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Ramesh Arjunji, VP at Nanoscope Therapeutics, outlined a roadmap for generating real-world evidence (RWE) to secure gene therapy reimbursement at Advanced Therapies USA 2025. Key steps include defining the value proposition, evaluating patient journeys, addressing evidence gaps, stakeholder alignment, and streamlining value communication. Arjunji emphasized tailoring RWE to show durability and differentiation of therapies like gene treatments for conditions like Hemophilia ATo maximize value, Arjunji urged focusing on the evidence needed to reflect cost savings, extended quality of life, and population-specific benchmarks. He stressed leveraging diverse data sources like health records and wearable tech, while collaborating with stakeholders, policy makers, and advocacy groups. Evidence gaps should be prioritized by impact and feasibility, with adaptive planning to address payer concerns and highlight curative advances effectivelyArjunji shared strategies to break down access barriers, urging partnerships with patient advocacy groups for newborn screenings and unmet-need validation. He recommended a proactive value communication approach using tools like Core Value Dossiers, field training, and pre-approval payer dialogues. Citing ICER’s past reviews, he advised companies to prepare for payer assessments and emphasized patient-centered transparency for better access to therapies like vision loss treatments
Andrew York, Executive Director of Maryland’s Prescription Drug Affordability Board, discussed U.S. payer trends for cell and gene therapies at Advanced Therapies USA 2025. Despite fears of runaway costs, payers have integrated these treatments more steadily than expected. Key concerns remain high upfront costs and uncertainty about long-term benefits, as well as how to spread costs fairly across payers as patients move between plansThe federal CGT Access Model, focusing on Sickle Cell Disease, has expanded to 33 states and uses outcomes-based deals with manufacturers—an approach York sees as promising. However, many federal pricing initiatives like the 340B Rebate Model or tariffs on pharmaceuticals don't directly affect cell and gene therapies. Medicaid, commercial payers, and self-insured employers are using methods like stop-loss insurance and amortization to handle the risks of these pricey treatmentsPayer innovation on reimbursement models lags due to inertia and limited claims data. Strategies like outcomes-based agreements sound great but are tough to implement. Advice to pharma: support payers in overcoming barriers
Members of the Pharmaceutical Research and Manufacturers of America (PhRMA), including Pfizer CEO Albert Bourla, met with Japanese officials on Nov 18 to address Japan's drug pricing policies. They highlighted concerns over low initial drug prices, frequent price cuts, and the impact of the U.S.'s Most Favored Nation (MFN) pricing policy on Japan's pharmaceutical sectorAt the meeting, PhRMA called for reform of Japan’s system to ensure it does not continue to decline in competitiveness. Specifically, PhRMA urged the Japanese government to maintain drug prices during patent periods, limit price cuts, improve initial pricing for new drugs, and develop a national pharma innovation strategy for global competitivenessCompanies have already seen impacts on products in Japan due to MFN. All 10 companies recently surveyed by the Japanese arm of the European Federation of Pharmaceutical Industries and Associations (EFPIA) responded that they believe MFN will impact their global pricing strategy
Elizabeth White from Orphan Therapeutics Accelerator discussed alternative approaches to fund, develop, and commercialize gene therapies for rare diseases at Advanced Therapies USA 2025. The non-profit biotech firm focuses on treatments without profitable pathways and leverages a network of specialized service providers, Orphan ClinDevNet, to lower costs and streamline progressTackling ultra-rare drug challenges, White highlighted issues like high costs, low patient numbers, inaccessible genetic testing, and shrinking government funding. She also noted a shift in intellectual property back to academic centers and the large number of abandoned drug programs, leaving gaps that Orphan Therapeutics aims to fill by advancing and commercializing these neglected assetsThe organization is building a comprehensive database that uses AI to rank rare disease gene therapy assets. This tool will help connect investors with suitable opportunities. Orphan Therapeutics also works on creative commercialization strategies, including spin-offs, direct-from-academia deals, and partnerships, to bring overlooked therapies to market efficiently
