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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 (WODC) USA 2024, panelists emphasized that Europe remains an attractive market, but companies looking to launch rare disease therapies in this region must consider additional hurdles in order to succeed Speakers emphasized the importance of demonstrating a product's value to European payers, highlighting the benefit of early engagement with stakeholders and adaptability to different pricing and access conditions in European countries. These strategies are especially important with legislative and regulatory changes on the horizon Professor Matthias Schönermark, MD, Ph, Managing Director, SKC Beratungsgesellschaft mbH, highlighted that there is a misconception among developers that European payers do not understand value. He argued that this is not true; however, they want proof of value. “Some companies are little bit naïve coming to Europe because they do not understand this,” Dr. Schönermark commented. “It is important to have the right study design and find the right comparator"
Navigating the U.S. health system is challenging for all patients facing a rare disease diagnosis; however, the process can be especially daunting for patients and families covered by state Medicaid programs. Experts at the World Orphan Drug Congress (WODC) USA 2024 highlighted access challenges posed by Medicaid and discussed solutions rare disease stakeholders can pursue to overcome some of these barriersAccording to data collected by the National Organization for Rare Diseases (NORD), 39% of rare patients travel over 60 miles for care, while 17% of patients and their families have moved or considered relocating to be closer to care. Further, current Medicaid regulations require clinicians to be screened and enrolled in each patient’s home state Medicaid program to receive reimbursement. Funding is also a major issue in Medicaid, with state agencies often facing significant budget constraintsPanelists at WODC emphasized the need to work closely with Medicaid agencies and gain a better understanding of the challenges within the program. The Accelerating Kids’ Access to Care Act could help mitigate issues with obtaining cross-state care, according to one panelist. Other speakers emphasized the need for a proactive approach, suggesting that solutions should be in place before new products get approved
Panelists at the World Orphan Drug Congress (WODC) USA 2024 discussed the challenges rare disease patients face in accessing treatments even after they have been approved by regulatory bodiesThey highlighted that regulatory approval is often just the first hurdle in the road to access, with payer coverage and successful adoption presenting further obstacles for patients. All of the panelists noted that payers often view drugs approved via accelerated pathways as "experimental." The panelists stressed that just because a drug is approved through a flexible pathway does not mean it has not met rigorous safety and efficacy standardsThe panelists also emphasized the need for greater adoption of screening and genetic testing, in order to shorten the "diagnostic odysseys" many rare disease patients face. They also called for including pediatric patients earlier in the clinical research process
At the World Orphan Drug Congress 2024 conference, Ramaiah Muthyala, Indian Organization for Rare Diseases, noted that India's pharmaceutical sector is expected to grow to $100 billion by 2025While all of the world's approximately 450 orphan drugs are manufactured (APIs) in India, they are largely inaccessible and unaffordable in IndiaStill, access remains a large issue in India, with vast differences between the cost of imported orphan drugs and domestic costs
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
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