OBJECTIVES: Chronic diseases constitute a worldwide public health issue with important clinical unmet needs. Novel breakthrough therapies such as advanced therapy medicinal products (ATMPs) are in development to fulfill those unmet needs. ATMPs are expected to have high upfront costs. A key remaining question is the funding options of these new high cost therapies giving the large target population, and therefore the large budget needed. The aim of this study was to identify new funding sources for novel breakthrough therapies.

METHODS: A systematic review was conducted in Ovid Medline and Embase to identify innovative funding sources for novel therapies. Studies published between January 2000 and January 2017, written in English or French were included.

RESULTS: Four funding sources were mainly proposed in the literature: pooled funding, international transaction taxes, front-loading and debt reduction. Pooled funding is a combination of funding from multiple groups or multiple payers (in the case of the United States) to pay for a specific therapy. Another suggested solution is collecting funds through placing taxes and levies on specific transactions (e.g. plane tickets). Funds could also be provided through frontloading mechanism; some donors offer aids and resources to fund novel therapies like the International Finance Facility for Immunization that provided stable funding to achieve immunization goals. Furthermore, an international cooperation by debt reduction can constitute another solution, where a country creditor agrees to write off debt for a country debtor if the latter commits counterpart funding to an account that had been approved for a breakthrough therapy.

CONCLUSIONS: The suggested methods may be a potential source of additional funds for novel advanced therapies. Those methods have already been used for communicable diseases. A worldwide cooperation is needed to adapt these methods for non-communicable diseases in order to ensure the patient access to innovative therapies while maintaining the health care system sustainability.