reverse merger, signaling cautious optimism despite a subdued market environment. 5. New genetic markers related to delaying Alzheimer’s onset have been identified, providing a promising new avenue for therapeutic intervention in this crucial field. 6. The US and EU have pledged over $2 billion to enhance vaccine manufacturing in Africa, aiming to bolster global health security and pandemic preparedness. 7. Zealand Pharma’s weight loss candidate, an amylin analog, has met early efficacy goals, marking a significant step forward in the fight against obesity as it moves through clinical trials. 8. Gilead Sciences has reported that its biannual injection for HIV prevention was fully effective in a Phase 3 study involving cisgender women, suggesting a potential transformation in HIV prophylaxis. 9. A promising antibody, after multiple tests by Gilead Sciences, has been acquired by a smaller biotech firm with a $40 million investment. The firm seeks to unlock the antibody’s full potential. These developments underscore the dynamic nature of the biotech and pharmaceutical industries, driven by innovation, strategic realignments, and groundbreaking research, heralding a future of potential breakthroughs and enhanced therapeutic solutions. #pharma #biotech #topstories #biodatastudio

Everything you missed in #Pharma & #Biotech 🦠🔬 ✅ Approvals FDA approves Pfizer's hemophilia B gene therapy for patients with rare hemophilia B bleeding disorder FDA approves X4 Pharmaceuticals' oral treatment Xolair (mavorixafor) for ultra-rare immune disease WHIM syndrome FDA grants accelerated approval to Day One Biopharmaceuticals' Ojemda (tovorafenib) for pediatric relapsed/refractory BRAF-mutated low-grade glioma Amneal Pharmaceuticals' OTC Naloxone hydrochloride nasal spray approved by FDA for emergency treatment of opioid overdose FDA approves ImmunityBio, Inc.'s Anktiva, a first-in-class IL-15 receptor agonist for BCG-unresponsive non-muscle invasive bladder cancer 💸 Deals Xaira Therapeutics launches with $1 billion to accelerate development of cancer and neurodegenerative disease therapies using machine learning platform Ipsen and Skyhawk Therapeutics partner to develop rare neurological therapies using Skyhawk's RNA-editing platform Incyte acquires Escient Pharmaceuticals and its pipeline of first-in-class oral MRGPR antagonists for up to $750M Bristol Myers Squibb pays $65M to expand Repertoire Immune Medicines's autoimmune assets ONO PHARMA USA acquires Deciphera Pharmaceuticals for $2.4B after biotech's Phase 3 win Cerevance adds $47M in Series B-1 extension financing to advance pipeline for neurodegenerative diseases, bringing total Series B-1 raise to $98M Vertex Pharmaceuticals and TreeFrog Therapeutics enter strategic agreement to optimize production of cell therapies for Type 1 diabetes Moderna Therapeutics - Messenger RNA Therapeutics and OpenAI collaborate to advance mRNA medicine using AI and machine learning 🎯Late-stage developments AstraZeneca and Daiichi Sankyo US's Enhurtu (antibody-drug conjugate) shows significant improvement in Phase III trial for HER2-low breast cancer 👀 To watch Takeda Pharmaceuticals (formerly Nycomed), Astellas Pharma, and Sumitomo Dainippon Pharma form joint venture to support Japan-based biotech startups in research 📈 Access Icertis and NEWDIGS Initiative at Tufts Medical Center report suggests innovative approaches to gene therapy payment challenges, including value-based pricing, milestone-based rebates/warranties, subscription models or federal "carve-out" benefit Japan aims to approve new drugs within 3 weeks of Drug Subcommittee recommendation, but routine health economic evaluations may pose a conflict. Spain new guidance on economic evaluation for reimbursement of medicines provides greater clarity and transparency on assessment criteria used by AEMPS AIFA's CSE regulations define its role in providing mandatory and non-binding opinions on drugs, promoting access to innovative medicines, safe use, and recognizing added therapeutic value ✳️ Other: European Commission launches Critical Medicines Alliance to help prevent and address shortages of critical medicines 👉 What’s your favorite industry news? #pharma #biotech #pulsenews #curiousminds Hannah Kurth

