New challenges in health technology assessment (HTA): the case of Zolgensma.

Rare or orphan diseases have played an important role in the pharmaceutical industry. On the other hand, the impact of new technologies derived from genomic research has been growing in this industry, with new drugs being launched on the market at unsustainable prices for health systems and patients. This double tendency poses important and growing challenges to public policies on Health Technology Assessment, whose hegemonic rationale is based on cost-benefit analysis between therapies. The very high prices of these drugs require revisiting this rationale and the recent negotiations between the Brazilian Ministry of Health and Novartis regarding a possible risk-sharing agreement for the incorporation of the drug Zolgensma is an opportunity for this revisitation.