Simon-Kucher’s Post

What are the key considerations pharmaceutical companies need to address before launching a direct-to-patient offer, particularly regarding product suitability, capability assessment, and ensuring quality, governance, and credibility in healthcare delivery? https://lnkd.in/edpp9gfs

Our latest newsletter is ready! Check out our newsletter for the latest news, developments, and regulatory updates. This week's issue includes the publication of a new EU GLP recommendation paper, the launch of the WHO's MeDevIS platform to enhance access to medical technologies and devices, and updates from the #FDA and MHRA. https://lnkd.in/d2R-f9Rr To receive our weekly newsletter directly to your inbox, just send an email to info@towermains.com with the subject “Weekly Newsletter Please”. #medicaldevices #regulatoryupdates #MHRA #pharmaceutical #GLP

Stories making headlines today in the biopharma industry: Johnson & Johnson has announced that the final pricing set under the Inflation Reduction Act (IRA) will not hinder its long-term sales projections. Jennifer Taubert, Worldwide Chairman of Innovative Medicine, confirmed that the company had already factored these figures into last year's financial guidance. This positions J&J as yet another pharmaceutical leader demonstrating resilience through strategic investment and pipeline management. The FDA has launched a Rare Disease Innovation Hub, intended to unify policies and develop new evaluation methods for rare disease treatments. Led by CBER Director Peter Marks and CDER Director Patrizia Cavazzoni, the hub aims to streamline regulatory pathways and provide consistent guidance to companies working on rare disease therapies. Merck has asked the US District Court for the District of Columbia to expedite its decision regarding its challenge to the Inflation Reduction Act. The company is seeking a swift resolution to allow sufficient time for appeals before the IRA's full enactment in 2026. This follows a series of government victories in similar cases against other major pharmaceutical companies such as Bristol Myers Squibb and AstraZeneca. Executives from Cigna, CVS Health, and UnitedHealth Group are set to testify before Congress on July 23. Organized by the House Committee on Oversight and Accountability, the hearing follows a critical Federal Trade Commission report and will focus on how Pharmacy Benefit Managers (PBMs) interact with drugmakers and pharmacies. These three companies collectively hold 79% of the PBM market. London-based hVIVO is actively seeking acquisitions of small contract research organizations (CROs) to meet its £100 million revenue target by 2028. CEO Yamin Khan indicated that the company is particularly interested in UK-based CROs with Phase 1 trial facilities. hVIVO specializes in human challenge trials to assess vaccine efficacy and safety. Gilead Sciences has announced that Chief Medical Officer Merdad Parsey will depart by the first quarter of 2025. Parsey's departure comes amid challenges in Gilead's pipeline and cancer drug development. The news has prompted a slight rise in Gilead's stock, which has been down 10% since the beginning of the year. Analysts suggest this transition could be an opportunity to rejuvenate Gilead's R&D efforts. #pharma #biotech #topstories #biodatastudio

In a recent article from BioSpace, Senator Bernie Sanders criticized Novo Nordisk for the high price of its #diabetes drug #Ozempic and weight-loss medication #Wegovy. However, professionals in the pharmaceutical and biotech industries should be aware of the complex factors that contribute to drug pricing and the potential negative consequences of political pressure on the industry. The process of developing a new drug is prohibitively costly, with the estimated expense of bringing one to market falling between $1.3 and $2.6 billion. This figure encompasses the costs associated with research and development, clinical trials, and navigating the process of regulatory approval. To illustrate, in 2021, the pharmaceutical industry spent an immense $102 billion on research and development alone. Medications such as Ozempic and Wegovy carry high price tags, which mirror the substantial investments made to bring these drugs to fruition. It's important to note that the journey from lab to pharmacy is fraught with challenges, with only about 12% of drugs that embark on clinical trials ultimately receiving #FDA approval. The pharmaceutical industry functions within a free market where prices are set by supply and demand. It’s tempting to compare drug prices in the US with those abroad, but it’s key to realize that other nations often have price controls or obtain discounted rates, which are not present in the US. Moreover, the US market makes up a substantial share of the global pharmaceutical sales, giving companies the financial ability to pioneer new treatments. In 2021, the US #pharmaceutical industry generated $550 billion in revenue, claiming 48% of the worldwide market. Political pressure on the pharmaceutical industry might negatively impact innovation and the availability of new treatments. If companies are pushed to slash prices, they could scale back investment in research and development of new medicines, potentially adversely affecting patients. Furthermore, price caps could cause shortages or delays in accessing treatments. The value of the US pharmaceutical market reached $527 billion in 2022, and with a projected compound annual growth rate of 3.67% from 2023 to 2030, it showcases how pricing plays a crucial role in continuing the growth and progress of the industry. In conclusion, although the steep prices of drugs like Ozempic and Wegovy raise legitimate concerns, it’s vital to acknowledge the intricate factors that influence drug pricing and the potential drawbacks of political interference. Those in the pharmaceutical and #biotech sectors should strive for policies that balance innovation and treatment access while also considering affordability issues.