Ncardia Launches Panel of Ready-to-Use Assays to Streamline Screening and Selection of New Therapeutic Candidates - https://lnkd.in/g6QEWYV7 New assays based on #iPSC help accelerate drug discovery by giving drug innovators in neurodegenerative diseases high quality data on a broad range of disease-relevant read-outs. September 10, 2024, LEIDEN, Netherlands — Ncardia, a leading human iPSC company, announced today the launch of a new panel of ready-to-use assays designed to streamline the screening and selection of new therapeutic candidates, with a specific focus on neurodegenerative diseases. The new panel features standardized, highly robust, and reproducible assays for Alzheimer’s Disease, Parkinson’s Disease, ALS/FTD (Amyotrophic Lateral Sclerosis/Frontotemporal Dementia), Neuroinflammation, and Neurotoxicity. Using human models and disease-relevant readouts, the assays ensure that the data obtained is highly translational, enhancing the potential for successful therapeutic outcomes in later stages of development. “Our new panel of assays represents a major step forward in accelerating drug discovery,” said Jeroen De Groot, PhD, Divisional CEO at Ncardia. “By offering assays that are already standardized, we eliminate much of the preliminary work, giving researchers more time to focus on what matters most – progressing fast and confidently with their most promising candidates.” The assays can be put to work in any stage of drug discovery – from target identification and validation to hit finding and lead optimization – and with any drug modality, including small molecule, gene therapy, RNA therapy and biologics. “Our shared commitment here is to lead the integration of human iPSC technologies into the drug discovery process,” said Shushant Jain, PhD, Director of Discovery Technology. “And a critical part of that is to give our clients timely, relevant efficacy and toxicity data on their drug candidates. That’s why the new panels include readouts for gene and protein expression, protein aggregation, lysosomal function, neuronal health and function, and the release of neurofilament light chain (NF-L), which is an important biomarker for neurodegeneration.” Due to the advanced development of the new assay panel, only minimal optimization steps are often required for Ncardia to configure custom assays to match a client’s exact needs. Read more at: https://lnkd.in/g6QEWYV7 #ipsc #drugdiscovery #drugdevelopment #neurodegenerative Timothy Keefe Jeff Bissen

Ncardia Launches Panel of Ready-to-Use Assays to Streamline Screening and Selection of New Therapeutic Candidates - https://lnkd.in/g-Yff4pB New assays based on #iPSC help accelerate drug discovery by giving drug innovators in neurodegenerative diseases high quality data on a broad range of disease-relevant read-outs. September 10, 2024, LEIDEN, Netherlands — Ncardia, a leading human iPSC company, announced today the launch of a new panel of ready-to-use assays designed to streamline the screening and selection of new therapeutic candidates, with a specific focus on neurodegenerative diseases. The new panel features standardized, highly robust, and reproducible assays for Alzheimer’s Disease, Parkinson’s Disease, ALS/FTD (Amyotrophic Lateral Sclerosis/Frontotemporal