Outcomes of Pharmaceutical Therapy Program (OPT) Update
Understanding which agents work for which patients and at what cost is important in managing the selection of pharmaceutical therapies and services in a changing health care environment. The Agency for Healthcare Research and Quality (AHRQ) develops and administers research programs related to patient outcomes associated with pharmaceutical therapy.
This program update summarizes research results and lists published papers to date from AHRQ's research portfolio on pharmaceutical therapy.
Impact of Pharmaceuticals on Health Care Delivery
The Need for Research
AHRQ's Pharmaceutical Research Program
Overview of Study Findings
The Cost and Economics of Health Care
Tools for Patient Management
Target Populations: Racial and Ethnic Groups, the Elderly
Public Health and Prevention, and Chronic and Persistent Disease
RFA HS-92-03 Projects: Study of Patient Outcomes Associated with Pharmaceutical Therapy
RFA HS-96-003 Projects: Research on the Outcomes of Pharmaceutical Therapy
Appendix A. Other Pharmaceutical-Related Projects
Appendix B. CERTs Fact Sheet
Impact of Pharmaceuticals on Health Care Delivery
Prescription and over-the-counter pharmaceuticals are central to many of the most challenging issues faced in health services delivery and financing today. Representing a major portion of health care dollars spent in the United States, the role and management of pharmaceuticals raise multiple complex questions for providers, patients, policymakers, and researchers. During the past decade significant advances have been made in developing tools to help providers evaluate current clinical research information as it applies to individual patients.
Critical appraisal and evidence-based techniques continue to evolve as new technical approaches to analyzing research data are developed. The cost of pharmaceuticals continues to be one of the most debated issues of this decade as Medicare, Medicaid, and private insurance programs seek to work within public budgets and/or private competitive products while at the same time making available the best that medical treatment has to offer.
Understanding which agents work for which patients and at what cost is important in managing the selection of pharmaceutical therapies and services within a changing health care environment. However, this information is often not available for many drugs, since the Food and Drug Administration (FDA) approval process only requires pharmaceutical manufacturers to provide evidence of safety and efficacy for one clinical indication and requires pharmaceutical manufacturers to provide evidence of safety and efficacy for one clinical indication and only within controlled clinical trials that often include a relatively homogeneous population. An approved drug may enter the market with relatively little information available to the practitioner, the third-party payer, or the patient about how the drug compares with the array of other therapies available. Once approved by the FDA, medications may legally be prescribed for any use deemed appropriate by a licensed physician.
Additionally, patients receiving the medication may differ from patients included in the trials. They may be sicker, older, younger, or have additional diseases. Frequently, adherence to the prescribed regimen may be different than in trials.
Within the U.S. Department of Health and Human Services, AHRQ supports the study of the relative effectiveness, appropriateness, and cost-effectiveness of alternative strategies for the prevention, diagnosis, treatment, and management of clinical conditions. The Center for Outcomes and Evidence (COE) has the lead responsibility in AHRQ for the development and administration of research programs related to patient outcomes associated with pharmaceutical therapy.
A total of 26 research grants have been funded under three COE programs. Sixteen were funded through COE's Study of Patient Outcomes Associated with Pharmaceutical Therapy, which focused on:
- Data and analytic methods involved in the study of drug therapy effectiveness.
- Factors affecting the appropriateness of drug prescribing.
- The role of the patient in drug therapy effectiveness.
Two additional projects were funded under the program Research on the Outcomes of Pharmaceutical Therapy, which gave preference to projects focusing on pharmaceutical economic analysis and effects of changes in the health care environment on patient outcomes.
The remaining seven projects and a coordinating center were funded under the Centers for Education and Research on Therapeutics (CERTs) program, a national initiative to increase awareness of the benefits and risks of new, existing, or combined uses of therapeutics through education and research. Additional information about this program is contained in the CERTs Fact Sheet.
In the context of these research objectives, Pharmaceutical Outcomes and CERTs address many of today's most critical health care issues, including those related to treatment effectiveness, the cost and quality of care, research and patient management tools that support evidence-based practice, racial and ethnic disparities in service delivery, prevalent chronic conditions in the U.S. population, health care prevention, and the needs of the elderly.
Following are some of the notable study findings related to these areas. The grant number is included so that the original publication may be located further on in this document.
