How Will Demands for Effectiveness Evidence Change Drug Development?

Changes in the clinical and economic milieu of health care systems worldwide are creating changes in the evidence required for both regulatory approval and reimbursement. This includes in particular evidence about how well new treatments work compared with existing alternatives in the ‘real world’, i.e. as used by clinicians in everyday practice. Such research is commonly known as comparative effectiveness research (CER) in the US and relative effectiveness (RE) research in Europe.

The project reported in this Occasional Paper was intended to determine how changing demands for evidence are affecting drug development in five global pharmaceutical companies: Amgen, Eli Lilly, GSK, Novartis and Sanofi-Aventis[1].  A literature review helped elucidate concepts and define focus. The authors then conducted semi-structured interviews with an international sample of 19 senior pharmaceutical executives in various positions in the five companies: R&D, outcomes research, medical affairs, and pricing and reimbursement. The intent was to capture information about the effect of CER/RE requirements on the drug development process now and in the future.

Five common themes emerged from the interviews. Explored in far greater detail in the paper, these are as follows.

  • The drug development process has begun to change to deliver more CER/RE evidence. Currently, this is primarily payer focused, but the respondents highlighted the importance of better capturing patients’ perspectives.
  • The development phase during which CER/RE investments are made varies across companies. Typically, however, this usually is after the investigational compound has satisfied safety and early efficacy requirements.
  • A number of barriers to fuller incorporation of CER/RE into the drug development process exist. These include corporate structures and cultures that do not yet provide strong incentives, the cost of such research and, particularly in the US, promotional and regulatory complexities.
  • Factors that facilitate integration of CER/RE into drug development include, for example, a senior leader in the company who acts as champion, highly talented CER/RE researchers and external pressures.
  • The role of CER/RE increasingly will influence the drug development process. As yet unclear is how great that influence will be and whether CER/RE will provide a positive return on investment.

In their conclusions, the authors identify several issues that must be resolved if CER/RE is to become more integrated into drug development.

  • The willingness of industry, HTA bodies and regulators to work more closely together in designing workable, realistic approaches that integrate CER/RE
  • The use of active comparators in clinical trials and indirect comparisons
  • The impact of CER/RE evidence requirements on the cost of drug development, including an increase in costs as well as earlier discontinuation of compounds based on their CER/RE profiles
  • Changes in corporate culture that enable more effective collaboration across disciplines and explicit incentives for considering the perspectives of all stakeholders

For additional information, please contact Jorge Mestre-Ferrandiz. 

Download Mestre-Ferrandiz, J., Deverka, P., Pistollato, M. and Rosenberg, E., 2014. The current drug development paradigm: responding to US and European demands for evidence of comparative effectiveness and relative effectiveness. Occasional Paper, 14/01. London: Office of Health Economics.

[1] The project was funded jointly by these companies. Of course, any views expressed in the paper are solely the responsibility of the authors.    

Posted in Drug Development/R&D, Health Technology Assessment | Tagged Occasional Papers