Dementia), Neuroinflammation, and Neurotoxicity. Using human models and disease-relevant readouts, the assays ensure that the data obtained is highly translational, enhancing the potential for successful therapeutic outcomes in later stages of development. “Our new panel of assays represents a major step forward in accelerating drug discovery,” said Jeroen De Groot, PhD, Divisional CEO at Ncardia. “By offering assays that are already standardized, we eliminate much of the preliminary work, giving researchers more time to focus on what matters most – progressing fast and confidently with their most promising candidates.” The assays can be put to work in any stage of drug discovery – from target identification and validation to hit finding and lead optimization – and with any drug modality, including small molecule, gene therapy, RNA therapy and biologics. “Our shared commitment here is to lead the integration of human iPSC technologies into the drug discovery process,” said Shushant Jain, PhD, Director of Discovery Technology. “And a critical part of that is to give our clients timely, relevant efficacy and toxicity data on their drug candidates. That’s why the new panels include readouts for gene and protein expression, protein aggregation, lysosomal function, neuronal health and function, and the release of neurofilament light chain (NF-L), which is an important biomarker for neurodegeneration.” Due to the advanced development of the new assay panel, only minimal optimization steps are often required for Ncardia to configure custom assays to match a client’s exact needs. Read more at: https://lnkd.in/g-Yff4pB #ipsc #drugdiscovery #drugdevelopment #neurodegenerative Timothy Keefe Jeff Bissen

𝗣𝗵𝗮𝗿𝗺𝗮𝗰𝗼𝗴𝗲𝗻𝗼𝗺𝗶𝗰𝘀 – 𝗧𝗮𝗶𝗹𝗼𝗿𝗲𝗱 𝗧𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁𝘀 𝗳𝗼𝗿 𝗕𝗲𝘁𝘁𝗲𝗿 𝗛𝗲𝗮𝗹𝘁𝗵! 🌟   Pharmacogenomics is revolutionizing healthcare by tailoring drug treatments to individual genetic profiles. This approach optimizes therapy, reduces adverse effects, and enhances patient outcomes.   𝗪𝗵𝗮𝘁 𝗶𝘀 𝗣𝗵𝗮𝗿𝗺𝗮𝗰𝗼𝗴𝗲𝗻𝗼𝗺𝗶𝗰𝘀? Pharmacogenomics studies how genes affect a person’s response to drugs. By understanding genetic variations, we can predict drug efficacy and safety, enabling personalized medicine.   𝗞𝗲𝘆 𝗔𝗱𝘃𝗮𝗻𝗰𝗲𝘀 Personalized Cancer Treatment 🎗️: Genetic profiling of tumors helps oncologists choose the most effective chemotherapy drugs, minimizing trial-and-error treatments. Targeted therapies based on genetic mutations are improving survival rates and quality of life for cancer patients   Tailored Medication Dosages 💊: Genetic testing for enzymes like CYP2D6 and CYP2C19 guides dosage adjustments for medications such as antidepressants and anticoagulants, reducing the risk of side effects and increasing efficacy   Improving Cardiovascular Treatment ❤️: Pharmacogenomic testing identifies patients who benefit from specific antihypertensive drugs, improving blood pressure control and reducing adverse reactions. This approach is particularly beneficial for drugs with narrow therapeutic indices, such as warfarin   𝗥𝗲𝗰𝗲𝗻𝘁 𝗔𝗱𝘃𝗮𝗻𝗰𝗲𝗺𝗲𝗻𝘁𝘀 CRISPR and Gene Editing: Advances in CRISPR technology enable precise gene modifications, offering potential treatments for genetic disorders by correcting defective genes directly   Biomarker Discovery: Identifying genetic markers that predict drug response is leading to the development of more effective and safer drugs, particularly in oncology and psychiatry   𝗖𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝗔𝗽𝗽𝗹𝗶𝗰𝗮𝘁𝗶𝗼𝗻𝘀 Pharmacogenomic testing is increasingly integrated into clinical practice, guiding treatment decisions and improving patient care. For example, the FDA has approved several pharmacogenomic tests that help determine the best treatment strategies for various conditions 𝗧𝗵𝗲 𝗙𝘂𝘁𝘂𝗿𝗲 𝗼𝗳 𝗣𝗵𝗮𝗿𝗺𝗮𝗰𝗼𝗴𝗲𝗻𝗼𝗺𝗶𝗰𝘀 As research progresses, pharmacogenomics will continue to expand, offering more personalized and effective treatments across a wide range of medical conditions. This field is set to become a cornerstone of precision medicine, ensuring that patients receive the right drug at the right dose, based on their genetic makeup.   