- A study examining short-term outcomes in diabetic patients found that improved glycemic control of the patients' diabetes was associated with substantial short-term quality of life and health economic benefits (Testa, Simonson, 1999). Grant HS07767.
- A study examining the difference in quality of life produced by the same hypertension treatment (calcium channel blockers) using two different medication delivery systems (nifedipine gastrointestinal therapeutic system and amlodipine) suggested medication-specific effects on quality of life may be due to difference in delivery (Testa, Turner, Simonson, Krafcik, Calvo, Luque-Otero, 1998). Grant HS07767.
- Focus groups of HIV-infected individuals were conducted to assess how the HIV wasting syndrome had affected them in several areas (physical, psychological, social). Generic measures of quality of life fail to provide the sensitivity and depth required to evaluate major issues raised by the focus group participants with respect to the specific impact of HIV wasting on their lives (Testa, Lenderking, 1999). Grant HS07767.
- A retrospective cohort study that used linked New Jersey Medicare and drug claims data found that the use of long-acting dihydropyridine calcium channel blockers after acute myocardial infarction (AMI) was associated with substantially lower rates of re-hospitalization and death compared with use of their short-acting counterparts (Gillman, Ross-Degnan, McLaughlin, Gao, Spiegelman, Hertzmark, Goldman, Soumerai, 1999). Grant HS07631.
- Only 21 percent of eligible New Jersey Medicare beneficiaries who survived an acute myocardial infarction (AMI) receive beta blocker therapy following the heart attack. These patients were almost three times more likely to receive a new prescription for a calcium channel blocker than for a beta blocker after their AMI. Eligible patients receiving calcium channel blockers instead of beta blockers doubled their risk of death (Soumerai, McLaughlin, Spiegelman, 1997). Grant HS07631.
- The Lyme Disease Project calculated the incidence of Lyme disease in the 24 Maryland jurisdictions by gender and age from 1993 through 1996. The State's overall incidence rate was 7 cases per 100,000 inhabitants, with males being 1.2 times more likely to be affected than females, peaking during the age groups 10-19, 20-29, and 50-59 (Pena, Strickland, 1999). The Project also conducted an epidemiological assessment of antibiotics prescribed by physicians in Maryland to treat Lyme disease between 1993 and 1995 (Pena, Mathews, Siddiqi, Strickland, 1999). Grant HS07813.
- Thirty percent of children treated for new-onset acute otitis media in Colorado's fee-for-service Medicaid program received expensive antibiotic therapy, rather than less costly, equally efficacious products. More expensive antibiotics accounted for approximately 77 percent of the dollars spent for medications to treat otitis media in this population, but were not associated with better outcomes (Berman, Byrnes, Bondy, Smith, Lezzotte, 1997). Grant HS07816.
- Prior authorization by Tennessee Medicaid programs for expensive arthritis medications (non-steroidal anti-inflammatories, or NSAIDs) reduced prescription NSAID expenditures by 53 percent over 2 years and saved $12.8 million, without increasing the use of other medical services or less desirable drugs (Smalley, Griffin, Fought, Sullivan, Ray, 1995). Grant HS07768.
- The impact of a three-prescription per month cap in New Hampshire on the use of resources by Medicaid enrollees with schizophrenia resulted in reductions in the use of drugs, coincident increases in visits to community mental health clinics, and dramatic increases in the use of emergency services. Limits on prescription drug coverage may increase the use of acute mental health services among low-income patients with chronic mental illnesses and increase costs to the government (Soumerai, McLaughlin, Ross-Degnan, Casteris, Bollini, 1994). Grant HS07631.
- An automated Web-based patient interview tool was developed to elicit individuals' willingness-to-pay (WTP) utilities under conditions of uncertainty and to evaluate the tool's potential usefulness for clinical decisionmaking (Flowers, Garber, Bergen, Lenert, 1997). Grant HS07818.
- This randomized controlled trial utilized a pharmacy intervention group and two control groups to develop algorithms to facilitate pharmaceutical care for the treatment of asthma. Similar levels of medication compliance and health related quality of life improvement were observed in the pharmaceutical care group and in both control groups (Weinberger, Murray, Marrero, Brewer, Lykens, Harris, Tierney,�2001). Grant HS09083.
