Regulatory/GMP Compliance

Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, answers some commonly asked questions about regulatory filings.

FDA recently published a final guidance for making post-approval manufacturing changes to drugs and biologics, the culmination of a long-running effort to facilitate improvements in medical product quality through the product life cycle.

The guidance provides a framework for the management of postapproval chemistry, manufacturing, and controls changes.

The guidance document gives recommendations to support biopharmaceutics classification of drug substances and the Biopharmaceutics Classification System-based waiver of the in-vivo bioequivalence study requirement for drugs.

The agency has published multiple guidance documents and policy initiatives for updating and advancing the clinical research process.

The agency’s human medicines committee has recommended extending the approved storage period of unopened thawed vials at 2–8 0C from five days to 31 days.

The COVID-19 pandemic has triggered additional considerations to supplier oversight of contract manufacturing facilities.

FDA expects to conduct only a small portion of scheduled facility visits in 2021 due to continued limitations on travel imposed by the COVID-19 pandemic.

The agency released a report that outlines inspectional activities conducted during the COVID-19 pandemic and discusses plans for future inspection operations.

Sandoz will soon begin enrolling patients with neovascular age-related macular degeneration in a Phase III study with its proposed biosimilar to aflibercept.

Combating COVID-19 is bolstering efforts to limit patent protections on innovative medicines and vaccines.

Bio/pharma can learn ways to prevent recurring events and ineffective CAPA from the nuclear power sector.

Updates to user fee programs that fund FDA operations are being finalized by industry stakeholders for approval by Congress.

A FDA report cites multiple ongoing violations that undermine assurance of quality in products from this plant and the need for a continuing “pause” in production.

Even before Emergent disclosed that it had to discard 15 million doses of the J&J vaccine drug substance, FDA inspectors had uncovered multiple deficiencies and quality control issues.

Scrutiny of the vulnerability in the biopharmaceutical supply chain has increased pressure on FDA to renew site visits of domestic and foreign facilities.

The agency’s human medicines committee has approved new manufacturing sites for COVID-19 vaccines.

Is FDA approving too many new drugs and added indications too quickly based on surrogate endpoints that fail to pan out?

BIA has released comments urging the UK government to take a science-based approach to the regulatory framework for gene editing and GMOs.

Many biologic drug manufacturers encounter similiar challenges; precompetitive collaboration can help resolve these issues. Representatives of the IQ Consortium’s Biologics CMC Leadership Group describe efforts to address the safe handling of biologic drugs in closed-system transfer devices in healthcare settings and the need for phase-appropriate specifications.

Extrapolating data from adult clinical trials to a pediatric population is a proven method to avoid unnecessary pediatric clinical trials. Representatives of the Pediatric Working Group of the IQ Consortium share case studies that demonstrate how adult clinical trial data can be used to support the dose selection and regimen—or waive the need for a placebo control arm—for pediatric studies.

Maintaining a quality culture across the diverse functions in a bio/pharmaceutical company is difficult. Representatives of the Quality Coordinating Committee of IQ Consortium report on collaborative efforts to address data integrity and the modernization of FDA’s good laboratory practice regulations.

A plant inspection backlog caused by COVID-19 restrictions threatens innovation and public health. Now pressured to step up on-site inspections, will FDA embrace new methods?

FDA’s newly proposed draft guidance will support IND filings of antisense oligonucleotides, a new class of drugs.

A growing pipeline of gene therapy candidates aimed at treating neurodegenerative diseases has prompted new guidance.