In the recent extension of the Prescription Drug User Fee Act, the risk management programs that are to be funded by additional user fees combine ascertainment of risks in the initial postapproval phase with activities to confront these risks. Better recognition of risks, a prerequisite for success in managing risks, will require strategies to supplement the present system of spontaneous event reporting. This overview considers the merits of pharmacoepidemiology studies in examining gaps in drug safety resulting from the inherent limitations of clinical trials. After drugs become widely used following regulatory approval, many health outcomes potentially related to drug exposures can be studied in large health insurance claims databases using pharmacoepidemiology designs tailored to this venue. We clarify types of safety investigations particularly suited to examination in a large claims database, as well as the benefits and caveats of expanding postapproval follow-up through Prescription Drug User Fee Act funding.
US General Accounting Office.Food and Drug Administration: Effect of User Fees on Drug Approval Times, Withdrawals, and Other Agency Activities. Report to the Chairman, Committee on Health, Education, Labor, and Pensions, US Senate. GAO-02–958. http://www.gao.org. Accessed Oct 14, 2002.
2.
StromB. What is pharmacoepidemiology? In: StromBL, ed. Pharmacoepidemiology.Third edition. New York, NY: John Wiley & Sons, Ltd; 2000:3–16.
3.
US Food and Drug Administration.Managing the Risks from Medical Product Use: Creating a Risk Management Framework. Report to the FDA Commissioner from the Task Force on Risk Management. May 1999. http://www.fda.gov/medwatch/articles.htm. Accessed Oct 14, 2002.
4.
GlasserDBDieckGS. A view from industry. In: StromBL, ed. Pharmacoepidemiology.Third edition. New York, NY: John Wiley & Sons. Ltd; 2000:91–108.
5.
BenayounSErnstP.SuissaS. The impact of combined inhaled bronchodilator therapy in the treatment of COPD. Chest.2001;119:85–92.
6.
SuissaSErnstP. Inhaled corticosteroids: Impact on asthma morbidity and mortality. J Allergy Clin Immunol.2001;107:937–944.
7.
DownsJRClearfieldMWeisSWhitneyEShapiroDRBeerePAPrimary prevention of acute coronary events with lovastatin in men and women with average cholesterol levels: results of AFCAPS/TexCAPS, Air Force/Texas coronary atheroschlerosis prevention study. JAMA.1998;279:1615–1622.
8.
PradhanADMansonJERossouwJESiscovickDSMoutonCP. Inflamatory biomarkers, hormone replacement therapy, and incident coronary heart disease: prospective analysis from the Women's Health Initiative observational study. JAMA.2002;288(8):980–987.
9.
ICD-9-CM.International Classification of Diseases 9th Revision Clinical Modification.Sixth Edition. Professional for Hospitals Volumes 1,2,3. HartACHopkinsCA, eds. Salt Lake City, UT: Ingenix, Inc.; 2002.
10.
US Food and Drug Administration. Center for Drug Evaluation and Research. Postmarketing surveillance and epidemiology: Human drugs (Chap 13). September 30, 1999. http://www.fda.gov/cder/aers/chapter53.htm. Accessed Oct 14, 2002.
11.
KennedyDLGoldmanSALillieRB. Spontaneous reporting in the United States. In: StromBL. ed. Pharmacoepidemiology.Third edition. New York, NY: John Wiley & Sons, Ltd; 2000:151–174.
12.
WoodAJ. Thrombotic thrombocytopenic purpura and clopidogrel: a need for new approaches to drug safety. N Engl J Med.2000;342:1824–1826.
13.
LasserKEAllenPDWoolhandlerSJHimmelsteinDUWolfeSMBorDH. Timing of new black box warnings and withdrawals for prescription medications. JAMA.2002; 287:2215–2220.
14.
GarbeESuissaSLe LorierJ. Association of inhaled corticosterioid use with cataract extraction in elderly patients. JAMA.1998;280:539–543.
15.
JosephKSBlaisLErnstPSuissaS. Increased morbidity and mortality related asthma among asthmatic patients who use major tranquillisers. Br Med J.1996;312:79–82.
16.
KelseyJLWhittemoreASEvansASThompsonWD.1996.
17.
KirkingDMAmmannMAHarringtonCA. Comparison of medical records and prescription claims files in documenting prescription medication therapy. J Pharmacoepidemiol.1996;5:3–15.
18.
StromBLCarsonJLHalpernACSchinnarRSnyderESShawMTilsonHHJosephMDaiWSChenDA population-based study of Stevens-Johnson syndrome. Incidence and antecedent drug exposures. Arch Dermatol.1991;127:831–838.
19.
WestSLStromBLPooleC. Validity of pharmacoepidemiology drug and diagnosis data. In: StromBL, ed. Pharmacoepidemiology.Third edition. New York, NY: John Wiley & Sons, Ltd, 2000:661–706.
20.
KronickRGilmerTDreyfusTLeeL. Improving health-based payment for Medicaid beneficiaries: CDPS. Health Care Financ Rev.2000;21:29–64.
21.
TaylorDHJr.FillenbaumGGEzellME. The accuracy of medicare claims data in identifying Alzheimer's disease. J Clin Epidemiol.2002;55:929–937.
22.
MarkowitzJSPashkoSGuttermanEMLinde-ZwirbleWTNewboldRIII. Death rates among patients hospitalized with community-acquired pneumonia: A reexamination with data from three states. Am J Pub Health.1996;86:1152–1154.
23.
HemmelgarnBSuissaSHuangABolvinJPinardG. Benzodiazepine use and the risk of motor vehicle crash in the elderly. JAMA.1997;278:27–31.
SteinmanMAGonzalesRLinderJALandefeldCS. Changing use of antibiotics in community-based outpatient practice, 1991–1999. Ann Int Med.2003;138:525–533.
26.
SuissaA. Novel approaches to pharmacoepidemiology study design and statistical analysis. In: StromBL, ed. Pharmacoepidemiology.Third edition. New York, NY: John Wiley & Sons, Ltd; 2000:785–807.
27.
GuttermanEMMarkowitzJSLewisBFillitH. Cost of Alzheimer's disease and other dementia in Managed-Medicare. J Am Geriatr Soc.1999;47:1065–1071.
28.
CharlsonMEPompeiPAlesKLMacKenzieCR. A new method of classifying prognostic comorbidity in longitudinal studies: Development and validation. J Chronic Dis.1987;40:373–382.
29.
DeyoRACherkinDCCiolMAAdapting a clinical comorbidity index for use with ICD-9-CM administrative databases. J Clin Epidemiol.1992;45:613–619.
30.
EngelS. The new FDA. R & D Directions.2002;8(5):42–44.
31.
WolfeSPeckBClementeF. Comments on: the Prescription Drug User Fee Act (PDUFA). Public Citizen Health Research Group and Congress Watch. Docket No. 01N-0450. http://www.citizen.org/publications/release.cfm?ID=7145. Accessed Sept 25, 2002.
32.
StromB. When should one perform pharmacoepidemiology studies? In: StromBL, ed. Pharmacoepidemiology.Third edition. New York, NY: John Wiley & Sons, Ltd; 2000:63–72.
33.
FriedbergMSaffranBStinsonTJNelsonWBennettCL. Evaluation of conflict of interest in economic analyses of new drugs used in oncology. JAMA.1999; 282:1453–1457.
