Outcomes research

Final report: PCM Report – Clinical effectiveness

Investigator: Ulla Wilking

This is a report on aspects of Clinical Effectiveness (CEss) in relation to the Personalised Cancer Medicine (PCM) research program at Karolinska Institutet (KI).
CEss research study effects of interventions in clinical practice (real-world effects). The obtained data provide important additional information to results from clinical trials (clinical efficacy, CEy). In CEy patient populations are relatively homogenous and treated under controlled conditions. Outcomes research (OR) includes all aspects of CEss as well as costs and resources (end-result); outside the scope of this report.

As compared to Clinical Studies, CEy (homogenous patients treated under controlled conditions), CEss studies with a biological approach (biological-CEss) inform on wider biological aspects affecting treatment effects (heterogeneous patient groups treated in clinical practice) can provide increased understanding of side-effects, and may better sub-group patients. Furthermore, studies with a biological-CEss approach are only feasible in very few countries (Nordic countries, The Netherlands, the UK), as these countries have population based cancer registers, making it possible to identify all patients in a certain population.

New innovative cancer drugs are increasingly developed for small and targeted patient populations, and therefore many cancer drugs have orphan drug status, meaning that the drugs were approved based on limited data on few patients. New cancer drugs are also taking up an increasing share of the cancer health care budgets. This contributes to the importance of defining which patient benefits the most from each treatment, and hence CEss studies can be very useful.

The aim of this report is to provide an overview of CEss aspects in order to identify areas where the PCM-program at KI can facilitate CEss research in Stockholm. The overarching questions: What can be achieved by using a biological-CEss approach in PCM research? What changes or improvements are required?

CEss research comprises several challenges:

  •   Level of evidence with CEss studies related to established methods
  •   Common view of disease management
  •   Infrastructure for data collation
  •   National and international competing initiatives

There are numerous efforts in the CEss research field today, both in Sweden and internationally. These efforts use different approaches and different methods. In Stockholm, as well as elsewhere in Sweden there is little coordination and collaboration in relation to PCM research, and the initiatives result in a fragmented research environment often with doubling of efforts.

The suggestion for the PCM program, in relation to CEss, is to contribute to the formation of a structure for establishing the role of CEss as a key component in the total translational research process. The actions will need to focus on short-term, medium-term, and long-term perspectives. In the short-term the focus is awareness, training, and education, the medium-term should focus on study set-ups and collaborations. This could best be achieved within the existing structure of clinical studies. The long-term would need to focus on creating sustainable infrastructures for biological-CEss research.

The competitive research environment in Sweden does not encourage collaboration efforts. If the PCM program can establish a true translational/PCM infrastructure at KI and in Stockholm, faster (better?) results will be achieved, providing faster improvements in cancer care.

In summary, CEss research can be used

  •   to define subgroups of patients in a heterogeneous population
  •   for longitudinal healthcare databases
  •   to analyse large health care databases
  •   as ethical basis for the practice of health care
  •   for improvement in the quality of care
  •   for medical informatics
  •   for implementation research in health care
  •   as a basis for decision making in health care
  •   for decision analyses in health care