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

Context: Assess how relevant precedent within the European Medicines Agency (EMA) (a key regulator) and the National Institute for Health and Care Excellence (NICE) (a key health technology assessment (HTA) body) can provide insights into how such novel medicines targeting asymptomatic conditions can be brought to market from both a regulatory and payer perspective. Methods: All phase 2 or phase 3 clinical trials with keywords such as asymptomatic, pre-symptomatic, subclinical, premanifest, or preclinical keywords were downloaded from clinicaltrials.gov. Trials were cleaned to identify those for novel medicines with a primary focus on treating asymptomatic conditions. The EMA and NICE report for such drugs were downloaded online in December 2021 and analyzed to understand how they assessed the evidence. Results: Two hundred seventy-eight clinical trials were identified. Of those, only 8 had relevant matching reports from the EMA or NICE, 7 received a positive review from the EMA, and 4 received a positive recommendation from the NICE. Those who received positive recommendations showed or were assumed to show statistically significant survival benefits given the short life expectancy of untreated patients. Conclusions: There is no precedent within the EMA or NICE for approving or funding new medicines for asymptomatic patients where survival benefits cannot be established. Additional research is warranted to understand how regulatory and payer agencies can prepare for future generations of innovative medicines.