Comprehensive Evidence Development: Progress and Opportunities

With the introduction of Accountable Care Organizations in the United States—and increasing evidentiary requirements from payer organizations around the world—this is both an exciting and nerve-wracking time to be in the healthcare industry.

Richard Gliklich, MD

The business of treating patients has always been about providing care of the highest quality, but the paradigm shift in the industry today is forcing all those who are part of providing care to prove the value of that care through demonstrable real-world outcomes.

Not only do we face newly empowered stakeholders with different priorities, but old stakeholders with new priorities for the information that will drive their decisions. At the same time, the methods we must employ to develop evidence have also expanded from randomized trials to prospective observational studies and registries, to harvesting big data—including rapidly accumulating electronic health records. Ultimately, the goal is to have an evidence development framework that can answer a range of questions simultaneously and provide a more efficient, timely, and complete picture for better decision making—from approval to coverage to use.

As physicians and group practices intensify their efforts to provide patient outcomes data to satisfy this challenge, myriad initiatives and projects are underway globally to assist all stakeholders in assessing the efficacy, safety, and cost-effectiveness of medical interventions. Yet much work remains to be done. The FDA, for example, recognizes the agency's need to better assess the risk/benefit profile of new interventions (in addition to safety and efficacy), but Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research, stated at the recent Post-Approval Summit at Harvard Medical School that the agency needs better tools and training to assess new types of evidence.

Regulatory and resource challenges notwithstanding, significant progress toward developing comprehensive evidence for medical interventions can be seen in numerous areas. The ongoing Registry in Glaucoma Outcomes Research study aims to compare three treatment paths and outcomes among patients with open-angle glaucoma. This type of study will not only provide longer-term outcomes data for interventions with differences in access and costs, but will also identify sub-populations of patients who may benefit from one path versus another. The identification of treatment response—on an individual level and in the real world—is one of the hallmarks of the new world of clinical research.

Isolated streams of data, however, only provide a glimpse of an intervention's overall profile. By leveraging electronic health records, biobanks, patient registries, and data from randomized clinical trials, among others, all relevant questions about individual response to a medical intervention can be answered.

In response, biopharmaceutical companies must gain comfort with real-world outcomes data being used as a basis for reimbursement, pricing decisions and ultimately physician decision-making. Biopharma has gotten very comfortable over the past decade in approaching regulatory authorities for guidance. They must now engage in the same types of conversations with payers, providers, and patient advocacy groups—in the very early stages of a product's lifecycle—to ensure that the evidence needs of these stakeholders are being addressed in the development of new therapeutics. Coupled with a strategy to collect, analyze, and openly share outcomes data, ongoing dialogue, and coordination amongst all members of the healthcare ecosystem will ultimately enable evidence-based treatments to get to the right patients, at the right price.

Richard Gliklich, MD, President Quintiles Outcome. E-mail: richg@outcome.com