Regulatory Authority and Responsibility
- The Bureau of Food and Drugs (BFAD) ensures safety, efficacy, purity, and potency of gene therapy products.
- Regulation covers all gene therapy products and related studies.
- The BFAD mandates compliance with established safety standards before use in patients.
Scope of Regulation and Compliance Requirements
- Gene therapy products must meet BFAD standards of safety, efficacy, purity, and potency.
- Required submissions for Investigational New Drug (IND) status include:
- License to Operate for local manufacturers.
- Certificate of Current Good Manufacturing Practice (local or country of origin).
- Summary of manufacturing processes and quality assurance protocols.
- Detailed product information (name, active ingredients, chemical composition, dosage form, administration route, indications).
- Laboratory safety analyses.
- Animal study protocols and results.
Clinical Trials and Approval Procedures
- Companies must secure permits for clinical trials phases 1 through 3 before conducting human studies.
- Submission of detailed study protocols explaining methodology, patient selection, drug administration, risk-benefit analysis, efficacy determination, and patient protection measures is mandatory.
- Protocols must adhere to the Declaration of Helsinki principles, Good Clinical Practice (GCP 1997), and relevant Philippine laws.
- Informed consent, including disclosure of risks and benefits, must be obtained from all participants.
Review and Oversight
- BFAD approval for IND is contingent upon careful evaluation by a panel of expert advisors in microbiology, molecular biology, pharmacology, toxicology, and immunology.
- The panel assesses the appropriateness, safety, and efficacy of gene therapy for the intended disease.
- Study protocols require approval by a Hospital Institutional Ethics Review Board comprising scientific, legal, medical, religious, and consumer representatives.
- Patient welfare and protection are primary concerns.
Ethical and Quality Assurance Obligations
- Manufacturers and medical researchers must prioritize patient and consumer safety.
- Obligations include use of high-quality experimental products, compliance with good clinical practice, and transparent communication with BFAD and patients.
- The regulatory framework aims to safeguard health and welfare in line with BFAD’s mandate.
Adoption and Formalization
- This regulatory policy was adopted on July 6, 2005, under the authority of Prof. Leticia Barbara B. Gutierrez, MS, Director of BFAD.