For a deeper dive, check out this research paper: https://lnkd.in/eAvcnzxe   Stay tuned for Day 11 as we continue our series! See you tomorrow with a new topic.    #Pharmacogenomics #PersonalizedMedicine #GeneticTesting #PrecisionMedicine #Biotech #MedicalResearch #100Days100Learnings 💯📚

Enabling Access to Innovative and Orphan Medications with HEPA Hub 📢 How does your healthcare organization ensure access to cutting-edge treatments like gene therapies and oncology drugs? Accessing innovative therapies such as gene therapy treatments and orphan medications is vital for improving patient outcomes, especially in oncology and rare diseases. These therapies often require detailed economic evaluations, health technology assessments (HTA), and stringent compliance to be introduced effectively into healthcare systems. 🔍 How does HEPA Hub support the introduction of innovative therapies? HEPA Hub provides a comprehensive solution to simplify the access of these advanced treatments for all stakeholders—from pharmaceutical companies to clinical pharmacists and healthcare payers: • Economic Evaluation and Cost-Effectiveness Analysis (CEA): HEPA Hub enables pharmaceutical companies to conduct and submit detailed economic evaluations, proving the value of gene therapies, oncology drugs, and other high-cost medications. • Health Technology Assessments (HTA): Through our HTA modules, HEPA Hub supports the evaluation of innovative therapies, ensuring that they align with both clinical effectiveness and cost-benefit models essential for value-based care. • Facilitating Orphan Drug Access: HEPA Hub integrates real-world evidence (RWE) and outcome tracking, allowing for a comprehensive assessment of orphan drug performance, particularly for rare disease treatments. • Streamlined Access for Payers and Providers: By offering comprehensive insights through RWE and market access data, HEPA Hub ensures that healthcare providers and payers can make informed decisions about the introduction of costly, life-saving treatments into their systems. 💡 Why this matters: Innovative therapies like gene therapy and advanced oncology drugs offer transformative potential for patients, but their high costs and complex reimbursement processes can delay access. HEPA Hub simplifies these processes, ensuring that patients benefit from the latest medical innovations as soon as possible. 🚀 Join our pilot program and discover how HEPA Hub can streamline the process of bringing innovative therapies to healthcare systems, making your organization a leader in medical advancements. https://lnkd.in/dkqptTS7 #genetgerapy #Oncology #OrphanDrugs #HealthTechnologyAssessment #Pharmacoeconomics #RWE #MarketAccess #ValueBasedCare #HealthcareInnovation #PatientCare #MedicalAdvancements #HEPAHub

Everything you missed in #Pharma & #Biotech 🦠🔬 ✅ Approvals FDA approves Adaptimmune’s Tecelra (afamitresgene autoleucel), first gene therapy for metastatic synovial sarcoma Alpha Cognition, Inc.’s Zunveyl, an oral therapy for mild-to-moderate Alzheimer's disease addressing gastrointestinal side effects associated with galantamine, approved by FDA SUN PHARMA's Leqselvi, an oral JAK inhibitor deuruxolitinib, approved by FDA for alopecia areata 💸 Deals Boehringer Ingelheim expands oncology pipeline with novel immune checkpoint inhibitors through acquisition of Nerio Therapeutics for up to $1.3 