# Presentation: 371937

## 

Norman W. Baylor, Ph.D.,
 Office of Vaccines Research and Review
Center for Biologics Evaluation and Research
 U.S. FDA

***Norman W. Baylor, Ph.D.,***
*** ******Office of Vaccines Research and Review***
***Center for Biologics Evaluation and Research***
*** ******U.S. FDA***

**2****nd**** NVAC Workshop on Strengthening the Supply of Vaccines in the U.S.**
**Streamlining the FDA Regulatory  Process**

## NVAC Recommendations for Streamlining the Regulatory Process	

**Harmonizing content and format for regulatory submissions**
**Review and implementation of cGMP standards**
**Reviewing the regulatory process **
**Conclusions**

## International Harmonization 

**The FDA has made progress in information exchange using MOUs, and confidentiality agreements, e.g., Health Canada, Paul Ehrlich Institute, TGA and the EMEA**
**CBER/OCBQ’s deputy director has been assigned to a detail to Office of International Programs to expand MOUs to other vaccine regulators **
**Notwithstanding, there are still some limitations due to different statutes (laws) and regulations**

## International Harmonization

**In September 2004, FDA announced it will apply to the Pharmaceutical Inspection Cooperation Scheme (PIC/S)**
**Membership consists of inspectors/investigators from countries around the world**
**Opportunity to discuss approaches, obtain information, harmonize**
**CBER has been active in this area with respect to blood and blood products for many years.**
**Expand to vaccines**

## Partnering With Foreign Regulators: the Chiron Experience

**Since MHRA license suspension; October 5, 2004 – **
**Meetings with MHRA and Chiron**
**Ability to freely exchange information**
**Regularly scheduled teleconferences between FDA and MHRA**
**Observation of counterpart inspections**

## Review of GMP Standards

**Two-year agency initiative on pharmaceutical GMPs for the 21****st**** century concluded September 2004**
**Science and risk-based approach to review and regulation**
**Implementation phase now underway with formation of council on pharmaceutical quality**

## Review of GMP Standards: Relevant Guidance to Industry

**Scientific and technical cGMP formal dispute resolution; August 2003**
**Part 11; Computer system validation; August 2003**
**April 2004 federal register notice of intent to amend part 11**
**Completion of GMP analysis in June 2004**
**FDA intent to take incremental approach to modifications to parts 210/211 announced September 2004**
**Quality system approaches to pharmaceutical cGMP; September 2004**
**Sterile drug products produced by aseptic processing; September 2004**

## Team Biologics Program

**Development of systems-based compliance program for inspection of biological drug manufacturing facilities, including vaccines**
**Implemented December 1, 2004   **
**Streamlined approach with levels based on compliance history**
**Team biologics quality system**
**CBER and ORA actively involved in establishing system of checks and balances (QC/QA)**
**Metrics developed for many processes and in implementation phase**
**Additional training for investigators and product specialists conducted in October 2004.**

## Reviewing the Regulatory Process

**Formal Mechanisms**
**Fast Track**
**Priority Review**
**CMA: Pilots 1 and 2**
**Accelerated Approval**

## Fast Track

**Designed to facilitate the development and expedite the review of new drugs that are intended to treat *****serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.***** **
**Incorporates an end of Phase I meeting **
**Allows for more frequent communications with the FDA**
**May allow for a “rolling” review of the BLA **

## Priority Review

**For products that have the potential for providing significant preventative, diagnostic, or therapeutic advance as compared to existing treatments for serious or life-threatening conditions **
**Complete action due 6 months after the date of submission**

## Accelerated Approval

**Allows expedited marketing of new drugs or biologics intended to treat serious or life-threatening illnesses and that appear to provide meaningful therapeutic benefits compared with existing treatments.**
**Approval based on a determination that the effect of a product on a surrogate endpoint is *****reasonably likely***** to predict its clinical benefit (21 CFR 314.510 & 601.41)**
**Post-licensure confirmatory studies required  **

## Continuous Marketing Application

**FDA is conducting 2 pilots to determine whether providing early review and additional feedback and advice to sponsors during drug development for selected products can further shorten drug development and review times**
**Pilot 1: FDA has agreed to provide discipline review letters under PDUFA timelines for pre-submitted reviewable units of a BLA**
**Applies only to Fast track designated products**
**Pilot 2: frequent scientific feedback and interactions during drug development**

## Conclusions

**Despite ongoing budgetary challenges and increasing responsibilities, e.g., counter-terrorism activities, influenza vaccine shortages, and other mandates, CBER has made significant progress in addressing all of the recommendations outlined in the NVAC Report on Vaccine Supply**

## Conclusions

**CBER has met or exceeded all of its PDUFA goals**
**CBER has increased the number of formal as well as informal meetings with industry to facilitate the development of new vaccines, and continued supply of currently licensed vaccines**
**CBER has increased communications with its international regulatory counterparts through MOUs**