2 Lessons Learned

Article

Brief Summary

ICMRA Summit and Plenary 2021 Meeting Report, Dec-2021

• Pandemic preparedness

• Collaboration and convergence

• Post approval change management

• Communication and transparency

IFPMA Regulatory agilities and regulatory processes, Jun-2022

IFPMA Regulatory agilities applied to clinical trials, Jun-2022

1. Efficiency

• Digitalization

2. Ways of working, decision-making, reliance

3. Risk-based regulatory agilities

• Collaborative assessments

• Recognition and reliance

• Risk-based approaches

• Post approval changes

4. Harmonization

• Early dialogue

• Transparency

5. Practicalities

• Real world evidence

• Post approval changes

• Labelling and packaging

6. Sustainability

EFPIA and Vaccines Europe: Lessons Learned from COVID-19, May-2022

Short to Medium Term Priorities

1.    Strengthening Healthcare System Capacities:

a.     Collaboration across professions for vaccination campaigns.

b.    Maintaining infrastructures for routine immunization.

2.    Addressing Vaccine Hesitancy:

a.     Managing misinformation and educating the public through robust communication plans and digital technologies.

3.    Continued R&D:

a.     Ongoing research and testing of vaccines and treatments against new variants.

Long Term Opportunities

1.    Research Ecosystem:

a.     Importance of intellectual property (IP) rights for medical innovation.

b.    Need for long-term investment in R&D, skills, networks, and health data infrastructure.

2.    Global Supply Chains:

a.     Strengthening policies to support production and free flow of medical technologies.

b.    Removing export restrictions and ensuring open borders for vaccine and medicine supply.

3.    Regulatory Flexibilities:

a.     Continuing the use of regulatory flexibilities (e.g., rolling review) beyond emergencies.

4.    Joint Procurement:

a.     EU-level coordination for procurement during pandemics.

b.    Limiting joint procurement to emergency situations to avoid delays in access.

5.    European Health Emergency Preparedness and Response Authority (HERA):

a.     Providing funding for high-risk R&D projects and sustainable surge capacity during health emergencies.

Additional Considerations

1. Equitable Access:

Ensuring equitable access to vaccines and treatments, especially in low- and middle-income countries.

Addressing healthcare system absorption capacity and vaccine acceptance.

2. Digitalization:

Utilizing digital tools for monitoring vaccine coverage rates and improving routine immunization.

3. No-Fault Compensation Systems:

Establishing compensation systems for adverse events from medical countermeasures to build public trust.

4. Pathogen Surveillance and Sharing:

Ensuring timely sharing of pathogen samples and genetic information to enable rapid response to pandemics.

5. European Health Data Space (EHDS):

Investing in digital infrastructure and data governance to enhance preparedness for future crises.

EMA HMA (EU): COVID-19 lessons learned, 2023

1.      Crisis Preparedness:

a.      Early Activation: EMA’s emerging health threats plan and the Emergency Task Force (ETF) were crucial in the early stages.

b.      Coordination: Regular interactions with vaccine/therapeutic developers and experts, and strengthened interaction with the EU expert network on shortages.

c.       Governance Structures: New governance structures and discussion fora were established to manage the crisis effectively.

2.      Support to Development and Approval of Medicines:

a.      Agile Support Measures: Early teleconferences, expedited scientific advice, and fast-track procedures for paediatric investigation plans (PIPs).

b.      Rolling Reviews: Allowed for continuous data assessment before formal marketing authorisation applications.

c.       Conditional Marketing Authorisations (CMAs): Enabled expedited approval of urgently needed treatments and vaccines.

3.      Regulatory Framework and Procedures:

a.      Flexibility: Use of CMAs and rolling reviews to expedite authorisation.

b.      Emergency Use Authorisation: Highlighted the need for an EU-level mechanism for emergency use authorisation.

4.      Scientific Recommendations and Public Health Activities:

a.      Public Health Communications: Issued recommendations on the use of medicines outside of regulatory procedures when needed.

b.      Collaboration with ECDC: Joint efforts to provide scientific positions on vaccination policies.

5.      Clinical Trials and Real-World Evidence (RWE):

a.      Guidance on Conducting Trials: Provided urgent guidance on conducting clinical trials under pandemic restrictions.

b.      RWE Studies: Highlighted the importance of real-world data for monitoring safety and effectiveness.

6.      Safety Monitoring:

a.      Enhanced Pharmacovigilance: Strengthened safety monitoring systems, including monthly summary safety reports (SSRs) for COVID-19 vaccines.

b.      Identification of Safety Signals: Prompt identification and regulatory action on emerging safety signals.

7.      Medicines Supply and Shortages:

a.      Coordination: Established the Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG) to coordinate actions on medicine shortages.

b.      Regulatory Flexibility: Implemented measures to extend supply capacity and manage shortages effectively.

8.      Network Coordination and Resourcing:

a.      Mobilisation of Experts: Unprecedented mobilisation of experts from the European network.

b.      Business Continuity Plans: Ensured rapid response while maintaining core regulatory activities.

9.      Global Collaboration:

a.      International Cooperation: Increased collaboration and information exchange with international regulators.

b.      Vaccine Access Equity: Efforts to promote vaccine access equity for third countries.

