Snake Oil or Super Drug: How Have Regulators and Payers Perceived New Medicines that Target Asymptomatic Conditions?

Abstract

Objectives: Assess how relevant precedent within the EMA (a key regulator) and NICE (a key HTA body) can inform how such novel medicines targeting asymptomatic conditions can be brought to market from a regulatory and payor perspective. Methods: All phase 2 or phase 3 clinical trials with "asymptomatic", "presymptomatic", "subclinical", "premanifest", or "preclinical" as a condition key word were downloaded from clinicaltrials.gov. Trials were cleaned to identify those for novel medicines with a primary target to treat asymptomatic conditions. EMA and NICE reports for such drugs were downloaded online in December 2021 and analysed to understand how they assessed the evidence. Results: 278 clinical trials were identified. Of those, only 8 had relevant matching reports from EMA and/or NICE. 7 of these 8 received a positive review from EMA, and 4 of these 8 received a positive recommendation from NICE. Those receiving positive recommendations showed or were assumed to show statistically significant survival benefit given a short life expectancy of untreated patients. Conclusions: There is no precedent within EMA or NICE for approving or funding new medicines for asymptomatic patients where survival benefit cannot be established. Additional research is warranted to understand how regulatory and payor agencies can prepare for future generations of innovative medicines.

Keywords

• Prevention
• HTA
• Regulatory
• Endpoints