Innovation issues for regulators society and industry overview of conference topics
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Innovation; issues for regulators, society and industry. Overview of Conference topics. Alasdair Breckenridge Medicines and Healthcare products Regulatory Agency London 23 September 2008.

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Innovation; issues for regulators, society and industry. Overview of Conference topics

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Innovation; issues for regulators, society and industry.Overview of Conference topics

Alasdair Breckenridge

Medicines and Healthcare products Regulatory Agency

London 23 September 2008

“It is a frustrating aspect of benefit-risk evaluation that there is no defined and tested algorithm or summary metric that combines benefit and risk data that might permit straightforward quantitative comparisons of different treatment options, which in turn might aid decision making”

CIOMS, 1998.

Current perceptions

  • Approval thresholds heightening

  • Increased focus on safety

  • Regulators dislike uncertainty

  • Increasing cost of drug development

  • Demand for increased transparency

  • Negative perceptions of pharmaceutical industry and regulators

  • What information do regulators and industry need to enable them to undertake benefit : risk assessments?

  • Are available frameworks adequate?

  • How can we best communicate benefit : risk to the general public?

Benefits of an agreed framework

  • Shared understanding of issues between industry and regulator.

  • Consistency in expressing benefit and risk.

  • Transparency of regulatory decisions

  • Tool to compare products

Methods of assessing benefit

  • Nature of disease-incidence

  • natural history

  • impact of treatment

  • consequences of no intervention

    • Purpose of treatment-prevention

      prevention of recurrence

  • symptomatic treatment

  • cure

    • Assessment of benefit-morbidity



      quality of life

  • Methods of assessing risk

    • Development or post marketing

    • ADRs affecting different systems

    • Seriousness of ADRs


    Reporting bias

    Quality of reports

    Cannot estimate frequency of events

    • Spontaneous reports of ADRs



    Timing of market authorisation




    Earlier to market?



    Post-licensing assessment


    “A systematic approach to population based drug safety surveillance which seeks to ascertain the number of adverse events by means of a continuous pre-organised process.”

    • Active Surveillance

    Record of drug exposure

    Record of outcomes

    Access to detailed information about individual cases

    • Data requirements for

    • active surveillance

    Computerised databases of users and non users

    Medical records

    • Sources of information for

    • active surveillance

    British Society of Rheumatology Biological Register(1)

    • For patients with severe RA prescribed anti TNF αmedicines.

    • To receive drug, patient must be entered on central register.

    • Short and long term ADRs documented.

    • Cohort of similar patients prescribed conventional DMARDs

    British Society for Rheumatology Biological Register(2)

    • Rheumatologists contacted 6 monthly about specific patients

    • Data on severe infections and hospital admissions obtained

    • Patient diaries

    • Patients flagged with ONS to ascertain new cases of cancer and deaths

    • Over 20000 patients on register

    GPRD: population figures

    • Contains 6.1 million acceptable patients from 464 active practices

    • Currently collecting data on 3.58 million active patients from 405 up to standard practices

    • >45% of patients have 5 or more years of follow-up, >60% have at least 3 years

    • Contains over 40 million patient-years data

    • Geographically representative

    The Benefit-Risk Spectrum


    The worst balance

    Forrestricted use only

    Balance may require further study


    Acceptable balance






    Principle of three

    • Seriousness, duration, incidence

      as applied to adverse reactions

    • Seriousness, chronicity, extent of control or cure

      as applied to benefit

      Describe verbally or numerically


    3 = high, 2 = medium, 1 = low, 0 = no effect

    Shortcomings of models

    • Do not enhance objectivity of benefit-risk decision making

    • Time consuming

    • Conflict between stakeholders in assessment

    Benefit-risk : outstanding issues

    • Need for lexicon of terminology

    • Framework for benefit: risk assessment in which different models might be used

    • Different models for different situations

    • Agreed framework would improve underlying science of innovation

    • Importance of taking patient preferences into account

    • Learning from other disciplines, including HTA

    • Communication difficult but crucial

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