10.  Transparency, Stakeholder Engagement, and Communication:

a.      Proactive Communication: Enhanced transparency and public engagement to build trust.

b.      Combatting Misinformation: Addressed misinformation and disinformation through factual scientific information.

Areas for Improvement

1.      Clinical Trials:

a.      Larger, Methodologically Sound Studies: Need for larger and more robust clinical studies to inform regulatory decision-making.

b.      Coordination of EU-Funded Initiatives: Improved coordination of clinical trials and prioritisation of investigational medicines.

2.      Real-World Data (RWE):

a.      Proactive Establishment of RWE Networks: Need for better infrastructure and governance for RWE studies.

b.      Timely Data Collection: Importance of timely and high-quality data from healthcare systems.

3.      Resource Capacity:

a.      Sufficient Reserve Resources: Ensuring sufficient resources are available for crisis situations.

b.      Streamlined Processes: Further streamlining of regulatory and communication processes.

4.      Regulatory Tools:

a.      Emergency Use Authorisation: Consideration of an EU-level emergency use authorisation mechanism.

b.      Rolling Reviews: Continued use of rolling reviews with selective criteria.

5.      Communication and Engagement:

a.      Crisis Communication Plan: Updating the crisis communication plan based on experiences from COVID-19.

b.      Addressing Misinformation: Continued efforts to combat misinformation and improve public understanding.

6.      Global Collaboration:

a.      Enhanced International Cooperation: Continued collaboration with international regulators and alignment of regulatory requirements.

b.      Observational Studies: Development of large observational studies in future public health

Never the same again: How COVID-19 created seismic change in global life sciences regulations 

• Part 1. And just like that, everything changed: Governments declared health emergencies.

• Part 2. Underregulating for the common good: Regulators modified processes and collaborated with the industry at unprecedented levels

• Part 3. Allied for a common cause: Regulatory agencies collaborated across borders

• Part 4. Pulling in the same direction: Life sciences companies reached new levels of collaboration

• Part 5. Mixed blessings and hard lessons: How the life sciences industry was impacted

PAHO: Regulatory System Strengthening in the Americas, 2022

Regulatory Emergency Response to the COVID-19 Pandemic in the Americas

Foundations of NRA Involvement in Emergencies

• The report, initially planned before the pandemic, was adapted to include emergency measures taken during COVID-19.

• Regulatory systems for medicines and health technologies are crucial in public health emergencies, but many countries’ systems were not well-prepared or integrated into national responses.

Legal and Organizational Frameworks

• Strengthening capacities to respond to pandemics has been a focus of multiple World Health Assembly resolutions.

• The International Health Regulations (IHR) and the Pandemic Influenza Preparedness (PIP) Framework are key initiatives for preparedness.

• The WHO Global Benchmarking Tool (GBT) helps assess and improve NRA emergency response capacities.

NRA Emergency Response in Practice

• Identification of Essential Health Products: Lists of necessary products like medicines and PPE were made available.

• Flexibility of Regulatory Requirements: Emergency Use Authorizations and other flexible mechanisms were used to expedite approvals.

• Market Control: Measures to prevent shortages and promote rational use of essential products were implemented.

Key Regulatory Functions

• Pharmacovigilance, Technovigilance, and Hemovigilance: Guidelines and active surveillance for adverse events were established.

• Clinical Trials: Procedures were prioritized to accelerate the approval of investigation protocols.

Best Practices and Efficiencies

• Flexibility in Regulations and Processes: Up-to-date policies and procedures, such as emergency use authorization and extension of certifications, are crucial.

• Virtual Strategies: Use of virtual documentation and work formats.

• Faster Timelines: Expedited review processes, especially for clinical trials.

• Prioritized Resources: Focus on 24/7 operations and prioritization of emergency-related products.

• Learning and Information Sharing: Continuous learning from other agencies and information exchange.

• Communications: Enhanced communication with stakeholders, including the public, industry, academia, and government representatives.

• Reduction of Duplication of Efforts: Increased use of reliance to improve regulatory efficiency, such as in GMP inspections.

Recommendations

• NRAs should use WHO GBT indicators to develop strong regulatory emergency response regulations, policies, and procedures.

• Adoption of best practices and efficiencies for regulatory emergency response is recommended.

Regulatory agilities impacting review timelines for Pfizer/BioNTech’s BNT162b2 mRNA COVID-19 vaccine: a retrospective study, Nov-2023

•  Introduction

• Objectives

• Materials and Methods

  • Categorization of agilities

  • Data Collection

  • Study Design and Data Analysis

• Results

  • Part I - regulatory review time

  • Part II - impact of regulatory agilities on review time

• Discussion

  • Agilities that could be applied outside of a pandemic setting

  • Harmonization and convergence are key to reliance

  • Future state - where are we heading

  • Limitations

• Conclusion

WHO: Lessons learned in expediting prequalification and registration of Ebola Zaire vaccine, Aug-2020

• Global collaboration

• Communication

• Intellectual property

• Emergency use

• Advanced preparation

• After initial authorization

• Equitable access

• Pharmacovigilance