At Quintiles, we continuously monitor the assessments published by global Health Technology Assessment (HTA) organizations to identify the trends in payer decision-making that are shaping our industry. Currently, HTA bodies are paying close attention to drugs being approved on accelerated pathways and the challenges of accurately assessing their value.
Too soon to tell?
Over the past several years, a number of new policy and legislative options have been introduced to accelerate patient-access to newly approved therapies. These accelerated options, which include conditional approval and adaptive pathways in Europe and breakthrough therapy designation and fast track review in the USA, are designed to help speed medicines to market so that the industry can more rapidly address unmet medical needs. However, as these drugs come to market, often with slim portfolios of evidence demonstrating their safety and efficacy, it is difficult for HTA bodies to fully assess their value and to price them accordingly. In some cases, these drugs are approved on a single arm study with no comparison to other treatments. And because this is relatively new to payers, there are few roadmaps to follow in defining value based on a limited body of evidence.
The good news is that our data shows the products that succeed on accelerated regulatory pathways also perform relatively well in HTA. These bodies clearly support the goal of getting drugs to patients with unmet medical needs, as many of them target orphan disease conditions where there are literally no other medical options.
Of the 22 drugs granted conditional marketing authorisation (CMA) by EMA, as of April 2016 ten have an orphan indication and 15 are indications for oncology. As of May 2016 all but three of the 22 drugs had been evaluated by one or more key HTA agencies before receiving full market authorisation.
However, our data from the last five years also shows that drugs granted CMA have a higher probability for a negative recommendation compared to the average of all HTAs from the same agencies. This higher percentage of negative recommendations seems to be partly driven by the high number of oncology drugs with CMA. The data also showed that while five out of 20 negative recommendations mentioned lack of clinical benefit as a contributing factor, most of the rejections were driven by lack of cost-effectiveness.
If you are on the fast track
This data underscores an important strategy for developers seeking conditional approvals regarding the value of engaging with payers from the outset of research efforts. Receiving regulatory approval on an accelerated pathway does not guarantee payers will recommend your drug. Our research suggests that the chances of receiving a positive HTA can be improved when developers meet with HTA bodies in parallel with pursuing regulatory approval to understand what the challenges are likely to be, and what steps if any they can take to overcome them. Developers need to be sure that they are well prepared going into these meetings, as the quality of the advice they receive will depend entirely on the quality of the questions they ask. The HTA bodies cannot tell developers what to do, but they can respond to specific questions about their process. So developers should come armed with data and analyses, including an early health economic model, to allow for specific feedback on their evidence generation plan. In our experience we have found that parallel scientific advice is one environment where closed questions are more powerful than open ones.
Accelerated pathways are a vital tool in getting new drugs to market, but HTA bodies must find a balance between the need for robust evidence of safety and efficacy and the need for new medicines to address unmet need. Ultimately, HTA decisions for such products are most likely to be influenced by high unmet needs and a lack of therapeutic options for the patient population in question, but having an effective evidence package can improve the chances of a positive rating. In these projects every piece of data captured will be valuable in potentially demonstrating the safety and efficacy of a new drug. Working with payers early in the process can help developers understand what strategies they can employ to gather the most robust data to meet HTA needs.
> Read more about the impact of accelerated regulatory approval on HTA outcomes in the July issue of HTA Uncovered.