Outcomes Data: A Critical Insight for Market Access

While clinical trial data is critically important to advancing the science of oncology, there is an important need for additional clinical insights to guide treatment decisions. Comparative prospective trials can bring valuable information into therapeutic superiority, and health economic outcomes research (HEOR) generally can provide insight into the determinants of a treatment and an outcome, sequencing strategies, and relative effectiveness between competitors within a given indication.

Today, all stakeholders throughout the US healthcare arena are making greater use of HEOR, which involves using a variety of mechanisms to gather and assess true clinical benefits and cost effectiveness in real-world settings. The goal, from the standpoint of the pharmaceutical and biotech companies, is to augment the clinical data package in order to clearly articulate the product’s full value proposition. Such mechanisms include:

· Clinical-effectiveness studies to find out how a product performs in real-world settings.

· Comparative-effectiveness studies to compare Drug A and Drug B, based on a specific set of criteria.

· Patient-reported outcomes data to capture the patient’s experience in taking a particular drug, including ease of administration, quality of life, ability to function and so on.

· Cost-effectiveness studies to help determine how Drug A compares to Drug B when you account for efficacy, safety and costs.

Traditionally, Phase IIII clinical trials, which are conducted in accordance with specific protocols with clear inclusion/exclusion criteria, are meant to demonstrate the drug’s impact on specific safety and efficacy endpoints relative to a control agent. By comparison, HEOR data can provide a more-realistic picture of how specific therapeutic options perform under non-ideal conditions, in real-world settings. Notably, HEOR trials often represent treatment patterns outside of major academic centers, which can be very relevant as 80% of oncology in the U.S. is practiced in the community setting. It’s only by combining clinical trials and HEOR studies that the different stakeholders can begin to understand the benefits and limitations of the treatments available.

Moving HEOR to the forefront

Once considered largely a support function, the need for, and use of, HEOR data is now being recognized as a central component to the overall brand strategy of any biopharmaceutical product (Figure 1). Today, all stakeholders in the healthcare arena — ranging from patients and physicians, to drug developers, regulators and payers — are able to make treatment decisions and improve patient care through the use of HEOR data.

Payers. The insurance community’s desire to identify the most cost-effective treatment options is more important than ever, as US healthcare costs have continued to rise in the face of numerous drivers, such as:

• Over-reliance on hospitals and emergency room settings rather than lower-cost practice environments for routine healthcare

• The need for physicians to adapt to new technologies and costly laboratory tests

• Growing administrative costs associated with the healthcare plans

• Varying treatment strategies when no prospective comparative data exists.

Payers are demanding HEOR data that can provide evidence that a drug is “worth paying for.” This is especially important in therapeutic categories for which there are numerous competing treatment options, and where costs need to be controlled. More often than not, payers rely on cost-effectiveness data to help justify reimbursement and formulary-designation decisions, to cut costs and reduce inappropriate medication use.

Physicians. In recent years, across many disease states, the sheer number of drug options in a given therapeutic category has increased dramatically. While this proliferation of options is promising on many levels, it can also create considerable challenges for prescribers. Prospective (and retrospective in the absence of prospective) comparative effectiveness data can greatly guide the treatment decision process. Data on optimal therapy sequencing can be of particular value as well, and patient reported outcomes (PRO) data help physicians factor in the patient experience with regards to varying treatment choices, including meaningful health-related quality of life indicators.

Across a variety of disease states, US physicians and payers have been placing growing emphasis on the use of formalized Treatment Guidelines or Clinical Pathways to help guide treatment decisions and increase standardization. Such treatment protocols are typically developed, published and updated by independent committees comprised of physicians, pharmacists and other experts — such as the National Cancer Care Network (NCCN), The US Oncology Network, and many others. As both payers and physicians are increasingly cost-sensitive when it comes to treatment decisions, some pathways reflect not only the evidence of efficacy and safety, but also include treatment costs as criteria for recommendations.

