Focus on Evidence-Based Medicine: Improving Patient Outcomes and Managing Healthcare Costs

Practicing according to the principles of evidence-based medicine involves the application of national practice guidelines, current clinical research, and experience to guide the diagnosis of a condition and selection of appropriate treatment. Although this practice is important in all fi elds of healthcare today, it is becoming increasingly important in specialty pharmacy. Given that drug spending for specialty pharmaceuticals is growing at 2.5 times the rate of nonspecialty spending¹ and that total healthcare costs for specialty patients is 17.5 times higher than nonspecialty patients,² increased focus has been placed on the use of specialty medications and concern over how to manage costs while ensuring appropriate utilization and quality care.

Specialty pharmacy is a growing area of pharmacy practice focused on providing drug therapy management and dispensing services for patients requiring treatment with medications that are used to treat rare, chronic, or genetic conditions. These agents are often high cost, but more importantly, patients taking these medications require frequent monitoring and training, coordination of care with multiple healthcare providers, comprehensive patient education, and adherence management to ensure appropriate utilization. By following the principles of evidence-based medicine, specialty pharmacy is able to achieve the goal most important to patients, payers, and other healthcare practitioners—optimizing clinical treatment outcomes at the lowest cost.

An example that illustrates how the principles of evidence-based medicine can be applied is the management of hepatitis C. Affecting approximately 2.7 million people in the United States, hepatitis C is a blood-borne virus that affects the liver, causing chronic infl ammation that can lead to cirrhosis, liver failure, and even liver cancer.^3,4 Several types of hepatitis C exist—genotype 1is the most common in the United States and is the most difficult to eradicate.³ Patients are often asymptomatic, and diagnosis is usually prompted when liver abnormalities are identified during a routine medical examination. Evidence-based guidelines indicate that key factors to successful viral eradication include the use of and adherence to the current gold standard for treating hepatitis C—pegylated interferon in combination with ribavirin for up to 48 weeks.^3-5 Even with optimal medication use and adherence, there is still only a 40% to 50% success rate for genotype 1 patients, and patients that do not experience a clinical response at 12 weeks are not likely to ever achieve successful viral eradication.⁵ Further, side effects associated with these medications, including flu-like symptoms, depression, and blood dyscrasias, create a huge barrier to successful treatment, with many patients discontinuing therapy after the fi rst month.

Specialty pharmacists are in a prime position to apply best clinical practices at the patient level by helping patients understand the complexity of treating and living with hepatitis C, coaching them to remain adherent to and complete their treatment regimen, as well as monitoring their clinical response to ensure that those patients not responding early in the treatment cycle are identified so that other, more appropriate treatment options may be pursued. A recent study showed that specialty pharmacists can help hepatitis C patients achieve an average adherence of 94.5% (95% confidence interval, 94.2% and 94.7%), with greater than 90% of patients remaining on therapy beyond the first month.⁶ Further, by monitoring early treatment response, the specialty pharmacy was able to identify that approximately 12% of the population was not responding to therapy, resulting in discontinuation of the prescribed regimen and initiation of a more appropriate option, resulting in payer cost savings of approximately $1500 per month for each member identified as a nonresponder.⁷

Specialty pharmacy has demonstrated the ability to support the application of evidence-based medicine as part of the medication utilization and management process. As the fastest growing segment of drug spending and the largest proportion of the drug development pipeline, understanding the evolving evidence supporting the use of these treatment options and identifying their appropriate place in therapy will continue to be a focus for healthcare providers and payers alike. The application of these evidence-based practices is critical to ensuring the safe and effective use of specialty medications, optimizing treatment outcomes, and minimizing healthcare costs both today and in the future.