Cystic Fibrosis

SANTA BARBARA, Calif., May 5, 2025 /PRNewswire/ -- Cottage Health has partnered with Clearway Health, a specialty pharmacy services company, to develop a specialty pharmacy program that enhances patient access to medications with concierge-level service at a lower cost. This collaboration brings personalized pharmacy support to Cottage Health's specialty outpatient clinics, enhancing patient access to medications. Continue Reading Cottage Health partners with Clearway Health to develop a specialty pharmacy program, enhancing access to medications. Post this “Integrating pharmacy services directly into our clinics allows us to address the unique challenges patients face with specialty medications,” said Lisa Moore, executive vice president and chief operating officer at Cottage Health. “This ensures our patients receive the therapies they need while reducing barriers and improving outcomes.” The program is designed to simplify the prescription process by assisting with insurance approvals, financial assistance, medication delivery, and ongoing support, making it easier for patients to manage their treatment without delays or added stress. As part of the partnership, pharmacists and patient liaisons are integrated directly within Cottage Health's specialty clinics, working closely with patients to navigate insurance coverage, secure financial assistance and manage prior authorizations—alleviating administrative burdens for both patients and providers. "Integrating pharmacy services directly into our clinics allows us to address the unique challenges patients face with specialty medications," said Lisa Moore, executive vice president and chief operating officer at Cottage Health. "This ensures our patients receive the therapies they need while reducing barriers and improving outcomes." The program has already launched at the Cottage Cystic Fibrosis Clinic and pediatric clinics, where patient liaisons are assisting patients with accessing both specialty and non-specialty medications. Specialty medications typically require special handling, administration and monitoring and are used to treat chronic conditions such as multiple sclerosis, cystic fibrosis, cancer and inflammatory conditions. "Clearway Health is pleased to work alongside Cottage Health to provide their patients and community with transformative specialty pharmacy services," said Allison Arant, senior vice president, client development and marketing, Clearway Health. "Cottage Health's patient-first approach and commitment to helping patients get back to living their lives directly coincides with our ability to improve access to care and manage the complex medical needs of patients. Every patient deserves the care we provide. We celebrate and congratulate Cottage Health for choosing to build this program and offer specialty pharmacy services to their patients." Future plans include expanding specialty pharmacy services to other Cottage Health clinics and opening an outpatient retail and specialty pharmacy, further enhancing medication access and convenience for patients. About Clearway Health Clearway Health's specialty pharmacy services create sustainable assets and build lasting legacies for hospitals and health systems, providing a service that every patient deserves. Our team began at Boston Medical Center Health System, improving access to care and managing the complex medication needs of vulnerable patient populations. Equipped with this unique experience to solve challenges, Clearway Health partners with other hospitals and health systems to provide transformative specialty pharmacy services that put patients first. Our strategic partnerships build and strengthen the infrastructure for specialty pharmacy programs through improving operational workflows, implementing clinical programs, hiring and embedding skilled pharmacists and patient liaisons into the clinical team, providing proprietary software technology, unlocking drug access and leading accreditation processes. Clearway Health delivers a clinically driven service that eliminates barriers to medication access, improves the health of patients and communities, grows revenue at healthy margins for health systems and reduces costs for patients. To learn more, visit . About Cottage Health | CottageHealth.org Cottage Health is a not-for-profit health system providing advanced medical care on California's Central Coast. In the past year, its hospitals in Goleta, Santa Barbara and Santa Ynez Valley cared for 20,733 inpatient admissions, 91,649 emergency department visits and 2,005 newborn deliveries. With more than 700 physicians, including specialists typically found only at university medical centers, Cottage offers expert, comprehensive care. As a teaching institution, it trains physicians through residency programs in medical, surgical, pediatric and radiology specialties. Cottage Health is home to the Central Coast's only Level I adult and Level II pediatric trauma center and an advanced comprehensive stroke center. Its specialty services include Cottage Children's Medical Center, Cottage Rehabilitation Hospital, the Santa Barbara Neuroscience Institute, Cottage Heart & Vascular Center and Cottage Center for Orthopedics. Beyond its hospitals, Cottage expands care through primary and specialty clinics, urgent care centers and 24/7 provider access via Cottage Virtual Care. 