In today's cost-constrained healthcare environment, stakeholders need not only evidence on efficacy and safety, but also information that allows them to assess multiple competing product claims within their own healthcare systems. The evolving scientific discipline of outcomes research has been able to provide a critically important set of research methods for pharma companies to demonstrate product value to the sometimes competing, perspectives of regulators, payers, patients, pharmacists and physicians.
In an attempt to clarify some inconsistencies in terminology being used to describe a range of overlapping research methodologies, the International Society for Pharmacoeconomic and Outcomes Research (ISPOR) has defined outcomes research as "a scientific discipline which evaluates the effect of health care interventions on patient related clinical, humanistic and economic outcomes."
Clinical outcomes considered include:
Humanistic outcomes, increasingly referred to as patient reported outcomes (PROs) include:
•Health Related Quality of Life (HRQoL)
• Compliance and persistence
• Satisfaction with treatment/medication
• Preference/willingness to pay
Economic outcomes evaluated comprise:
• Units of resource use
Components of these three categories of outcomes are being used individually or as composite measures to demonstrate product value to varying target audiences using endpoints such as:
• Survival and disease-free survival
• Satisfaction with intervention or with overall treatment
• Resources saved
• Incremental cost effectiveness
• Cost per quality adjusted life year
Each category of outcomes research has its own set of scientific and regulatory issues. For example HRQoL is assessed in a similar way to a clinical end point but to produce credible results, an instrument which is sensitive to change and produces findings which are complementary to those from clinical outcomes is necessary.
Also, as HRQoL measurement usually involves assessing multiple endpoints, attaining consistent results can be difficult and findings have to be carefully interpreted. A growing inclination by manufacturers to use HRQoL value claims has prompted the development of regulatory guidelines on the design, use and reporting of findings relating to HRQoL. Health economic analyses can be very complex and some of the major challenges is ensuring the relevance of evaluations to national HTA and /or re-imbursement requirements as well the communication of findings in a way that is relevant to decision makers. There are also differing national guidelines on the performance of these studies.
As a result of the ever increasing hurdles to achieve optimum market access, outcomes research is now being used to support a product throughout its life cycle to meet the demands for evidence from different stakeholders. Consequently there has been an increase in the performance of Phase III trials, peri-approval and post-marketing studies that inform on humanistic and economic outcomes in addition to clinical outcomes.
PAREXEL's Peri/Post-Approval Services group has a respected team of health outcome professionals and an international network of senior consultants to provide input to study design and local requirements for international studies. To discuss your health outcome study requirement, contact the Peri/Post-Approval Services group.