New Regulatory Paradigms For Biologicals & Vaccines - an industry perspective

Transcription

1 New Regulatory Paradigms For Biologicals & Vaccines - an industry perspective ICDRA Pre-Meeting April 2, 2006 Elaine C. Esber, MD Chair, IFPMA Biologicals and Vaccines Committee

2 Outline IFPMA - representing a global R&D industry Commitments Technological advances Responding to emerging threats Impact of a changing environment Challenges to Industry Regulatory requirements WHO and other harmonization efforts NRA Strengthening Recommendations

3 International Federation of Pharmaceutical Manufacturers and Associations - IFPMA IFPMA members... are committed to the research and development of new high quality medical interventions that save lives, reduce overall health care costs and improve the quality of life of people around the world believe that a viable, R&D pharmaceutical industry operating in an open market, with adequate and effective protection of intellectual property and with regulatory policies designed to ensure the rapid introduction of new chemical and biological products, is essential for patients well-being and for the economic development of all countries around the world

4 A Global Industry... GAVI WHO UNICEF World Bank Gates Foundation UNAIDS Chiron Vaccines Merck KGaA LEEM FARMA INDUSTRIA Abbott Almirall PIF VFA Member Associations Pfizer Merck Boehringer Ingelheim EFPIA/ EVM INTER PHARMA IFPMA Novartis GSK Bayer ABPI PReMA PhRMA Eisai Sanofi Aventis Schering- Plough > 40 other associations Member Companies Roche astellas RxD INTER FARMA WTO Wyeth FIFARMA Others Rx&D Berna Crucell 13 others JPMA

5 ... working together with governments and other partners in the interest of patients and public health Pictures of children with infectious diseases have been deleted

6 Technology is advancing giving rise to unprecedented numbers of exciting new biologics and vaccines

7 Vaccines - Innovations at accelerating pace 21 rst century: Age of proteins and polysaccharides 1990 s: Numerous Mab s & recombinant proteins 1991: Conjugated H. influenzae type b vaccine 1986: Recombinant protein vaccine (HBV) 1955: Cell culture technology (Enders) 1945: Triple therapy DTP combination 1889: Bacterial filtrate vaccines e.g. diphtheria (von Behring) 1885: Organ source rabies immunotherapy (Pasteur) 1796: Jenner administers Smallpox vaccine

8 New Vaccines against Infectious Diseases - Ongoing research and development Diarrheal Cholera ETEC Rotavirus Shigellosis Typhoid Fever Respiratory Influenza Parainfluenza3 RSV SARS S. pneumoniae Tuberculosis Bacterial Anthrax Meningitis Plague Gp A strep Gp B Strep STD transmitted Chlamydia HSV-2 HIV/AIDS Vector Borne Dengue JE West Nile Parasitic Hookworm Leishmaniasis Malaria Schistosomiasis Hepatitis and Cancers H. pylori Hepatitis B,C,E HPV EBV Enteroviruses Polio Adopted from: IVR, WHO, April 2005

9 Biotechnology-Derived Medicines - Research and development ongoing Three hundred and twenty-four (324) new medicines and vaccines to treat nearly 150 diseases These include medicines to treat: cancer infectious diseases autoimmune disorders neurological disorders, and HIV/AIDS and related-conditions. PhRMA Survey October 25, 2004

10 Emerging Threats for the 21 st Century - e.g., Influenza Pandemic Flu: Are we ready? Current vaccine technologies Antiviral therapeutics Prototype vaccines, e.g., H 5 N 1 What more needs to be done? Preparedness planning Capacity building Interpandemic recommendations for use Cell-based vaccines New antiviral therapeutics New science, e.g., common antigens

11 Outline IFPMA - representing a global R&D industry Commitments Technological advances Responding to emerging threats Impact of a changing environment Challenges to Industry Regulatory requirements WHO and other harmonization efforts NRA Strengthening Recommendations

12 The Environment is changing... We are living in a global society Diseases know no boundaries The pharmaceutical industry is working globally Costs of development are rising; requirements are increasing Information is moving at an unprecedented pace

13 ...and yet, the challenges to global development of biologicals are increasing! Differing national/local requirements and procedures Complexity of meeting each set of requirements/ timelines Duplication of efforts and wastage of animal and human resources Delays restrict subject participation in global trials Variable interpretations of guidelines Differences in procedures, experiences and staffing of NRAs Variable enforcement of GCPs and GMPs Inconsistent adherence to IPR and Data Exclusivity Role of WHO in strengthening NRA s, harmonization and prequalification

14 IFPMA Key Messages Global access to vaccines & biologicals is facilitated by appropriate legal and regulatory frameworks Industry/governments should work together to ensure appropriate level of regulatory control Quality/efficacy/safety of vaccines & biologicals to be ensured in most efficient way

15 IFPMA Key Messages Global access to vaccines & biologicals is facilitated by appropriate legal and regulatory frameworks Industry/governments should work together to ensure appropriate level of regulatory control Critical factors: Quality/efficacy/safety of vaccines & biologicals to be ensured Predictable in most and efficient transparent wayregulatory review procedures and standards Appropriate approval timeframes/processes for clinical trials, marketing authorisations & post licensing variations Pragmatic use of Authority resources & harmonization of requirements

