With US health spending expected to reach $3.6 trillion by 2014,1 all stakeholders are seeking ways to control cost while maintaining quality of and access to care. Experts agree that the US healthcare system must address current and emerging problems of inefficiency, administrative costs, rising prices, and inappropriate patient care, as the number of healthcare consumers continues to rise, with a higher disease burden. As a highly visible component of healthcare expenditure, pharmaceuticals have been a lightning rod for various forms of cost and utilization control. The United States spends more than $200 billion ($700 per capita) annually on prescription drugs, a figure nearly twice as high as in many other developed countries.2 Since 2000, national spending on prescription drugs has increased between 8% and 15% annually.2,3 Faced with such rising costs, employers and health benefit plans have attempted to control drug utilization and cost by limiting access to newer, more expensive medications and by encouraging appropriate drug prescribing. Managed care organizations (MCOs) primarily use management strategies to control the utilization of these agents (eg, quantity limits, prior authorization, and higher cost-sharing [ie, high copays] requirements for more expensive or novel treatments). These efforts are all direct or indirect attempts to maximize medication value (ie, cost per outcome).
One area that holds promise for increasing medication value is improving medication compliance. Failure to properly use an appropriately prescribed medication translates into suboptimal patient outcomes, which decreases the value of the medication. The cost of poor medication adherence to the US healthcare system is significant. In 2005, the cost of nonadherence was estimated to be $300 billion annually.4
Improving compliance, although certainly an appropriate target, is challenging. Measuring patient compliance in a valid, reliable, and reproducible manner is one of the key elements for any compliance improvement initiative. However, there are no measures of compliance that meet these objectives; most compliance measures rely on surrogates, such as medication possession ratios. Studies of medication adherence have been hampered by methodological problems. Many studies rely on patient self-reports of adherence, which are unreliable indicators of actual medication use.5 Other studies have used cross-sectional prescription paid claims data to estimate medication possession ratios or other metrics of patient adherence.6 Moreover, many adherence studies were designed to increase screening for a condition, or involved modification of treatment regimens, hence con founding the association of outcomes with adherence.7,8
At the manufacturer level, the most significant measure of medication utilization throughout the pharmaceutical marketplace today is market share. In this model, manufacturers use trained professionals to communicate, educate, and promote their products to clinicians based on information from distribution channels (ie, wholesale and retail distribution). Prescriber data reported by wholesale and retail distributors are matched with zip codes and prescriber identifiers to pinpoint product use within a selected geographic area.
Although this is standard practice among manufacturers, there is no evidence to support a correlation between market share and actual patient compliance; market share metrics are associated with prescribers, not with patients. Pharmaceutical manufacturers that discover and market products for chronic conditions and even for life-threatening cancers are challenged by what the data show about compliance rates or utilization over time. Market share performance continues to be the basis for contractual relations between MCOs and pharmaceutical manufacturers. The model is simple: use more medication against the national market share performance provided by the wholesale distribution report and the MCO's discount for a product increases.
Much of the gap between the promise of evidencebased medicine (EBM) and the reality of improved patient outcomes can be attributed to problems referred to as the "last mile," "medical moment of truth" (MMOT),9 or patient compliance. Studies have generally shown little relationship between the type of disease and patient adherence to treatment recommendations. Interventions found to be effective have also often been complex, making it difficult to attribute improved patient outcomes to specific elements of interventions.10
Who are the losers in the current market share discount model? As managed care has made its mark on our healthcare system, many payor organizations use strict methods of utilization and cost control to offer reasonable premiums. Employers are demanding ways to reduce their healthcare expenditures and to shift some of the associated costs to the consumers. Pharmaceutical companies are poised to lose the battle on all fronts: government price controls or price negotiations are looming, benefit designs are becoming more restrictive, and historical managed care control mechanisms (eg, prior authorizations, step edits, quantity limits, and National Drug Code blocks) continue to be used in contrast to promoting compliance and access.
How do we address the current situation and motivate consumers and payors to "do the right thing?" The market requires a novel, patient-centered compliance metric, with incentives properly aligned for consumers and payors that will optimize the return on investment for pharmaceutical manufacturers who invest billions of dollars to create and bring their treatments to arguably the safest and best healthcare market in the world.
The picture is complex, and the evidence that we can improve compliance by increasing access is weak; studies that have successfully linked physician prescriptions to actual claims data suffer from time lag and have not been able to provide adherence data in real-time to allow for prospective or concurrent interventions.11,12 These delays result in a lost opportunity to influence patient behavior during the MMOT. Furthermore, interventions aimed only at the "supply side," (eg, in doctors' offices at the time of prescription writing) have failed. Decision support aids, including medication timetables and consumer product information, have not improved compliance.13 Interventions on the "demand side" may improve compliance, provided that physicians can prospectively identify patients who would likely demonstrate poor compliance. Real-time claim-derived data on compliance can give prescribers the information and impetus to provide additional patient education that will motivate patients to adhere to their regimens.14 Taken together, these factors point to a clear need for a novel patient-centered measure of performance that (1) prospectively identifies nonadherent patients in real-time, (2) allows for timely interventions in these patients, and (3) evaluates changes in adherence and clinical end points over time, all of which drive event reduction and improve morbidity and mortality.
We propose a new metric that can assess compliance within a pharmaceutical category is needed that will enable payors and manufacturers to calculate baseline compliance rate within that category. With a nationwide emphasis on quality improvement, e-health initiatives, and EBM, the time for change is now. Along with manufacturers, employers, consumers, and governments (state and federal) are beginning to develop compliance patient-centered tools by packaging products and services in ways that enable consumers to succeed with their treatments. Moving beyond a pillbased, product-only approach, disease management organizations, employers, and consumers appear ready and willing to invest in this area with the confidence that better compliance will lead to healthier, more productive employees and consumers.