Cell and gene therapies are booming but still face hurdles like regulatory complexity, as discussed at Advanced Therapies USA 2025. Experts advocated for a more flexible FDA review process, especially for rare conditions. Industry insiders highlighted significant progress despite the field’s early stageCompanies like Ocugen and Atara work through challenges like FDA applications and manufacturing issues, while regulators show openness to single-arm trials for rare pediatric therapies. Partnerships with pharma giants, like Bayer’s backing of AskBio, remain crucial for development and fundingLeaders see promise in the market’s growth but admit the broader industry needs to understand cell and gene therapies’ transformative potential. While advanced therapies like Roche’s Luxturna set precedents, each product demands unique strategies
Negotiators are entering the most sensitive stage of talks on the EU’s pharmaceutical legislative package, with a single issue now overshadowing all others, whether companies should be legally required to place medicines on the market in every Member State that requests them, under threat of sanctionsAfter months of technical drafting and two high-level political rounds with little movement, Diario Farma reports that sources familiar with the trilogues say the central dispute is no longer innovation incentives, data-protection periods or exclusivity vouchers for antibiotics. Instead, the deadlock hinges on a proposed obligation for manufacturers to launch products across the entire EU and ensure supply, a requirement that has split institutions and frozen progress across the wider reformEarlier this month, Adam Jarubas, Chair of the European Parliament’s Committee on Environment, Public Health and Food Safety (SANT), emphasized that the Danish Presidency seems “determined to reach an agreement by the end of this year. It will be difficult, but if the Presidency shows the necessary flexibility, it should still be possible”
The UK pharma industry and government have agreed to extend the deadline for companies to leave the 2024 Voluntary Scheme for Branded Medicines Pricing (VPAG) to 16 December 2025The extension supersedes both previous extensions agreed in September and OctoberCompanies that notify their departure before the new November deadline will not be able to reverse their decision. Those that do not submit notice must remain in the scheme throughout 2026. Any companies exiting VPAG will instead move onto the statutory scheme for branded medicines
The Centers for Medicare & Medicaid Services (CMS) has updated the Request for Applications (RFA) document for its recently announced GENEROUS drug pricing model, providing new details regarding its reference basket methodologyThe latest language included in the RFA clarifies that the benchmark used to calculate the Most Favored Nation (MFN) price for a COD will be the second lowest country-specific manufacturer-reported net price, adjusted by gross domestic product per capita using a purchasing power parity methodNAVLIN Daily will continue to monitor and report on any additional changes to the model as they develop
Industry leaders met yesterday at the FT Global Pharma and Biotech Summit in London to discuss dramatically changing policies on both sides of the Atlantic, from Europe’s new pharmaceutical strategy to the U.S.' Most Favored Nation (MFN) pricingWhile the room was packed out with senior industry representatives and payers who all harbor thoughts on these topics, those on stage kept their cards close to their chestsRead on to learn NAVLIN Daily’s learnings from the first day of the Summit
We’re back at the Financial Times’ (FT) Global Pharma and Biotech Summit in London for day two, where Dr Sam Roberts, Chief Executive Officer (CEO) of the National Institute for Health and Care Excellence (NICE), kicked off the day talking about NICE’s transformation journeyThe outgoing CEO, who will be leaving at the end of 2025, addressed the Agency’s closer collaboration with the Medicines and Healthcare products Regulatory Agency (MHRA), the premise of the Voluntary Pricing and Access Scheme (VPAG), and the controversy surrounding the Enhertu (trastuzumab deruxtecan) decision under NICE’s new severity modifierDr Roberts confirmed that the joint working pathway between NICE and the MHRA launched on 1 October, marking what she described as “20 years” of ambition finally realized. “We’ve got the first couple of medicines that are part of that pathway where we’re constantly sharing data with MHRA,” she said. “Our aim is to have NICE advice and MHRA license as close together as possible, ideally on the same day.”
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