16 Transition in Regulatory Authority Capabilities and Communication 10 years ago TODAY FUTURE Totally independent authorities little discussions or interactions between authorities Authorities focused locally Significant numbers of countries with no or little working regulatory system Several harmonization initiatives Sharing of information between some regulatory authorities (formal & informal) Speedy transfer of information globally. Development of technical & clinical expertise More authorities will strive for higher levels of excellence / leadership (globally or regionally) More authorities will come closer to ICHstyle regulatory review and system

17 Characteristics of of ICH-type an - like Authority authority CTD as basis of dossier Full transparency of guidelines Predictability of timelines Published metrics and measures Full independent review (no reliance on CPP) Resource and systems to manage post-approval activities Variations technical and clinical Label management and updates Administrative changes Resources and systems to manage the market Distribution, storage, supply: legal and illegal Adverse event reporting Sales and marketing practices Understands development and supply dynamics and engages with Industry to find solutions

18 Use of Authority Resources WHO observes 4 levels of Authorities depending on the development status of the local pharmaceutical industry: 2 models of licensing systems based upon submission of A WHO-type certificate of pharmaceutical product Evidence of registration in reference countries 2 models of licensing system based upon assessment of new drug applications including biological products Review of the dossier with some reliance on approval elsewhere Full independent evaluation *WHO publication How to Develop and Implement a National Drug Policy WHO, 2001

19 Impact of Regulatory Processes on Timely Approval, Access and Supply Reducing review timelines expedites patient access to medicines and vaccines Focus resources on most relevant areas of regulatory control Introducing more flexibility into review procedures allows reviewers to focus resources on priority areas of healthcare, including vaccines. Free up resources to focus on other areas e.g. pharmacovigilance and monitoring counterfeit medicines and vaccines Regularly check and reference the standards of best regulatory practice worldwide (e.g. ICH)

20 Use of Resources: Inspections Inspections cost time and money for Company and Inspectors All inspections are taken seriously and involve significant planning and support efforts, before, during and after Companies make products to the same GMP standards on same site Could Inspectorates communicate with each other and develop recognition systems, especially from a Regional viewpoint? Could resources be conserved by regulatory focus on local manufacturer (either foreign or local companies) and companies with previous unacceptable or unknown inspection history?

21 Use of Resources: Approval times for Clinical Trial Applications Regulatory Submission First Patient Trial recruits sufficient patients Set up Recruitment Period Regulatory Submission US / EU Recruitment cycle Set up Recruitment Period Long Regulatory cycle time As Countries with more efficient regulatory review timelines improve their recruitment times, the opportunity for countries with longer start up time to participate decreases

22 Use of Resources: Lot Release Many countries have functioning National Control Laboratories (NCL), e.g., OMCL, Official Medicines Control Laboratory) Check at time of registration Routine check at import Often need heavy support: training, Q&A, samples, equipment, reagents. Delays import/release, or registration Could more dialogue (including formal networking/communication) and recognition of work of the NCLs in country of manufacture add to efficiency?

23 Predictable regulatory review & approval: Best practice in use of CPP CPP accepted for Approval Requirement for CPP at submission contributes to delay in access due to long approval time Advantage of providing CPP at approval rather than at submission provides access to biologicals and vaccines in a similar timeframe to ICH regions Could the CPP be accepted at time of approval or replaced by other evidence of competent Authority review, e.g. approval letters? Article 58 provides EMEA (with WHO input) scientific opinion on a product not intended for commercialization in EU

24 Harmonization of Regulatory Requirements ICH harmonization activities are sound in principle providing a basis for regulatory review Appropriate global harmonization and recognition will ensure patients and healthcare practitioners have timely access to medicines and vaccines Harmonization should be pragmatic and aligned with international norms where possible rather than adding complexity Challenge to Regulators is to learn from outcomes of harmonization initiatives by sharing of experience, e.g. ICH Global Cooperation Group Work on harmonization within Areas e.g. ASEAN, SADC

25 Regulatory Harmonization Initiatives ICH ICH adopters

26 Regulatory Harmonization Initiatives WHO ICH ICH adopters PANDRH APEC ASEAN GCC-DR SADC WHO

27 Recommendations 1. Increased dialogue between industry and regulators 2. Increased transparency; predictability of processes and timelines 3. Increased recognition and adoption of internationally accepted standards to reduce duplication and wastage of animal and human resources 4. Consistent interpretation of internationally accepted standards and guidelines between NRAs and between NRAs and industry 5. Recognition of IP and data exclusivity to promote innovation

28 Summary Many countries are working independently and cooperatively to strengthen their regulatory systems Need different models based on country capabilities IFPMA, WHO and countries, themselves, must recognize the constraints Flexibility, dialogue, predictability and transparency are critical There is a need to lower costs of development, where possible, and without affecting safety Industry wants/needs a consistency in regulation - evenly applied to all companies Industry stands ready to work towards improving the efficiency of the regulatory system to increase access to patients of needed medicines and vaccines