Biopharmaceutical manufacturers are turning to health economics and outcomes research (HEOR) to obtain real-world evidence (RWE) that can support and improve their drugs.
Using HEOR to gather RWE helps biopharma companies address three forces reshaping how they do business:
- Competition in the biopharmaceutical manufacturing industry
- Interest from regulators, policymakers and patient advocacy groups
- Incentives from payers to enter into value-based contracts that base payment on outcomes
McKesson asked Sarah Alwardt, vice president, health informatics and HEOR at McKesson, about how HEOR works and the value RWE can bring to the biopharma clinical development and commercialization process at your organization.
How would you define HEOR as it applies to biopharmaceutical manufacturers?
Alwardt: HEOR is a very broad term that can apply to everything from drug efficacy to the value of a drug in the marketplace to different stakeholders. From our standpoint, and from the standpoint of biopharma companies, HEOR means using rich data sets to look at how drugs are being used by prescribers to treat patients and how the drugs are performing clinically with the targeted patient population. The overall intent of HEOR in this context is to answer relevant clinical questions that provide RWE and insight to biopharma companies and ultimately improve patient care.
How widely are biopharmaceutical manufacturers using HEOR to collect RWE?
Alwardt: HEOR has been around for a long time. It’s only now coming into its heyday as a method to collect RWE. Almost every single biopharma company is doing it in some form. The reason it’s coming into its own is twofold. First, the FDA has acknowledged the value of RWE in expanding the use of a biopharmaceuticals from its initial use. The initial use was based solely on clinical trial data. Second, biopharma prescribers, payers and patients have acknowledged that what happens in a clinical trial isn’t necessarily what happens in the real world after a new drug comes to market. By using HEOR to gather RWE, manufacturers can close the knowledge gap between clinical trial and real-world outcomes.
Why is there a knowledge gap between clinical trial and real-world outcomes?
Alwardt: During a clinical trial, patients meet very specific and narrow clinical and demographic criteria to participate. The pool of patients is fairly homogeneous. Patient engagement with a particular biopharmaceutical drug during a clinical trial is also different. They volunteered to participate. They typically are not paying for the drug being tested. They are also being reminded to take the new drug by a clinician who contacts them frequently and records their adherence and condition. In the real world, the mix of patients taking the new drug is much broader in terms of clinical factors and demographics. Biopharmaceuticals can affect patients very differently. And engagement is different for a variety of reasons that could affect adherence, from provider prescribing patterns to patients’ out-of-pocket costs. As a result, the drug may perform very differently after commercialization than during the clinical trial.
What type of RWE is most valuable to a biopharmaceutical manufacturer?
Alwardt: Most biopharma companies know the questions they want to ask. The questions determine the source of the RWE. The questions they most commonly ask fall into two areas: treatment patterns and drug efficacy. What is the drug treatment pattern for a specific diagnosis, like breast cancer? What drugs are commonly used as first-line therapy? What drugs are commonly used as second-line therapy? What are the clinical results of first-line and second-line drugs? What is the clinical outcome progression as patients move from first-line to second-line drugs?
Where do biopharmaceutical manufacturers obtain RWE to compare with their clinical trial results?
Alwardt: The bread and butter of using HEOR to gather RWE is retrospective analysis of data generated through an EHR. Biopharma companies are building databases of outcomes information collected from patients, prescribers, payers and pharmacies. Other biopharma companies are using databases built by third parties who collect the same type of outcomes information. Controlling for different variables, a manufacturer or a third party can run a statistical analysis on a specific cohort of patients to see which patients did well or poorly on the biopharmaceuticals and why.
How should biopharmaceutical manufacturers use RWE to close the knowledge gap?
Alwardt: The most important thing RWE gives to a biopharma company is information about how a drug is performing after approval and why, and why that performance may be the same, better or worse than the clinical trial results. It could be because of treatment patterns, patient demographics, patient comorbidities or adherence issues. It could be a combination of two or more of those factors. Armed with that information, a biopharma company can recommend changes in the use of a drug that will improve patient care. For example, a lower dose may produce the same results as in the trial but with fewer side effects. The drug may be useful in treating other medical conditions, and the biopharma company could seek FDA approval for an additional use for the same medication.
What other actions could a biopharmaceutical manufacturer take after collecting RWE?
Alwardt: Biopharma companies, or third-party vendors on their behalf, could perform a prospective analysis in addition to their retrospective analysis to obtain RWE. The retrospective analysis could identify a specific group of patients that could be surveyed about their use of the drug, how it’s affecting their health and their overall quality of life. They could also prospectively survey prescribers about prescribing patterns and treatment decisions uncovered in the retrospective analysis. The results of the prospective analysis could lead to interventions to improve use, adherence and outcomes. That could be a mobile app that reminds patients to take their medication. It could be a co-pay discount card that reduces patients’ out-of-pocket cost for their biopharmaceutical drug.
How can biopharmaceutical manufacturers use RWE to improve clinical trials?
Alwardt: They can use it to make clinical trials more pragmatic. A biopharma company may expand the clinical trial pool to include certain patients that RWE has shown would benefit from a particular type of drug or drug therapy. Maybe they were taking a different drug in combination with another. Maybe they have a genetic marker for a drug. From a biopharma clinical trial standpoint, RWE can be very exciting. It’s another example of how RWE can close the gap between clinical trial results and real-world results.
How does obtaining RWE help biopharmaceutical manufacturers from a business perspective?
Alwardt: It can help in a number of ways. It’s a way for biopharma companies to demonstrate the value of their biopharmaceuticals to payers and prescribers. It’s a way to expand the possible market for a new drug. It’s a way for companies to be precise about who would benefit most, clinically speaking, from a new drug. And it’s a way to make future clinical trials more efficient and effective.
Related: Learn more about McKesson’s Health Economics and Outcomes Research (HEOR) services for biopharma and life sciences companies.