Spark Therapeutics Inc.'s voretigene neparvovec is pending at FDA for treatment of a rare form of hereditary blindness and seems likely to become the first-ever gene therapy approval in the US. By default, it will then become the test case for how reimbursement and coverage policy works in the context of gene therapy.
Gene Therapy Reimbursement: Is Blindness A Bad First Test?
The first gene therapy approval could be coming soon – followed shortly by the first ever gene therapy coverage decision. An Express Scripts executive argues that the tone may be affected by the initial indication.
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Health technology assessment agencies are willing to pay more than usual for advanced therapies that treat rare or severe diseases, but an advanced therapy for a common condition would not qualify for such a modifier, a former senior figure at England’s NICE says.
Manufacturers still hope to advance legislation removing the “pill penalty” from the law, as well as changes they want included in the CMS guidance for the 2028 price implementation year.
US pharma leadership is painting its mission, particularly its goal to end Medicare price controls, as patriotic and essential to America’s global dominance over China, a move that seems designed to align with President Trump’s priorities.
A lower asking price from BMS and more insight into the benefits Breyanzi offers have convinced the health technology assessment institute, NICE, to reverse its rejection of the one-off treatment for large B-cell lymphoma.
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The International Generic and Biosimilar Medicines Association has introduced its first global intellectual property and competition report, addressing the urgent need to reform current regulations. But, in a bid to increase access to medicines across the world, did it stop short at just IP and patents?
US pharma leadership is painting its mission, particularly its goal to end Medicare price controls, as patriotic and essential to America’s global dominance over China, a move that seems designed to align with President Trump’s priorities.
A lower asking price from BMS and more insight into the benefits Breyanzi offers have convinced the health technology assessment institute, NICE, to reverse its rejection of the one-off treatment for large B-cell lymphoma.