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Vertex is pulling back on its genetic medicine research, ending all work related to adeno-associated viruses, or AAVs — the viral vectors commonly used to deliver gene therapies into the body, the company confirmed to Endpoints News . The cuts impact Vertex’s collaborations with at least two biotech startups: the AAV design company Affinia Therapeutics and tRNA therapy company Tevard Biosciences, which uses AAVs. It also calls into question Vertex’s work on CRISPR therapies for genetic muscle diseases. Several bigger pharmaceutical companies, including Biogen , Pfizer , Roche and Takeda , have cut or pared back on AAV gene therapy research in recent years. And several gene therapy biotech companies have trimmed their pipelines and staff as private and public investors sour on the field. This change of fortune was seemingly unimaginable a few years ago. AAV technology ushered in a new era of gene therapy investment and led to several commercial products for diseases, including Roche’s Luxturna for childhood blindness, Sarepta Therapeutics’ Elevidys for Duchenne muscular dystrophy, and Novartis’ Zolgensma for an otherwise fatal neurodegenerative condition in infants. But a panoply of challenges — including costly manufacturing, multimillion-dollar drug prices, slow progress in improving targeted delivery and lingering safety issues — has raised questions about the future of AAV medicines. Vertex did not have any AAV gene therapies in clinical development, despite work on the technology stretching back to at least 2019 when it acquired Exonics Therapeutics for its early-stage CRISPR therapies for two muscle diseases: Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). Exonics relied on AAV vectors to deliver its therapies to muscles. Vertex has shared few updates on the program since its acquisition. The approach was essentially a permanent version of the commercial exon-skipping therapies for DMD, meaning it wouldn’t fully restore the dystrophin protein that’s broken in people with the disease. A Chinese biotech called HuidaGene has developed a similar CRISPR therapy and has already treated two boys, Endpoints reported last month . The first patient had modest improvement in his walking ability three months after the treatment. It is unknown why Vertex’s own CRISPR therapy has seemingly lagged behind. It is possible that the company is holding out for a more curative approach. It may also have been spooked by multiple deaths in people who received the high doses of AAV needed to reach the body’s many muscles, including two from Pfizer’s experimental therapy , one from Sarepta’s approved drug and one from a custom CRISPR therapy . Heather Nichols, a spokesperson for Vertex, confirmed that the company has discontinued its AAV work, but in an email said that the company’s “commitment to cell and genetic therapies remains strong” and that other investments in DMD and DM1 continue. The company declined to provide further comment on its AAV cuts or make an executive available for an interview. The need for better AAVs, ones that could deliver more of the therapy at lower doses, has been clear for years. Vertex struck a partnership with Affinia in 2020 worth up to $1.6 billion for access to the startup’s viral vectors designed to better target the muscles for treating DMD and DM1 and target the lungs to treat cystic fibrosis, the disease responsible for most of the company’s $11 billion revenue last year. Affinia CEO Rick Modi told Endpoints in an email that his company regained the rights to its muscle-targeting viruses from Vertex in March. He said the earlier work on cystic fibrosis ended in 2022 and rights to those programs were returned then. The startup is currently focused on finishing preclinical studies and designing a clinical trial for its lead candidate, for a heart condition called BAG3 dilated cardiomyopathy. Modi said that Affinia received “the maximum amount of milestone payments” for the research phase of its DMD and DM1 collaboration with Vertex, but he wouldn’t specify the amount. He added that Affinia is looking to license its newly-regained muscle capsids to other companies. Vertex’s step back from AAVs also impacts Tevard Biosciences. The small startup, which is housed in Eli Lilly’s genetics-focused research center that opened in Boston last summer, struck a four-year partnership with Vertex in February 2023 to develop a new class of genetic medicines known as tRNA therapies. In nature, tRNA molecules are part of the machinery that cells use to translate genetic code into proteins. Tevard is one of a few companies developing synthetic versions of these molecules to help skip over genetic mutations that prevent cells from making fully functional proteins. Tevard believes its approach could help restore the vital dystrophin protein missing from the muscles of the 15% of people with DMD whose disease is caused by so-called nonsense mutations. While some companies are trying to package tRNA therapies in lipid nanoparticles, those delivery vessels are not readily taken up by the body’s many muscles, so Tevard planned to use AAVs to deliver its treatment. “We were doing great. The collaboration was going great. But in December, we were informed that they were realigning priorities, and multiple programs were being terminated, including this one,” Tevard CEO Daniel Fischer told Endpoints in an interview. “Two weeks ago, we got the rights back to this program.” Other tRNA companies have also struggled, including HC Bioscience, which shut down in March after “challenges in targeted delivery,” Endpoints previously reported . Fischer said the fate of the DMD program will depend on whether Tevard can find another drugmaker to partner with or raise enough money to move it forward on its own. The company is planning to present data showing that the DMD therapy works in mice at the upcoming American Society of Gene & Cell Therapy meeting later this month.

