10 trends

The biopharma industry is increasingly harnessing the power of data collected outside of a controlled clinical environment to generate valuable insights to support products throughout their development and commercial lifecycle. This real-world evidence (RWE) is based on outcomes generated by heterogeneous patient populations in real world practice settings. Relevant data come in multiple types and forms, including but not limited to:

  • Claims data from insurance reimbursements
  • Electronic medical records
  • Pharmacy data on prescription orders and fulfillments
  • Patient registries
  • Patient-reported outcomes
  • Patient and provider survey data
  • Safety surveillance/risk evaluation and mitigation strategy (REMS)
  • Effectiveness/comparative effectiveness research (CER)
  • Health economics and outcomes research

Multiple data elements, ranging from lab results and physician notes, to genetic and regional characteristics provide a comprehensive and longitudinal picture of the patient population. By gathering this data, researchers gain greater insights into practice patterns, treatment safety and effectiveness, cost of treatment or illness, benefit/risk, diagnosis and care plans, and disease prevalence.

RWE will soon be the currency for the value-based healthcare system. Respondents to a recent IMS survey indicated that in the future, they expect to use RWE more broadly, and industry trends indicate biopharma companies and payers will increasingly partner on RWE. Experience from existing partnerships, such as that between AstraZeneca and HealthCore, suggests that RWE analysis is of highest value in examining health outcomes and the costs of chronic disease care.

FDA: Efficacy to effectiveness

Regulators are increasingly looking for evidence of both efficacy and effectiveness, and RWE is an important tool for demonstrating those traits. RWE can support regulatory approval by augmenting RCT data on the safety and efficacy of new drugs and medical devices. RWE also plays a part in FDA’s accelerated approval program, which mandates that sponsors carry out post-approval RWE studies.

A paper by two Massachusetts Institute of Technology authors criticizes FDA’s judging of clinical trial requirements by a uniform statistical standard. The study suggests that for devastating diseases, current standards are too risk-averse, which could be preventing patients access to drugs even if they are facing dire consequences and are willing to accept higher risks related to efficacy and side effects.

What does this mean for biopharma?

Increasing demand for RWE is leading to a rise in the discipline of pharmacoepidemiology, the study of the utilization and effects of drugs in large numbers of people. Historically, pharmacoepidemiology ‘played defense’ for biopharma companies by studying potential adverse drug reactions. As regulators accelerate approval pathways, these studies will also provide information on product benefits, which have traditionally been handled by the clinical development team (i.e. efficacy measurement), as well as the health economic and outcomes research (HEOR) department (i.e. value measurement). However, regulators, payers, integrated providers, patients and other stakeholders want effectiveness data to better understand ‘real-world’ performance. RWE studies these take account of the chance, bias, confounders, and effect modifiers that pharmacoepidemiologists must address in observational designs. Complementing their traditional risk equation with benefit assessment will enable them to ‘play offense’ and help pharma capture more real-world value from their agents.