OBJECTIVES

Acute myeloid leukemia (AML) has the lowest survival rate among leukemias that occur in adults and delays in access to new drugs by AML patients further reduces the likelihood of survival. The review focuses on identifying the potential reasons for this delay in Europe and Canada, and ways to speed up the process.

METHODS

Relevant grey literature on regulatory approvals and Health Technology Assessment (HTA) process of new drugs in Europe and Canada were searched. We selected papers that followed the journey of a new drug through the approval process and identified reasons for the delay in patient access to drugs for AML treatment.

RESULTS

In Europe, it takes 245 days by the European Medicine Agency (EMA) for market authorization and 318 days on an average across all European countries for HTA recommendations and market access. In contrast, it takes 225 days to receive regulatory approval from Health Canada and 149 days for the Canadian Agency for Drugs and Technologies in Health (CADTH) pan-Canadian Oncology Drug Review (pCODR) process (except for Quebec). Furthermore, it takes 273 days for price negotiations by the pan-Canadian Pharmaceutical Alliance (pCPA) and 67 days for provincial formulary listings.

It is observed that HTA dossier submitted with clinical evidence generated using a shorter trial period doesn’t capture long-term clinical efficacy data required by HTA authorities, resulting in re-submission of application with additional evidence. Also, due to the use of international reference pricing for new drugs, pharmaceutical companies sometimes delay the submission of drug applications until they get initial approval in a higher-priced market.

CONCLUSIONS

Inefficiencies in patient access to innovative drugs affect patient survival and have adverse health consequences. Efforts and initiatives on part of drug manufacturers and government, such as a targeted timeline for review and a centralized drug listing would help to expedite access to AML drugs.