Armed with such information, prescribers are able to evaluate competing options more critically, favoring those that demonstrate the best balance among key considerations of safety, efficacy and cost.

Patients. Today’s patients face higher-than-ever out-of-pocket costs for healthcare, in terms of higher plan premiums, higher co-payments and co-insurance requirements and plan limits. And thanks to increased overall longevity in many disease states, many patients will require longer durations of therapy to manage chronic conditions. As educated consumers, many of today’s patients are becoming increasingly vested in getting more value out of their healthcare delivery system.

Regulators. HEOR data can also support the regulatory-approval process for prescription therapies. In recent years, US regulators have been putting increased emphasis on PRO data to make sure that the clinical benefits delivered by a specific drug are both demonstrable and meaningful to patients in real-world settings.

For instance, in its “Guidance for Industry” document around PROs, the U.S. Food and Drug Administration (FDA) states that “findings measured by a well-defined and reliable PRO instrument in appropriately designed investigation(s) can be used to support a claim in medical product labeling if the claim is consistent with the instrument’s documented measurement capability.” The FDA goes on to say that the “use of a PRO instrument is advised when measuring a concept best known by the patient or best measured from the patient perspective.”[i]

To date, several prescription drugs have received FDA approval for labeling claims based on PRO, including oncology drugs. The FDA has stated that for product-approval decisions in oncology, the agency will focus on “endpoints that demonstrate a longer life or a better life or a favorable effect on an established surrogate for a longer life or a better life.”

Putting HEOR to work

As demand for HEOR data grows among all stakeholders in the US healthcare arena, the onus is on drug developers to develop a complete, cogent HEOR strategy for each product that can demonstrate safety and clinical effectiveness, as well as economic performance in a real-world setting. The availability of robust, outcomes-related data can both ensure favorable coverage and formulary decisions from private and government payers, and encourage the most-appropriate prescribing practices among physicians and product acceptance among patients. To get started, drug companies should consider the following questions:

· How robust and comprehensive is your overall HEOR strategy?

· Is your HEOR strategy integrated and complementary to your R&D, reimbursement and commercialization strategies?

· Does your strategy address the unique needs of all key stakeholder groups?

It is important for drug developers to recognize that the strategic use of HEOR data is essential during all phases of the product lifecycle:

· During drug development. PROs should be included in the pivotal clinical trial design to create compelling support for regulatory approval.

· At product launch. Comparative-effectiveness studies, cost-effectiveness studies and a solid value dossier that can demonstrate not just the required safety and efficacy profile, but clinical effectiveness or financial advantage as well, are important as U.S. payers are assigning products to formularies and making reimbursement decisions.

· During product adoption. At this point in the product lifecycle, real-world effectiveness studies of the product in use, and comparative-analysis studies of competing products in the same disease class, can influence prescribing habits in a way that drives product use and increases market share.

As the needs and demands for HEOR data among U.S. stakeholders has increased steadily over the past years, McKesson Specialty Health has seen its volume of HEOR studies and publications growing by 10-fold between the early 2000’s to the early 2010’s. By combining the expertise of outcomes researchers and physicians with unique data assets that contain deep clinical and financial information, we support pharmaceutical and biotech companies to gather HEOR insights helpful to all stakeholders as part of the overall brand strategy. Such an undertaking most often brings significant payback. Armed with such information, the company is better positioned to hone its marketing outreach efforts, justify its drug pricing, inform reimbursement and drug formulary designations among private and government payers, and support better-informed prescribing practices. All of these outcomes will provide critical strategic insight, drive clinical improvements, and cost savings throughout the U.S healthcare arena.

About the authors

Debra Patt, M.D., M.P.H, serves as a Medical Director of the Pathways Task Force for the US Oncology Network and is Medical Director of Healthcare Informatics, McKesson Specialty Health.

Claude Bergeron, M.Sc., A.S.A, is Senior Director, Pharmaceutical & Biotech Solutions, McKesson Specialty Health.


[i] FDA Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims, December 2009.