Innovative trial design: where regulators and payers divide

15th November 2017 (Last Updated November 15th, 2017 11:25)

There is an increasing movement in the pharmaceutical industry away from traditional trial design as more dynamic and innovative design approaches offer new ways to demonstrate clinical effectiveness. Although these innovative trial designs have been accepted, and in some cases encouraged, by regulators, the same cannot be said for increasingly powerful payers.

There is an increasing movement in the pharmaceutical industry away from traditional trial design as more dynamic and innovative design approaches offer new ways to demonstrate clinical effectiveness. Although these innovative trial designs have been accepted, and in some cases encouraged, by regulators, the same cannot be said for increasingly powerful payers.

Traditional randomized clinical trials (RCTs) have been the standard requirement for regulatory approval over the decades. However, marketing approval is increasingly being based on more innovative designs, such as adaptive, umbrella and hybrid clinical trials.

Why has industry decided that these clinical trials are suddenly an appropriate replacement for the regimented RCTs? Well, drug development is getting increasingly expensive and resource-intensive, and these new clinical trial designs offer a more efficient development process, a key benefit in any developer’s eyes.

The nature of these trials allows for modification after initiation, the integration of prospective and retrospective data, and multiple drugs being tested in a single disease or a single drug being testing across multiple diseases. As such, they ensure that fewer patients receive ineffective treatments and allow for multiple analyses in single trials, reducing the development timeline and cost.

However, these trials do not come without risk.

Dynamic trial design: payers' concerns

Many payers view these innovative clinical trials as an opportunity for the industry to try and bypass key aspects of the regulatory pathway. An opinion in the payer world, as debated at the International Society For Pharmacoeconomics and Outcomes Research (ISPOR) 20th Annual European Congress in Glasgow, Scotland earlier this week, is that developers adopt these trials when they feel their product will not stand up to the rigors of RCT analysis and hence turn to a less validated trial system to hide the true efficacy of their product.

As such, payers are concerned that the wide adoption of these trials will mean that drugs with immature efficacy and safety data will be introduced into the market without a clear understanding of their true value. An increasing predicament among health technology assessment (HTA) bodies in Europe is uncertainty about the data they are receiving, whether from accelerated approval initiatives or the increasing number of non-comparator trials and the use of surrogate endpoints. These fluid clinical trials designs represent yet another point of uncertainty HTA bodies will have to face in the future.

Although these innovative clinical trials are being welcomed by regulators, patient access to these innovative products may be restricted by the HTA bodies and payers. If developers are looking to enter the market with these alternative clinical trial designs, GlobalData believes they will need to fully understand the perspectives of both regulatory agencies and payers.