Table 1 Comparison of incentives for products that are granted orphan drug designation
United States | European Union | Japan | |
|---|---|---|---|
Financial incentives | • Tax credits can apply to as much as 50% of qualified clinical development costs (US studies) • User fees paid to the FDA for review of the sponsors' application for marketing authorisation are waived | • No general tax credit on clinical trials and no specific subsidies for clinical trials • Regulatory fee reductions generally favour small and medium-sized enterprises, but are revised from time to time • Member states might offer a variety of price and reimbursement incentives as well as tax credits (see Ref. 7) | • Financial subsidies for up to 50% of expenses for clinical and non-clinical research • Subsidies through the NIBIO to reduce the financial burden of product development • User fee waivers, 15% tax credits, up to 20% corporate tax reduction and a 30% reduction in marketing application fees |
Marketing exclusivity | 7-year marketing exclusivity is granted to a product that, after receiving an orphan designation, goes on to receive a marketing approval as an orphan drug, meaning that the FDA cannot approve another (competing) marketing application for the 'same' drug treating the 'same' orphan diseases or conditions. | • The 10-year market exclusivity protects against a similar drug being authorized for the same therapeutic indication • Three derogations from this rule exist: first, sponsor's consent; second, lack of supply; and third, if a new product (although similar) could be demonstrated to be 'clinically superior', that is, 'safer, more effective or otherwise clinically superior' than the product already on the market | Extension of the re-examination period to 10 years at marketing authorisation |
Scientific advice (protocol assistance) | • Access to free scientific guidance at the FDA • Guidance by the relevant review division at the FDA on the regulatory requirements for quality, non-clinical development and the design of the clinical trials to demonstrate the efficacy and safety of the drug | • Access to free-of-charge protocol assistance at the EMA • Guidance on the regulatory requirements regarding quality, non-clinical development and the design of the clinical trials necessary to fulfil the regulatory requirements for the demonstration of efficacy and safety of the drug | • A 30% fee reduction for protocol assistance • Guidance is given on the regulatory requirements regarding quality and non-clinical development, as well as on the design of the clinical trials necessary to fulfil the regulatory requirements for marketing authorization |
Grants for research programmes | • The FDA Orphan Products Grant Program offers funding for clinical studies (investigating safety and/or effectiveness) that will result in or substantially contribute to market approval • The National Institutes of Health (NIH) also has a grants mechanism for rare diseases | • The European Commission supports rare disease research through its framework programmes and the call for proposals in the rare disease area usually includes Europe-wide studies of the natural history of rare disease, pathophysiology and the development of preventive, diagnostic and therapeutic interventions • Member states offer a variety of grants (see Ref. 7) | • Support measures include grants in aid for clinical and non-clinical research programs, price-control policies negotiated by Japanese National Health Insurance and pharmaceutical companies, and medical expense reimbursement for 56 diseases • NIBIO and AMED offer grant programmes to small and medium-sized enterprises and researchers who are developing products for rare diseases |
Regulatory tools to accelerate approval of drugs | • Fast-track approval • Breakthrough designation • Accelerated approval pathway • Priority review designation | • Priority medicines (PRIME) • Centralized procedure • Conditional approval • Approval under exceptional circumstances • Accelerated assessment | • Priority review • Fast-track approval |
