![]() ![]() “We’re no longer just a contracting entity whose work ends with throwing a discovery over the fence. The expansion of PCI-now about three times the size of its predecessor, the CTT-has empowered researchers to accelerate the advancement of their discoveries by positioning them as active research and development partners with pharmaceutical and biotech companies. “Faculty know to disclose big discoveries to us, but also to come to us if they’re looking for sponsored research or thinking that maybe they should start their own company-whatever’s the best way to take their program forward and get it to patients,” says Swartley, who today is associate vice provost for research at Penn and PCI’s managing director. (Image: Courtesy of Penn Medicine Magazine) There is also some question whether agents that serve only a relatively small patient population, even if their prices are higher, are appropriate targets for negotiation.Carl June, at the flash mob celebration of the FDA approval of the CAR T cell therapy he developed, in August 2017. In addition, biologics - and gene therapies are categorized as biologics - are not eligible for negotiation until 11 years beyond their FDA approval date. She also suggested that Congress broaden drug-price negotiation allowances, or even carve gene therapies out of the commercial domain and have them “paid for by the federal government using prices that are based on their documented therapeutic value.”Īnother factor possibly affecting gene therapy prices is the provision of the Inflation Reduction Act that authorized the Centers for Medicare and Medicaid Services (CMS) to negotiate prices for selected drugs, starting with 10 Part D drugs in 2026.Ĭlotting factor agents are covered by Medicare Part B and will not be among those eligible for price negotiation before 2028. In her editorial, Vokinger suggested that “Regulators and policymakers could encourage institutions that receive federal funds to engage in nonexclusive licensing for certain key platform innovations that are part of the gene therapy processes they have developed, in keeping with their mission and in recognition of the public funding that supports such discoveries.” “These results of the first parenteral administration of AAV demonstrate that administration of AAV vector by the intramuscular route is safe at the doses tested and effects gene transfer and expression in humans,” she wrote at the time. Manno at the time was working in the Department of Pediatrics at the University of Pennsylvania. AAV is now the vector underlying Hemgenix and pipeline gene therapies in hemophilia B. Manno, M.D., currently a hematology-oncology pediatrician at NYU Langone Healthwas lead author in an initial study that took positive AAV findings from mice and canines and applied them with success to adult men with severe hemophilia B. Some of the research into the use of an adeno-associated viral vector (AAV) to promote expression of factor IX in humans with hemophilia B was performed more than 20 years ago by clinicians and academics unaffiliated with the commercial partnership between CSL Behring and uniQure that launched Hemgenix.Ĭatherine S. “All gene therapies approved in the United States thus far have their origins in academic institutions or spinoffs from such institutions that developed indispensable know how and underlying forms of technology,” wrote Vokinger and her co-authors, Jerry Avorn, M.D., and Aaron Kesselheim, M.D., J.D., M.P.H., who are part of the Program on Regulation, Therapeutics, and Law Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital in Boston and Harvard Medical School.įDA approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with hemophilia B in November 2022. ![]() Nonprofit research centers could commercialize these products themselves by subcontracting the development and marketing of these agents, she suggested. ![]() One suggestion of Vokinger’s suggestions is offering nonexclusive licenses or licensing to multiple entities rather than just one. The lead author of a recent opinion piece in the New England Journal of Medicine, she wrote that academic research institutions could help solve the problem of high-cost gene therapies by changing the way they license their discoveries to the commercial entities that bring them to market. Vokinger, M.D., J.D., PhD, a professor of law, medicine, and technology at Zurich University in Switzerland. ![]() The high prices of these products can be tremendously disruptive to health care systems due to the strain on budgets and restricted access for patients, wrote healthcare economist Kerstin N. ![]()
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