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Speaking at the World Orphan Drug Congress (WODC) 2024 conference, Mark Trusheim, Strategic Director, MIT NEWDIGS, discussed the importance of innovative payment models for securing payer coverage of therapies for rare diseasesTrusheim noted that while collectively, rare disease treatments make up just a small fraction of overall health care spending in the U.S., their high upfront costs can present a financial challenge to payers. Small payers and self-insured employers are especially vulnerableThe NEWDIGS initiative identified five financing solutions designed to address concerns around covering durable therapies. These include (1) short-term, milestone-based contracts; (2) multi-year performance-based annuities; (3) warranty models; (4) stop-loss/reinsurance and subscription/Netflix models; and (5) orphan reinsurer and benefit manager (ORBM) and risk pools
At the World Orphan Drug Congress 2024 conference in Boston, Ellen Cho, Director, Specialty Programs, Direct Relief, explained that her organization provides access to medicines in countries with limited or no access due to emergencies, disasters, and povertyShe said low- and middle-income countries (LMICs) face the same barriers to access as the U.S. and Europe but to an amplified extent Improving access in LMICs means spreading disease awareness, training physicians, fixing transportation challenges, and partnering across companies and governments to secure long-term supply deals
At the World Orphan Drug Congress 2024 conference in Boston, Dean Suhr, President, MLD Foundation, shared signs of confidence among HTA agencies that have assessed Orchard Therapeutics' Libmeldy, a treatment for metachromatic leukodystrophy (MLD)Mirroring Suhr's optimism, Kristin Viswanathan Wolff, Vice President, Market Access, bluebird bio, said she has been “pleasantly surprised” at payers' increasing willingness to engage early regarding the reimbursement of novel therapies Wolff discussed the U.S.-specific problem of frequent patient migration to different insurers: “The biggest hurdle we have found is portability in commercial insurance and patients switching insurers. Employers are not set up to deal with these issues – so we're seeing middlemen who act on behalf of the employer but don't have a touchpoint with the patient”
At the World Orphan Drug Congress 2024 conference in Boston, Dr. Michael Salgaller from the National Institutes of Health (NIH) encouraged drug companies to take advantage of the Institutes' licensing and collaboration opportunities“There are many misconceptions about NIH,” noted Salgaller, “We don't just do basic research – we also do translational and clinical research. We work with industry in addition to academia, and we partner internationally”NIH has co-developed many blockbuster drugs like Pfizers' rare skin cancer treatment Bavencio (avelumab) and Janssen's depression drug Spravato (esketamine)
At the World Orphan Drug Congress 2024 conference in Boston, Dr. Michael Sherman, Investor, RA Capital, encouraged biopharma companies to partner with a consulting firm before Phase 1, to begin thinking about reimbursement possibilities and challengesOn the unexpectedly low uptake of Biomarin's Roctavian, Sherman speculated on contributing factors like fear around adverse events and a lack of alternatives for sensitized patientsSherman also critiqued Prescription Drug Affordability Boards (PDAB), calling them “highly unworkable” because U.S. state borders are “porous”
At Reuters Pharma Europe 2024, speakers agreed that while the EU’s pharmaceutical legislation revision is a positive step, it does still present uncertainties for investors and other issues relating to overall impactThey cited the significant disparities in patient wait times for new medicines in different EU countries as a major concern and EFPIA's Andy Powrie-Smith implored key actors, like EU institutions, regulators and patients, to collaborate and address these access issues rather than using innovation legislation as a solution. He also expressed disappointment over potential bureaucracy in the Health Technology Assessment (HTA) regulationNicola Bedlington, former Secretary General of the European Patients' Forum, and Sandoz's Europe President, Rebecca Guntern, discussed the idea of European-level joint procurement to tackle shortages and access problems and Guntern further highlighted the significance of generic competition in reducing costs, stressing the need for policies that focus on value rather than just price
Speaking at Retuers Pharma Europe 2024, Elena Petelos of HTAi and EUPHA highlighted the importance of trust for the upcoming EU Health Technology Assessment (HTA) regulationShe emphasized the need for teamwork among all players in the pharma sector, and believes in enhancing governance for elements like real-world evidence (RWE)The European Commission is currently seeking public opinion on the draft related to the European Regulation on Health Technology Assessment (EUHTAR), which is expected to take effect from January 12, 2025
Cathrin Schäfer, Head of Market Access Europe at BeiGene, spoke about the recent amendments to the EU pharma legislation at Reuters Pharma EuropeSchäfer praised the Commission's recent recognition that simultaneous product launch across all EU member states poses significant challenges due to varying systems and requirements. Still, she stressed the need to support innovation and avoid making the environment in Europe more challenging for pharmaceutical companies Despite the recent revisions to the proposals in the legislation review, there is still concern that the reforms might stifle innovation and investment in Europe - the European Federation of Pharmaceutical Industries and Associations (EFPIA) previously warned that if the legislation review goes ahead, Europe could lose 8% of its pharmaceutical innovation in the next 15 years
Speaking at Reuters Pharma 2024, Stefan Oelrich, a board member of Bayer AG, emphasized the need for the sector to work together to cope with future challenges such as legislation changes, increased innovation, and ageing populations. He stressed that only by working in unison, much like a rowing team, can the pharmaceutical industry fully leverage the potential of innovationImportantly, Oelrich noted a shift in attitudes among European Union policymakers towards the pharmaceutical sector, particularly since COVID-19. He specifically welcomed the recognition of pharma’s feedback by the EU Commission in the most recent review of pharma legislation, but expressed concern that the final policy change still does not sufficiently foster innovationHe also touched on room for collaboration to ensure pharma can be efficient enough to make the most of the innovation coming through the pipeline
Monica Shaw, SVP of European Markets at Bristol Myers Squibb, has emphasized the importance of collaboration across the pharmaceutical sector, governments, and health bodies to ensure patient access to medicines at Reuters Pharma EuropeShe believes that the recent changes to the EU pharmaceutical legislation and the incoming Health Technology Assessment (HTA) regulation bring both opportunities and challenges - the legislation review offers a chance to improve the regulatory environment in favor of innovation. However, it is critical that healthcare is seen as an investment by policy holdersCollaboration, she notes, could be the key to overcoming these hurdles, but the "devil is in the detail" as it requires pragmatic implementation
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