- A study to incorporate complex heart failure treatment guidelines into a physician-interactive network of microcomputer stations revealed key challenges for the development of interactive information systems, including the need for explicit definitions and the need to account for co-morbid conditions, concurrent drug therapy, timing of interventions, and followup (Tierney, Overhage, Takesue, Harris, Murray, Vargo, McDonald, 1995). Grant HS07763.
- A study evaluating the validity of patient report, pharmacy dispensing records, and pill counts as measures of medication adherence found that: pharmacy dispensing records demonstrate predictive validity as measures of cumulative exposure and gaps in medication supply; adherence levels determined from pill counts and pharmacy dispensing records correlate more closely with quantity than with dose timing; and patient-reported non-adherence is a valid predictor of reduced adherence (Choo, Rand, Inui, Lee, Cain, Cordeiro-Breault, Canning, Platt, 1998). Grant HS07821.
- An evaluation of the relationship between non-steroidal anti-inflammatory drugs (NSAID) use in community-dwelling elderly found no substantial protective effect of prescription NSAID use on cognitive function, and in fact suggested that higher doses may be associated with memory deterioration in this population (Hanlon, Schmader, Landerman, Horner, Fillenbaum, Pieper, Wall, Koronkowski, Cohen, 1997). Grant HS07819.
- Inadequate routine primary care among African American patients may increase their risk of asthma exacerbation and hospitalization, and is associated with higher emergency department use compared with white males (Murray, Stang, Tierney, 1997). Grant HS07763.
- Researches found racial disparities in the receipt of medication to prevent Pneumocystis carinii pneumonia (PCP) among patient with HIV disease (Moore, Stanton, Gopalan, Chaisson, 1994). Grant HS07809.
- Among patients with HIV infection who received medical care from a single urban center, there were no differences in disease progression or survival associated with gender, race, injection drug use, or socioeconomic status. Differences found in other studies may reflect differential use, quality, and access to care (Chaisson, Keruly, Moore, 1995). Grant HS07809.
- Two randomized controlled trials, one for community dwelling elderly and the other for nursing home patients, were conducted to reduce the use of non-steroidal anti-inflammatories (NSAIDs) for osteoarthritis in an elderly population. The nursing home intervention effectively reduced NSAID use, while the community-dwelling intervention only modestly reduced NSAID exposure among elderly patients without undesirable substitution of other medications (Stein, Griffin, Taylor, Pichert, Brandt, Ray, 2001). Grant HS10385.
- An assessment of the long-term safety of adjunctive corticosteroids in the treatment of Pneumocystis carinii pneumonia (PCP) found that adjunctive corticosteroids do not increase mortality or the risk of most common HIV-associated complications (Gallant, Chaisson, Moore, 1998). Grant HS07909.
- Researchers concluded that active injection drug users who are HIV positive but without clinical disease have less contact with health care providers and are less likely to receive antiretroviral therapy than other HIV positive patients (Celentano, Vlahor, Cohn, Shadle, Obasanjo, Moore, 1998). Grant HS07809.
- An analysis of information stored in electronic medical records for patients with evidence of ischemic heart disease or heart failure successfully predicted patients at high risk for mortality. Routine clinical data store in patient's electronic medical records are capable of predicting mortality and can be used to focus increasingly scarce health resources on those at highest mortality risk (Tierney, Takesue, Vargo, Zhou, 1996). Grant HS07809.
- A retrospective cohort study examined factors associated with failure to suppress HIV-1 RNA levels and adverse drug reactions in patients receiving protease inhibitor-containing therapy. The study concluded that patients in whom highly active antiretroviral therapy (HAART) is started in a clinical setting achieved viral suppression substantially less frequently than patients in controlled trials (Lucas, Chaisson, Moore, 1999). Grant HS07809.
These grants answer many important questions regarding the management of drug prescribing. Their findings are successfully being used by organizations such as the Centers for Medicare & Medicaid Services to evaluate programs that seek to manage care and improve quality and outcomes. In addition to studies funded through the Pharmaceutical Outcomes and CERTs programs, AHRQ has other projects in its portfolio that, although not solely focused on pharmaceuticals, have findings of interest to the pharmaceutical research community. These are listed in Appendix A.
AHRQ Publication No. 04-R205
Current as of June 2004
Outcomes of Pharmaceutical Therapy Program (OPT) Update. AHRQ Publication No. 04-R205, June 2004. Agency for Healthcare Research and Quality, Rockville, MD. https://www.ahrq.gov/clinic/pharmtherapy/optupdat.htm