Decoding platform trials: defining aspects, benefits and challenges

By Tom Parke, Berry Consultants

The principal idea of a platform trial is as simple as it is radical. Whereas a conventional clinical trial tests a potential new treatment for a disease, ending it once we have enough data to conclude whether the treatment is effective or not, a platform trial tests more than one treatment at once and continues running, testing further new treatments, for as long as is necessary and possible.

There are two distinct settings for a platform trial. A large Pharmaceutical company that has a significant number of potential drugs in the same area can establish one to optimize their internal development. Alternatively, an organisation (normally not a Pharmaceutical company or Biotech) can set up a platform trial that will test treatments that come from a number of difference sources. Some may be novel drugs, other re-purposed drugs and some may be treatments that are not drugs at all. It is this latter setting, “cross company platform trials”, that EU-PEARL is particularly interested in exploring and fostering.

The potential benefits of a platform trial are considerable and varied, but so are the challenges to setting one up! At EU-PEARL we are working to reduce these challenges.

Platform trials, why?

The benefits that can potentially be realized in a platform trial are:

  • Just one control arm for many treatment arms. This means that fewer patients need to be given the standard of care and more can receive novel, experimental treatments. This can give patients more hope of benefitting from their participation in a clinical trial, but also contributes to make clinical science more efficient. We can test more potential treatments more quickly and cheaply compared to doing separate trials for each treatment.
  • Just one organization to run the trial, that can learn and improve and become more efficient over time. Compared to building up and tearing down a trial operation separately for each treatment, once a platform trial is up and running a new treatment can enter the platform trial much more quickly than setting up a separate trial for it, reducing its development time.
  • Because a platform trial is centred on finding a treatment for a disease, rather than centred on testing a treatment, there is more time, scope, and necessity for patient involvement. Indeed, charities and patients’ groups can be key players in establishing platform trials in their disease area. This implies giving them a say on their experience in the trial, the types of treatments tested and how treatments are evaluated.
  • As successful platform trial in a disease area will have significant impact on how treatments for that disease are developed, and generate a lot of scientific data around that disease. So the design process for the trial, provides an opportunity for regulators to become involved in the specification of how a particular disease will be studied.

What are the challenges?

Establishing a cross-company platform trial faces many challenges. Important questions need to be addressed:

  • What are the current and future treatments that could be tested in the trial? Are they sufficiently similar: “Do they address the same patients?”, “Can they be studied using the same tests on patients?”, and “Can their potential effectiveness be judged on the same criteria?”
  • Can the trial be made attractive and relevant to the patients it intends to study?
  • Can the trial be made attractive and relevant to the owners of novel treatments, so that they will prefer to have them tested in this trial rather than in one carefully crafted specifically for that treatment?
  • Will the trial need to take into account different types of patients who may respond better to different treatments?
  • Will the conduct of the trial and the data gathered be acceptable to regulators and ethics bodies?
  • Who will own the data and how will results be published when one treatment completes but other treatments are still in the trial?
  • How much will the trial be able to adapt, and how much will it need to adapt, to stay relevant over the years?
  • How will the trial be paid for, both initially and over time?
  • If there are many treatments that could be added to the trial who will vet which ones to include? If there is a danger that the trial will run out of treatments to test, who will go out and actively ‘sell’ the trial to the owners of potential new treatments?

EU-PEARL is attempting to explore these challenges to beat a path that others may follow who want to set up a platform trial. In order to be ‘real’ and not just theoretical, EU-PEARL is designing four different platform trials. In order to be general and not too disease specific, these four trials are in very different diseases: Major Depressive Disorder (MDD), Tuberculosis (TB), Non-Alcoholic Steatohepatitis (NASH) and Neurofibromatosis (NF). Supporting these four efforts are 3 working groups that as well as helping these teams with issues such as governance, sustainability, statistics, operations, data and patient networks, will produce reports, papers and tools to aid others in designing platform trials and Integrated Research Platforms (IRPs).

There are many good introductory papers on platform trials, see the EU-PEARL report D2.1 for some references. This report also lists references to some 14 Platform Trials that have been designed, many of which are up and running.


Tom Parke is Director of Software Solutions at ‎Berry Consultants, and member of EU-PEARL.