Thursday, 7 April 2016

Shall HTA depend on RCT (randomised controlled trilas) or RWD (real world data) or both?

Thursday, 14.00- 15.30

Chris Chinn, Head of Real World Investigations, Sanofi and GetReal 

outcome- comparator- patients- intervention

Outcomes are affected by patient behaviour, physician behaviour and the healthcare system and not all of these can be measured in a clinical trial.


RCT to inform clinical effectiveness
Observational study for budget impact

integrate both data sets using network meta analysis (NESTA involved in this- worth checking out!)

TWiCS- run a randomised trial within an observational cohort, using the registry as a whole as control. group in Manchester, failed to capture name


In the end, patients make personal risk- benefit assessments: are the benefits of the drug out-weighing the risks? That then affects the effectiveness of the treatment.

So in my mind, we would have to worry less about the gap between effectiveness (drug in the real world) and efficacy (drug in a clinical trial) if we paid more to those personal-risk assessments EARLY ON.

PAES- Post-Authorisation Efficacy Studies - applying RCT standards to RWD

Giovanni Tafuri, EMA

uncertainties about drug efficacy stem from:
  • (sub-)populations studies
  • endpoints
  • long term use of a product
  • co-treatment with other products
  • real life usage
  • change in the understanding of a disease or a drug
  • change in scientific factors for previous efficacy 

choice of study design depends on the need and can include:
  • randomised trials
  • non-randomised trials
  • explanatory trials
  • pragmatic trials
  • observational trials

Data sources-

1. can be collected for the purpose: clinical trials

2. analyze existing data- e.g. registries, electronic Health records

Importance to agree on a design prior to starting and the place of real world data during the life cycle of the product: worth noting the registries.

The PARENT joint action patient registry initiative: purpose to reduce duplication between registries. Worth looking up!

And important- who sets the standards for the registries?

Comment of Guido Rasi, EMA- we need to define what real world data means

Conducting HTA using RWD (real world data)

Francois Meyer, HAS

Achievements of EUnetHTA

1. Database EVIDENT - HTA bodies can find out what others have collected


3. position papers on research recommendations

  • how to best formulate research recommendations
  • how to decide on the appropriate study design to answer those questions
4. core protocol pilot for AEG (additional evidence generation)
first pilot of a joint HTA process
would be worth looking this up- and see whether we agree or not


Comment from Chris Chin
When to set up a registry? At launch of a product is too late- one should start earlier, so that there is a good quality registry in place at launch and one can focus on answering questions!

Luca Pani, AIFA
differential MEA in place in Italy with the existing registry - a different MEA (management entry agreement) for every indication of the same drug
it has been a major investment for the healthcare system to have a registry of this granularity and quality standards but he thinks it has paid of!


  1. and here the registry of registries :