fda and drug approval

FDA and Drug Approval! Gerald A. Faich MD, MPW Index term: Drugs JVIR 1995; 6:24S 1 Address correspondence to G.A.F., Phar- maceutical SafetyAssessments, Inc, 104 Foxhall Lane, Narberth, PA 19072. 2 Paid consultant for Abbott Laboratories. © SCVIR, 1995 DRUG regulation spans from animal studies through the postmarketing period. Developmental studies (phases I-III), approval, labeling, and cost-effectiveness are extensively addressed by the U.S. Food and Drug Administration (FDA). Unlabeled use, promotion, and cost- effectiveness claims have received particular attention in recent years. Typically, drug development requires 8-10 years including investigative (preclinical and clinical-IND) work and time for New Drug Appli- cation (NDA) preparation, submission, and approval. There has been some shortening of FDA review time from 24 to 19 months in the past 2 years. Nonetheless, the process of drug development and approval is remarkably protracted, difficult, and expensive. Some estimates are that it costs over $300 million to bring a new drug to market. Drug approval is dependent on substantiation of efficacy and safety based largely on two well-performed, adequate, randomized, controlled trials conducted by qualified experts. Clinical and statistical differences between the active agent and placebo, surgery, and/or another active agent must be shown. The definition of primary and secondary outcome measurements, power estimates, and monitoring for protocol adherence and data verification are crucial to the successful conduct oftrialso Once the agent is approved, the product label (package insert) is developed by the drug sponsor and approved by the FDA. This describes indications, doses, efficacy, and safety of the product. Such labeling should be seen as a contract between the FDA and the sponsor for the allowable use claims of the product. While initial marketing and labeling are strictly regulated by the FDA, the agency does not regulate the unlabeled use of marketed drugs by practitioners. However, pharmaceutical manufacturers are prohibited from promoting off-label use. This prohibition extends to the need to ensure only "hands-off' involvement in continuing medical education where objectivity, fair balance, and independence of program conduct must be maintained. Cost-effectiveness studies and associated claims have become increasingly important to both managed care and the FDA. Usual phase III trials examine drug effects in the highly atypical setting of carefully selected patients and investigators with intense monitoring of dosing and outcomes. While having high internal validity, generalizing to "real world" patients and use is often difficult. Preapproval data may be limited by duration oftrials, the exclusion of complicated patients, and comedication. To obtain effectiveness data, larger simplified trials are often needed. These may be conducted in late phase III or phase IV and must focus on outcomes, care utilization, and comparisons between alternate therapies. This evolving area may be subject to FDA regulation because advertising claims will derive from such studies, and the FDA has said it intends to review the scientific basis for such claims. 248 ----'

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FDA and Drug Approval!

Gerald A. Faich MD, MPW

Index term: Drugs

JVIR 1995; 6:24S

1 Address correspondence to G.A.F., Pharmaceutical Safety Assessments, Inc, 104Foxhall Lane, Narberth, PA 19072.2 Paid consultant for Abbott Laboratories.

DRUG regulation spans from animal studies through the postmarketing period. Developmental studies(phases I-III), approval, labeling,and cost-effectiveness are extensively addressed by the U.S. Foodand Drug Administration (FDA).Unlabeled use, promotion, and costeffectiveness claims have receivedparticular attention in recent years.

Typically, drug development requires 8-10 years including investigative (preclinical and clinical-IND)work and time for New Drug Application (NDA) preparation, submission, and approval. There has beensome shortening of FDA review timefrom 24 to 19 months in the past 2years. Nonetheless, the process ofdrug development and approval isremarkably protracted, difficult, andexpensive. Some estimates are thatit costs over $300 million to bring anew drug to market.

Drug approval is dependent onsubstantiation of efficacy and safetybased largely on two well-performed,adequate, randomized, controlledtrials conducted by qualified experts.Clinical and statistical differencesbetween the active agent and placebo, surgery, and/or another activeagent must be shown. The definitionof primary and secondary outcomemeasurements, power estimates,and monitoring for protocol adherence and data verification are crucial to the successful conduct oftrialso

Once the agent is approved, theproduct label (package insert) is developed by the drug sponsor and approved by the FDA. This describesindications, doses, efficacy, andsafety of the product. Such labeling

should be seen as a contract between the FDA and the sponsor forthe allowable use claims of the product.

While initial marketing and labeling are strictly regulated by theFDA, the agency does not regulatethe unlabeled use of marketed drugsby practitioners. However, pharmaceutical manufacturers are prohibited from promoting off-label use.This prohibition extends to the needto ensure only "hands-off' involvement in continuing medical education where objectivity, fair balance,and independence of program conduct must be maintained.

Cost-effectiveness studies and associated claims have become increasingly important to both managed care and the FDA. Usual phaseIII trials examine drug effects in thehighly atypical setting of carefullyselected patients and investigatorswith intense monitoring of dosingand outcomes. While having high internal validity, generalizing to "realworld" patients and use is often difficult. Preapproval data may be limited by duration oftrials, the exclusion of complicated patients, andcomedication. To obtain effectiveness data, larger simplified trialsare often needed. These may be conducted in late phase III or phase IVand must focus on outcomes, careutilization, and comparisons between alternate therapies. Thisevolving area may be subject to FDAregulation because advertisingclaims will derive from such studies,and the FDA has said it intends toreview the scientific basis for suchclaims.

248 ----'

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