The view was set out in a paper by European Society of Cardiology experts who said the large patient populations and long time needed to prove a CVD drug works make for costly and inefficient randomized controlled trials.
Author Paulus Kirchhof said: “Cardiovascular drug development is partially a victim of its own success, where mortality benefits are the accepted benchmark in the field, and new therapies have traditionally been evaluated in large cohorts.
“We have developed ever more robust systems to conduct such trials, and are now faced with a total cost that seems prohibitive for some of the novel developments.”
He told us “Most [cardiovascular] conditions are chronic with a long time lag between initial diagnosis and events, hence requiring longer treatment durations for disease modifying therapies.”
Kirchhof added that: “Large [patient] sizes are the consequence of the past successes, where cardiovascular survival has been improved through good evidence-based therapies.”
Instead the authors suggest a pre-approval evaluation process be used for cardiovascular medicines, wherein candidate drugs are granted conditional approval if they are successful in trials in smaller patient populations.
Further efficacy and safety data would then be collected when the drug is generating revenue, which the authors suggest would be likely to encourage pharma firms to invest in the development of new products.
Real world safety data
Under the system, conditionally approved drugs would not be generally available Kirchhof said, explaining that: “The model that we propose will provide a limited clinical availability for persons that are most likely to benefit, with the possibility to enlarge to bigger populations.
“This has the advantage of generating safety data, which usually require large data sets, and cost effectiveness data in a real-world scenario early on.”
Drugs that prove to be a safety risk would have their approval withdrawn, however, the researchers argue that the threat of a product coming off the market is still an improvement on the current trial model.
“All drug makers take this risk. It is the excessive size of the risk, and the high probability of late stage failure where large investments are at risk that renders drug makers more risk averse” Kirchhof said.
Source: European Heart Journal
Improving clinical trials for cardiovascular diseases: a position paper from the Cardiovascular Round Table of the European Society of Cardiology.
doi:10.1093/eurheartj/ehv2013