ISPOR Forums


6:15 PM–7:15 PM Nov 12, 2018

Room: 212 (P2)

Through innovation, pharmaceutical industry responds to unmet needs of diseases that affect the society. In recent times, society has sued the pharmaceutical industry solutions for the so-called "rare diseases". A disease is considered rare when it affects a limited number of the total population, defined in Europe as less than 1 per 2,000 citizens (EC Regulation on Orphan Medicinal Products). Rare diseases have not been the focus of innovation in the pharmaceutical industry due to the difficulty of finding a sufficient return on the investment needed to investigate this type of solution because of the few patients susceptible to treatment. Funding systems of different countries have evolved substantially in recent years, in order to achieve the best access to medicines by patients as well as to safeguard health systems making them sustainable over time, in times of budgetary uncertainty and future epidemiological and population challenges.

In this context, health systems have taken into account other factors in the funding of medicines beyond the cost of production. Economic evaluation has been given a leading role as a tool to distribute available resources in the most efficient and fair way possible. The funding of orphan drugs involves different challenges for healthcare systems. The way in which regulators undertake the funding of pharmaceutical therapies aimed at rare diseases will be a crucial factor to create incentives for investment in R&D and will be determinant to achieve greater access to medicines by patients, as well as ensuring the sustainability of public health systems. It is in this triad of challenges, where the economic evaluation of medicines for rare diseases, which allow the prioritization of resources, can not give an adequate response. Therefore, a consensus is needed on how regulators should try to pay for these drugs, in relation to the value they bring to the health system and the society.

ISPOR Spain Chapter