Are Accelerated Approval Mechanisms A Predictor To Early Access And Coverage? A Global Study Of Cancer Drugs

Abstract

To explore the interrelationship between accelerated approval schemes for cancer drugs and national HTA processes across thirteen jurisdictions globally, by investigating the impact HTA and value assessment has on drugs approved through accelerated pathways. 15 drug-indication pairs with cancer indications (melanoma, lung and haematology) were selected based on whether they received accelerated approval in the US or Europe via an early access mechanism (one of the FDA Accelerated Approval pathways or the EMA Conditional Marketing Authorisation [CMA], or both) until December 2015. In-depth analysis was conducted to assess the impact HTA and value assessment had on coverage and funding pathways in 13 countries (Australia, Brazil, England, Canada, France, Germany, Italy, Japan, the Netherlands, Scotland, Sweden, Spain and USA). The analysis relied on an analytical framework investigating in-depth: (a) Similarities and differences in clinical and economic evidence submitted; (b) Evidence interpretation; (c) Uncertainties; (d) Other considerations, drug or therapeutic-area related; and (e) Time difference between MA and HTA recommendation. Preliminary analysis indicates that market access for drugs authorised under an early access mechanism continues to rely heavily on HTA requirements. Additionally, HTA for these drugs does not necessarily lead to positive recommendations and may result in longer HTA review processes than drugs approved under standard MA. Moreover, some countries often resort to special funding arrangements to enable access to cancer drugs. Accelerated approval pathways do not guarantee coverage or early market access at population level and may result in delays in access. Alternative data sources and evidence to meet HTA requirements are important, including real world data generation or managed entry schemes, in order to enable access to new cancer therapies.