Osteoporosis Treatment Advancements: Henlius and Organon Achieve European Medicines Agency Validation for HLX14 Biosimilar Henlius and Organon have reached a significant milestone with the European Medicines Agency's (EMA) validation of the Marketing Authorization Applications (MAAs) for HLX14, a biosimilar to Prolia and Xgeva (denosumab), aimed at treating osteoporosis. This development could expand treatment options for postmenopausal women at high fracture risk. In 2019, approximately 32 million Europeans aged 50 or older had osteoporosis, with 25.5 million being women. HLX14's validation follows a comprehensive Phase III trial assessing its efficacy and safety against the European Union-sourced reference denosumab. Henlius and Organon's 2022 licensing agreement allows Organon to commercialize HLX14 in key markets, excluding China. For more details please click the link! https://lnkd.in/dhPdCfdd #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

Urgent Call: Systemic Changes Needed Across Drug Supply Chain to Save Lives Systemic changes across the drug supply chain are deemed necessary to prevent further crises and save lives. Discussed at the National Comprehensive Cancer Network® (NCCN®) Annual Conference, there are pressing issues of drug shortages in oncology care. Panelists highlighted the significant impact on patient care and explored potential solutions, such as multi-sourcing and fair distribution programs, alongside legislative initiatives for transparency and manufacturing resilience. 1TCC® helps businesses navigate disruptions to the global supply chain. With 1TCC®, a fully integrated VMI platform, businesses can navigate complexities and maintain unparalleled visibility without disrupting existing trade relationships. 1TCC® streamlines every facet of your supply chain and inventory, freeing up working capital, and paving the way for long-term success through delivering capital-efficient supply chains™. 1TCC®, delivering capital-efficient supply chains™. Learn more at 1tcc.com. https://lnkd.in/g3kKYBTa #SupplyChainDisruption #CrisisManagement #SupplyChainOptimization #BusinessContinuity #SupplyChainVisibility #LogisticsSolutions #InventoryVisibility #TCC #TradeCapitalCorporation #1TCC #1TCCPlatform #BusinessResilience #LogisticsInnovation #WorkingCapitalManagement #InventoryManagement #Uncertainty #Challenges #Resiliency #CapitalEfficientSupplyChains #IndustryLeaders #TradeFinance #Leadership #BusinessSuccess #GlobalTrade #EconomicResilience #VMI #Healthcare #Pharmaceutical #PharmaceuticalShortages

Expanded Access Programs (EAPs), also known as Compassionate Use Programs (CUPs), allow patients and physicians to use investigational drugs outside of clinical trials. EAPs can be an important treatment option for patients with rare diseases or cancers. In a recent Clinical Leader article, Christopher Ohms, Executive Director, Supply Chain at Rigel Pharmaceuticals, highlights the differences between Investigator Sponsored Trials and EAP and focuses on how vital both are in providing patients with access to investigational therapies and advancing medical research.  WEP Clinical has been working with pharmaceutical companies to provide Early Access Programs on a global basis, for over a decade. Through our portfolio of programs, we currently supply treatment to patients suffering from a variety of rare diseases and cancers. #WEPClinical #PatientAccess #ExpandedAccess

Exciting news from Ferrer, a B Corp-certified pharmaceutical company! They have launched the Phase II clinical trial, PROSPER, to evaluate FNP-223's safety and efficacy in slowing the progression of Progressive Supranuclear Palsy (PSP). PROSPER is a rigorous randomized, double-blind, placebo-controlled trial with up to 220 participants spanning sites in the US, UK, and the EU. PSP is a rare neurodegenerative disease with no known cure, characterized by speech difficulties, imbalance, cognitive impairments, and eye control issues. FNP-223 shows promise in halting the abnormal accumulation of tau protein in specific brain regions associated with PSP. Ferrer aims to prove the drug's safety and effectiveness in PSP patients to potentially decelerate the disease's advancement. Jorge Cúneo, Ferrer's Chief Medical Officer, underlines the company's dedication to enhancing the lives of individuals with rare diseases. Patient organizations like CurePSP and PSPA UK have been instrumental in shaping the PROSPER trial. Kristophe Diaz, Executive Director and Chief Science Officer of CurePSP, stresses the trial's significance in seeking treatments for PSP, an ailment currently without a cure. Stay informed about Ferrer's progress in the Phase II trial by visiting the link: https://lnkd.in/dfmZDDnn Tania Nadal Matthew Pepe SHRM-CP Alba Soler Conangla

Exciting news from Ionis Pharmaceuticals, Inc. and AstraZeneca today! The U.S. FDA has granted Fast Track designation to eplontersen, an investigational therapy for the treatment of ATTR-CM (transthyretin amyloid cardiomyopathy) in adults. ATTR-CM is an underdiagnosed and potentially fatal disease that affects the heart and is associated with heart failure. Eugene Schneider, M.D. notes, "Receiving Fast Track designation from the FDA reinforces our belief that eplontersen has the potential to be a transformational treatment for patients with ATTR-CM, which remains a progressive and fatal condition for hundreds of thousands of people worldwide despite available treatment options." The Fast Track designation is designed to facilitate the development and expedite the review of drugs that demonstrate the potential to treat serious conditions and fill an unmet need. Today's news provides hope and optimism to those faced with ATTR-CM. #pharma #hearthealth #innovation

Clinical Trials Market size is estimated to grow at 5.4% CAGR from 2024 to 2032. To know more, visit :- https://lnkd.in/djKTtyJx Key players are :- Clinipace Eli Lilly and Company ICON plc IQVIA Medpace . . . . #ClinicalTrials #ClinicalResearch #Pharmaceuticals #DrugDevelopment #MedicalResearch #ClinicalStudies #ResearchAndDevelopment #Biotechnology #Pharma #HealthcareResearch #NewTreatments #ClinicalTrialManagement #MedicalInnovation #ClinicalTrialsMarket #Healthcare #ResearchTrials #MedicalStudies #DrugTesting #ClinicalTrialServices #ClinicalData