billion GSK and Flagship Pioneering partner to identify and develop up to 10 novel medicines and vaccines, focusing initially on respiratory and immunology (methylphenidate hydrochloride) Otsuka Pharmaceutical Companies (U.S.) to acquire Jnana Therapeutics for over $1.1 billion to expand its drug pipeline and research base in the Boston area Apogee Therapeutics and Spyre Therapeutics founder launch­ an­oth­er au­toim­mune biotech, Jade Biosciences, with $80M 🎯Late-stage developments AstraZeneca's fixed-duration Calquence (acalabrutinib) combination with obinutuzumab shows shown improved progression-free survival in phase 3 trials for first-line chronic lymphocytic leukemia (CLL) Bayer's finerenone meets primary endpoint in phase 3 study of patients with heart failure with mildly reduced or preserved ejection fraction Redhill’s RHB-104 demonstrates positive phase 3 data in Crohn's Disease FibroGen, Inc.'s drug pamrevlumab fails to meet primary endpoints in two late stage trials in pancreativc cancer and metastatic pancreatic ductal adenocarcinoma Agios Pharmaceuticals' late-stage Pyrukynd trial in children with rare blood disorder fails to meet its primary endpoint Vertex Pharmaceuticals receives FDA priority review for suzetrigine, a non-opioid pain drug for moderate to severe acute pain, with decision expected by January 30, 2025 ✳️ Other The European Union's AI Act (2024/1689) takes effect, introducing risk-based classification of AI systems, with stringent requirements for high-risk AI to ensure safety, ethics, and transparency, and enhanced human oversight to maintain informed decision-making, positioning Europe as a leader in ethical AI regulation Synchron announces first successful use of Apple Vision Pro with an implantable brain-computer interface, allowing a patient with ALS to control the device hands-free through brain signals 👉 What’s your favorite industry news? #pharma #biotech #pulsenews #curiousminds #techbio Hannah Kurth Patricia Machado Tamsin Lacourte Alexandra SILVAIN

𝐀 𝐂𝐨𝐦𝐩𝐥𝐞𝐭𝐞 𝐆𝐮𝐢𝐝𝐞 𝐎𝐧 𝐊𝐧𝐨𝐜𝐤𝐨𝐮𝐭 𝐌𝐢𝐜𝐞 𝐌𝐨𝐝𝐞𝐥 IndustryARC™ updated the market research study on 𝐊𝐧𝐨𝐜𝐤𝐨𝐮𝐭 𝐌𝐢𝐜𝐞 𝐌𝐨𝐝𝐞𝐥 𝐌𝐚𝐫𝐤𝐞𝐭 Size is forecast to reach $ 5386.2 Million by 2030, at a CAGR of 5.40% during forecast period 2024-2030. 👉 𝐃𝐨𝐰𝐧𝐥𝐨𝐚𝐝 𝐑𝐞𝐩𝐨𝐫𝐭 𝐒𝐚𝐦𝐩𝐥𝐞 @ https://lnkd.in/gHsvAD-u 𝐇𝐞𝐫𝐞 𝐚𝐫𝐞 𝐬𝐨𝐦𝐞 𝐤𝐞𝐲 𝐟𝐢𝐧𝐝𝐢𝐧𝐠𝐬 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐫𝐞𝐩𝐨𝐫𝐭 𝟏. 𝐆𝐫𝐨𝐰𝐢𝐧𝐠 𝐔𝐬𝐞 𝐢𝐧 𝐃𝐫𝐮𝐠 𝐃𝐢𝐬𝐜𝐨𝐯𝐞𝐫𝐲 𝐚𝐧𝐝 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭: Knockout mice models are essential tools in understanding gene function and disease mechanisms, making them invaluable in drug discovery and preclinical testing. Pharmaceutical companies use these models to study the effects of potential drugs on specific genetic traits, leading to more targeted and effective therapies. The increasing focus on personalized medicine is further driving this trend. 𝟐. 𝐀𝐝𝐯𝐚𝐧𝐜𝐞𝐦𝐞𝐧𝐭𝐬 𝐢𝐧 𝐂𝐑𝐈𝐒𝐏𝐑-𝐂𝐚𝐬𝟗 𝐆𝐞𝐧𝐞 𝐄𝐝𝐢𝐭𝐢𝐧𝐠 𝐓𝐞𝐜𝐡𝐧𝐨𝐥𝐨𝐠𝐲: The emergence of CRISPR-Cas9 has revolutionized the creation of knockout mice models. This technology allows for more precise, efficient, and faster gene editing, reducing the time and cost associated with developing genetically modified mice. The widespread adoption of CRISPR has accelerated research in genetics, oncology, and neurobiology. 𝟑. 𝐑𝐢𝐬𝐢𝐧𝐠 𝐅𝐨𝐜𝐮𝐬 𝐨𝐧 𝐑𝐞𝐬𝐞𝐚𝐫𝐜𝐡 𝐟𝐨𝐫 𝐑𝐚𝐫𝐞 𝐚𝐧𝐝 𝐆𝐞𝐧𝐞𝐭𝐢𝐜 𝐃𝐢𝐬𝐞𝐚𝐬𝐞𝐬: Knockout mice models are increasingly used to study rare and genetic disorders, providing insights into disease mechanisms and potential treatments. With more funding and attention directed toward understanding rare diseases, these models play a crucial role in identifying therapeutic targets and testing experimental drugs. 𝟒. 