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FINAL REPORT or CLINICAL STATISTICAL REPORT
The product of all clinical studies is the FINAL REPORT or CLINICALSTATISTICALREPORT (as appropriate contingent on statistical analysesbeing included in the report). All of the safety and efficacy data arepresented in the report and analyzed. In addition, pages with multipletables comprising raw data in line item format (Data Listings) areincluded as appendices to the report.
2002: Best Pharmaceuticals ForChildren Act (BPCA)
– 6 months patent extension of drug if Sponsor submits clinical data to support pediatric label
– Includes “Orphan Drugs”
– Voluntary: FDA can requestpediatric studies, but Sponsor maydecline without penalty
– Effect: Not highly successful
2003: Pediatric Research Equity Act (PREA)
– FDA can require a Sponsor to conduct clinical studies to support pediatric label (chemotherapeutics & biologicals) if marketed under NDA review (i.e., NDA or BLA) and drug is potentially used in ≥ 50,000 kids in the USA.
– Some drugs can be waived of requirement if drug not likely to be used in pediatric population, if studies not practial, or if drug is of no benefit to pediatric population.
– “Orphan Drugs” exempt
Adverse Drug Experience Reporting Requirements
– Preferred Reporting Method Used by Consumers, Physicians, Pharmacists
• MedWatch Report (Form FDA 3500A)
– The NDA Holder must report an ADE that is both serious and unexpected within 15 calendar days of receipt of information to the FDA. The MedWatch Form is typically used.
Why does FDA sometimes require Phase IV Studies?
• To obtain additional safety and/or efficacy data on patient groups not wellrepresented in Phase I – III (e.g., women of child-bearing age or certainethnic groups)
• To satisfy requirements of the Pediatric Research Equity Act (PREA)
• Sometimes after marketing during which the new drug is exposed to greater numbers of patients, something untoward may be discovered for which FDA would request additional study
Sometimes the biopharmaceutical Sponsor will elect to perform Phase IV Studies on their own to:
• Pharmcoeconomic data (i.e., cost-effectivenes data)
• Obtain data for medical/scientific publications
• Evaluate new formulations or dose regimens leading to submission of a new NDA
At the completion of the Routine or For Cause Audit, the FDA inspector will issue an evaluation of a site inspection via the Establishment Inspection Report (EIR). The FDA auditor may also issue a Form 483 which includes a list of objectionable conditions/deficiencies found during the audit.
The EIR will have one of three ratings:
• NAI (No Action Indicated)
– No significant deviations from the Regulations/GCP were found
• VAI (Voluntary Action Indicated)
– Deviations from the Regulations/GCP noted which may or may not require a response from the investigator, Sponsor, CRO, etc.
- • OAI (Official Action Indicated)– Most serious of the three ratings
- – Serious deviations from the Regulations/GCP were identified by the inspector
- – Require immediate correction by the site
- – May involve regulatory action by the FDA such as issuance of a Warning Letter, fines, and/or criminal prosecution
The most common areas of deficiency at a clinical site that result in VAI or OAI ratings are:
– Failure to identify & document protocol violations, including enrollment of ineligible subjects
– Failure to properly consent study subjects (Informed Consent violations)
– Inadequate or inaccurate source data
– Failure to recognize and/or document adverse events
– Errors relating to study medication accountability