iStock, Abscent84 No Patient Left Behind says that drug value assessments used in countries like Canada and Germany undervalue innovative medicines by 90% and mislead U.S. policymakers into thinking Americans are overpaying. What is a drug worth? In a world of patchwork regulatory and market dynamics, it depends where you are. And at the moment, everyone seems to think there is something wrong with drug prices. President Donald Trump is said to be considering another attempt at a Most Favored Nations rule, which would set U.S. drug pricing in line with other countries where medicines are cheaper. But across the pond, European pharmas are fighting to have drug prices increased to better support innovation. A new report from No Patient Left Behind (NPLB) attempts to explain the pharma side of the equation with an evaluation of the value assessments that non-U.S. nations go through to figure out how much they will pay for a drug. NPLB argues that these assessments in countries outside the U.S. “significantly underestimate the true societal benefits of innovative medicines.” It’s similar to the argument the CEOs of Sanofi and Novartis made in a letter to the European Union last week. NPLB says that such foreign health technology assessments (HTAs), particularly those used in Canada and Germany where data was available, undervalue medicines by 90%. “When high-income foreign countries use traditional HTA as a bargaining tool to deny coverage and/or secure lower prices, they free ride on the R&D investments made outside their countries and reduce patient access to novel medicines in their own countries,” the report read. These value assessments give U.S. policy and lawmakers the false impression that a market-based system is “broken.” But this system is what “fuels” biopharma innovation, NPLB said. Typically, HTAs have used what NPLB calls “outdated” willingness-to-pay thresholds that tend to be built on “flawed” quality-adjusted life years. NPLB argues that these calculations do not adequately factor in benefits beyond those to the patient. “[T]hey ignore that new medicines not only benefit patients, but also their family members and society at large by enabling faster return to work, therefore increasing taxes paid and disability allowances not paid,” the report said. The assessments also do not consider ease of administration, which may allow a patient to skip a hospital visit, travel costs and increased adherence. Another problem is that these assessments typically set one price and stick with it, rather than following the curve of the exclusivity period, NPLB said. Rarely does a drug stay one price throughout its lifetime as a brand name drug. As an alternative, NPLB put forth a different method called the Generalized Cost-Effectiveness Analysis (GCEA), which was developed by a panel of health economists. The report stated that this model creates a more comprehensive estimate of how a treatment can benefit society. NPLB used this model to determine that treatments are undervalued by 90%. The report analyzed 10 medicines to compare pricing from the different models, without providing names of the therapies. One cystic fibrosis drug should be priced at $422,292, according to the GCEA, but Canada recommends a price of $25,542. Meanwhile, a cardiovascular drug valued at $7,819 in the GCEA model has price recommendations of $911 in Canada and $1,022 in Germany. And an obesity drug that Canada prices at $1,589 should actually cost $44,312. “These flawed assessments used in foreign countries demonstrate how those agencies care more about the government budget than value to society as a whole,” NPLB said. Medicines that do not adhere to the value assessments made by these countries can be blocked or delayed from entering. “Each of the 10 drugs in our analysis was initially developed and approved in the U.S. where market-based pricing system played an important role in incentivizing research into new innovation,” the report explained. “The political effect is that these underestimations of value and lower prices outside the U.S. have been used to argue that Americans are paying more than they should truly be willing to pay.” NPLB called foreign nations’ reliance on the outdated models “an excuse to pay less” said it is misleading U.S. policymakers. Fewer new medicines are reaching nations with these pricing policies after launching in the U.S. “Our analyses showed that a good case can be made that the U.S.’s patent-based, market-based competitive framework is working reasonably well, despite an imperfect insurance system, to bring about affordable biomedical innovation that benefits not only U.S. citizens, but everyone worldwide,” the report concludes. “Should U.S. policymakers impose foreign price controls on novel medicines in a misguided effort to spare U.S. payers from perceived ‘overpaying’, they would be undermining innovation that would be cost-effective for the U.S. and beneficial globally.”