𝐄𝐱𝐩𝐚𝐧𝐬𝐢𝐨𝐧 𝐢𝐧 𝐈𝐦𝐦𝐮𝐧𝐨𝐥𝐨𝐠𝐲 𝐚𝐧𝐝 𝐂𝐚𝐧𝐜𝐞𝐫 𝐑𝐞𝐬𝐞𝐚𝐫𝐜𝐡: There is significant growth in the use of knockout mice models for immunology and oncology studies. Researchers utilize these models to investigate immune system functions, cancer progression, and the effects of immunotherapies. The development of knockout mice that mimic human immune systems is particularly beneficial for studying cancer immunotherapies, autoimmune disorders, and infectious diseases. 👉 𝐆𝐞𝐭 𝐌𝐨𝐫𝐞 𝐈𝐧𝐟𝐨 @ https://lnkd.in/gJraR2ek 𝐌𝐚𝐣𝐨𝐫 𝐊𝐞𝐲 𝐏𝐥𝐚𝐲𝐞𝐫𝐬 𝐈𝐧𝐜𝐥𝐮𝐝𝐞:Eli Lilly and Company | UnitedHealth Group Johnson & Johnson | Abbott | Merck Group | Thermo Fisher Scientific | Danaher Corporation | Amgen | Pfizer | Stryker | Boston Scientific | Vertex Pharmaceuticals | Medtronic | Gilead Sciences | GE HealthCare | #KnockoutMice #GeneEditing #CRISPRTechnology #GeneticResearch #DrugDiscovery #BiotechInnovation #CancerResearch #PreclinicalStudies #Immunology #BiomedicalResearch

From Potential to Value: Carving a Slice of the CGT ‘PIE' In early 2019, the FDA predicted that, by 2025, 10 to 20 cell and gene therapies (CGTs) will be approved per year. As we approach 2025, the industry is on track to hit the projection. There are 10+regulatory decisions in the US this year, and with about 2,000 CGT clinical trials underway, the figure may climb in the coming years. As the positive trajectory offers great potential, the path from discovery and development to commercialization is complex and lined with challenges impacting product success and patient access.                                CGTs introduce unique challenges, including those related to patient access, regulatory requirements, and market access. Payors face several questions when evaluating CGT products, including how to balance the potential long-term clinical value with the cost of the therapy, and the uncertainty, regarding long-term durability at the time of regulatory approval; questions which demand early payor-engagement and demonstration of the product’s value. Companies can initiate that process by engaging in market research with payors in a variety of ways to gain feedback on evidence needs, value proposition, or launch sequences for multi-indication brands. The output of the market research will help inform clinical trial design, endpoint selection and commercial planning, if the research is conducted early enough in the product lifecycle. Following the initial communication, developers should engage in pre-approval information exchange (PIE) with payors, allowing them to share information, such as company overview, product and indication overview, patient use projections, clinical study results, disease overview, and pricing information. 60% of payers say they prefer information on CGT therapies 6 months prior to the anticipated regulatory approval, with 16% saying they prefer to receive the information more than a year ahead. The insights gained from the early conversations with payers may help companies develop evidence that shows the therapy’s potential benefits and justifies the value, while addressing concerns and priorities of key stakeholders, such as: Clinical evidence: Robust clinical trial data with meaningful and measurable endpoints, proving the therapy’s safety, and efficacy. Cost-effectiveness analysis: Compare the therapy’s cost to standard of care and incorporate quality-adjusted life year estimates. Patient perspective: Highlight the therapy’s impact on patient quality of life and disease burden. Biopharma companies should continue to engage payers even after commercial launch, sharing updated data and information from post-marketing surveillance and real-world evidence studies to further support the product’s value. #Pharma #Marketaccess #Callandgenetherapy #Lifecyclemanagement Visit us on https://lnkd.in/d5tuZczF or look for more information on https://lnkd.in/dytEHHne