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U.S. Sen. Bill Cassidy (R-La.) has published a report titled “Congress Must Act to Bring Needed Reforms to the 340B Program,” laying out potential reforms for Congress to improve transparency in the much-criticized 340B ProgramThe report urges Congress to mandate that covered entities provide detailed annual reporting on how 340B revenue is used to ensure direct savings for patients, as well as address potential logistical challenges caused by increased administrative complexity, among other suggestions The discount program has been under fire for a while for its massive growth and a perceived lack of integrity and oversight. According to pharmaceutical companies and other critics of 340B, the program has grown well beyond its original scope, with contract pharmacies driving up instances of duplicate discounting and diversion and large hospitals abusing savings to pad their already significant margins
Keynote speeches during World Orphan Drug Congress (WODC) USA 2025 reflected the rapidly changing policy and regulatory landscapes currently faced by pharmaceutical companies and other stakeholders in the rare disease spaceSpeakers focused less on specific policy issues this year and more on developing a unified roadmap for getting more cures to patients through utilizing innovative tools like artificial intelligence (AI) and continuing to advocate for streamlined regulatory pathwaysIndustry, policy, and advocacy leaders also led several important discussions focused on staying optimistic during uncertain times, as well as amplifying and centering patient voices during all stages of drug development and commercialization
Speaking at World Orphan Drug Congress (WODC) USA 2025, the U.S. Food and Drug Administration's (FDA) Amy Comstock Rick provided an update on the agency's new initiative, the Rare Disease Innovation HubShe also noted that despite changes at FDA, there have been no hints the Hub will be impacted. Rick explained that rare disease therapy development is not a partisan issue, commenting, "I remain optimistic that it may be a key time for the Hub to promote ideas of efficiency and coordination"The Hub is expected to host a workshop focused on clinical trial design for rare disease therapies in Summer 2025
A sustainable and flexible launch strategy is essential for commercialization across several markets, according to panelists speaking at the World Orphan Drug Congress (WODC) USA 2025Raquel Cabo, former SVP Corporate Affairs and Commercial Strategy, Ovid Therapeutics; Wills Hughes-Wilson, Chief of Patient Access & Commercial Planning, Mereo Biopharma; Gwen Hyland, Head of Commercial Excellence, Rare Diseases, Alexion Pharmaceuticals; and Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics, discussed a range of best practices for pharmaceutical companies hoping to achieve global commercializationCabo and Hughes-Wilson, coming from smaller biotech companies, also emphasized the importance of early preparation when battling resource constraints
Entod Pharmaceuticals launched Myatroo XL (atropine sulphate ophthalmic solution 0.05% w/v), the country's first approved low-dose atropine eye drops for managing pediatric myopiaThe eye drop aimed at slowing down myopia progression in children aged between 6 and 12Myatroo XL is set to be available by prescription across major eye clinics, hospitals, and pharmacies from July 2025
Panelists speaking at the World Orphan Drug Congress (WODC) USA 2025 discussed pathways to rare disease treatments in low- and middle-income countries, noting that humanitarian aid is not enough to ensure accessKey speakers on the topic included Ramaiah Muthyala, Professor at the University of Minnesota and President and CEO of I Am Not Alone; Diego Fernando Gil Cardozo, President of ERCAL; Jean-Pascal Roussy, Head, Global Public Affairs, Rare Diseases and Rare Blood Disorders, Sanofi; Ellen Cho, Senior Director, Specialty Programs, Direct Relief; and moderator Harpreet Ram, President, EVR ConsultingAll of the experts agreed that while humanitarian aid is important, multistakeholder partnerships are needed to build sustainable commercial access to innovative therapies around the world. They discussed innovative approaches like the so-called "Netflix model" and risk-sharing arrangements
At the World Vaccine Congress, Georges Benjamin, Executive Director, American Public Health Association, said, “We're in an era of eroding of trust in vaccines in the area of public health”Despite the progression of anti-vaccine policies, biotech leaders stressed the importance of retaining government support for vaccine development and accessRegarding insurance coverage for vaccines, Richard Hughes IV, Partner, Epstein, Becker & Green, The George Washington University Law School, does not expect any immediate, drastic changes due to anti-vaccine policies: “It's not going to look like an instant drop in coverage for a vaccine. It's going to be a $5 co-pay. The more you increase the co-pay, the less likely it is that the patient will get the vaccine”
At the World Vaccine Congress 2025, Maya Chandrasekaran, Director, Social Science Research Council, presented cost-effective, scalable interventions that build vaccine demandSupported by $25 million in funding, The Mercury Project has launched interventions across the Americas, Africa, and South AsiaAmong many other topics, the project has investigated low COVID-19 vaccination rates in the U.S. and inaccurate health information circulating among teens
At the World Vaccine Congress 2025, Isaac Uwins, a data analyst, presented an overview of combination vaccine development, opportunities, and future demandThe adult seasonal respiratory disease combination vaccine pipeline is divided, with COVID-19- or influenza-containing combinations on one side and RSV-containing combinations on the other sideUwins said pricing should be carefully considered, because COVID-19/influenza combination vaccines risk being priced too high versus standalone vaccines
Ukraine has introduced a major overhaul of its drug pricing system, with new laws on price declarations, international reference pricing (IRP), and a national catalog of drug pricesThe National Price Catalog will serve as the official record of declared maximum wholesale prices submitted by manufacturers and importers - entries will be published on the Ministry of Health’s website and updated on a monthly basis. Going forward, price declaration is mandatory for medicines on the National List of Essential Medicines, all prescription drugs and selected OTC medicines (as designated by the Ministry of Health)In parallel, Ukraine is introducing international reference pricing (IRP) against a basket of eight countries: Poland, Slovakia, Czech Republic, Latvia, Hungary, Moldova, Romania, and Bulgaria. Reference prices will be calculated as the average of the second, third, and fourth lowest prices among the four lowest prices available for a comparable product in these countries. The lowest price is excluded from the final average
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