Proposed ‘Grant-And-Access’ Program With Price Caps Could Stimulate Development Of Drugs For Very Rare Diseases

Abstract

The 1983 Orphan Drug Act created incentives for the development of orphan drugs. Despite its successes, including a substantial increase in new drugs, approved orphan drugs still treat fewer than 5 percent of registered rare diseases. In addition, concerns have arisen about the high prices of many of these therapies, which can cost hundreds of thousands of dollars per patient each year. In this article, we propose a new "grant-and-access pathway," in which drug developers could opt to compete for federal grants to subsidize the costs of clinical testing. In return for the grant funding, companies would no longer claim orphan drug tax credits and would agree to price caps for marketed products based on the duration and costs associated with drug development, expected market size, and target rate of return. We identify scenarios in which such a policy could provide a net benefit to society.

Estrategias para promover el acceso a medicamentos de interés en salud pública: revisión estructurada de la literatura

“…El conocimiento generado por esta i+d debe ser de libre acceso, con el fin de intercambiar conocimientos y tecnologías que faciliten el desarrollo de nuevos productos en salud [4,17,20]. En contrapartida, las compañías farmacéuticas podrían desvincular el precio final de las tecnologías en salud del costo de la i+d [4,17,20,24]; en este sentido, el precio podría establecerse basado en la duración de los tratamientos, el tamaño esperado del mercado y las tasas de retorno [30]. • Buscar el apoyo de organismos internacionales (Organización Mundial de la salud, Organización de las Naciones Unidas) para la financiación de proyectos de i+d nacional, que permitan la producción de medicamentos de interés en salud pública que más necesita el país [29].…”

We need a “made in Canada” orphan drug framework

“…3 Some have advocated for the elimination of tax credits in favour of a grant program in which the recipient would be required to adhere to a "price cap" for their orphan drug should it be brought successfully to market. 10 Canada must consider such novel incentives.…”

Fair pricing of “old” orphan drugs: considerations for Canada’s orphan drug policy

“…Payers tend to decide on reimbursement of new drugs with a view to their therapeutic value, and for orphan drugs, rarity is sometimes a consideration. 12 But for older drugs, prices should reflect the cost of secure, stable and safe production, and nothing more.…”

“…Another strategy, applicable to future orphan drugs developed in whole or in part with public research funds, is to attach stipu lations for reasonable pricing to those funds, as recently proposed in the US. 12 Downstream, manufacturers of orphan drugs could be required to account for their research and development expenditures to determine an appropriate rate of return 20 for new orphan drugs. As Canada moves to design its framework for orphan drugs, it must provide for sustainable access to essential prod ucts on reasonable terms, and this will likely involve a set of innovative strategies.…”