Measuring Value In the Real World
By: John Doyle, DrPH, MPH | April 18, 2014
One of the biggest themes to emerge from our recent survey on value is the demand for proof. Payers and providers want detailed evidenced about the efficacy a treatment before determining whether they will prescribe and pay for it.
Historically, the industry has relied on evidence of value generated in clinical trials that simulate the performance of the product in the real world, allowing scientists to draw conclusions about their application. Such pivotal clinical research is still a vital component of the drug verification process, but it is no longer enough. Now market stakeholders expect that evidence to reach beyond traditional clinical trials, to include information about real world results. That adds complexity to the value definition process.
In the real world, patients are typically older and sicker than those in clinical trials. They may take concomitant medications for comorbidities, and they often have varying levels of adherence to medicinal regimes and heterogeneous follow-up care. And because investigators in experimental settings behave differently than clinicians treating patients in the real world, this can further impact outcomes. All of these real-world variables can impact the risk-benefit profile of treatments, and create situations in which clinically successful products fail to gain payer support.
Payers are currently trying to decipher how to determine whether a product will behave differently in the real world compared to the experimental world, and to what extent. For now, they are relying on educated guesses, coupled with models and simulations. But they want more. And they are waiting for a biopharma to step up and say, ‘now that our product has FDA or EMA approval, we want to prove that it is effective in your setting.’
To do that, biopharma companies need to be prepared to generate and analyze real-world data that supports their clinical results, and to create checkpoints with providers and payers to evaluate how a new product is performing in the real world. Such efforts will require more observational long term studies, but will result in the more precise valuations that the industry seeks.