Advance market commitments for paediatric and rare cancer drugs
Payers would promise in advance to buy a set number of doses at a set price for any drug that meets a defined bar in a rare or childhood cancer, so companies know the market exists before they invest.
Modelled on Gavi's pneumococcal advance market commitment, a consortium of national payers and foundations pledges a guaranteed purchase (volume times price, for example $300 million over ten years) for the first therapy meeting a target product profile in a specified rare or paediatric indication: diffuse midline glioma, relapsed neuroblastoma, Ewing sarcoma, rare fusion-driven cancers. In exchange, the developer accepts a long-run affordable price and supply commitments. The commitment is only paid on delivery of a product that meets pre-registered efficacy criteria, so payers bear no risk of failure.
- Incentives reward me-too drugs and marginal gains · The system pays the same for a drug that adds two months as for a cure, so companies race to copy rather than to cure.
- Rare and paediatric cancers without markets · Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.