Additional Risk Minimisation and Burden: A CIOMS IX Perspective. Stephen Heaton, MD BayerHealthCare Member of CIOMS IX Working Group

Transcription

1 Additional Risk Minimisation and Burden: A CIOMS IX Perspective Stephen Heaton, MD BayerHealthCare Member of CIOMS IX Working Group

2 Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug Information Association, Inc. ( DIA ), its directors, officers, employees, volunteers, members, chapters, councils, Special Interest Area Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. Drug Information Association, DIA and DIA logo are registered trademarks or trademarks of Drug Information Association Inc. All other trademarks are the property of their respective owners. 2

3 Special Thanks Special thanks to Philippe Close, Gerald Dal Pan, Panos Tsintis and other members of the CIOMS IX Working Group The text and figure used in this presentation are modified from CIOMS IX Working Group (in press 2014) 3

4 Agenda Overview of CIOMS IX, Practical Approaches to Risk Minimisation for Medicinal Products (in press 2014) Burden of Additional Risk Minimisation Risk Minimisation Tool Selection and Burden General Considerations Stakeholders Burden Factors Measurement of Effectiveness and Burden 4

5 Key Words Burden Stakeholders Risk Minimisation Risk Minimisation Plan Risk Minimisation Programme 5

6 CIOMS IX Working Group: Practical Approaches to Risk Minimisation for Medicinal Products, CIOMS IX Working Group Report provides the most current thinking for global, but practical approaches to additional risk minimisation including: INTERNATIONAL REGULATORY CONTEXT AND BACKGROUND PRINCIPLES OF IDENTIFICATION AND APPLICATION OF ADDITIONAL RISK MINIMISATION TOOLS GOVERNANCE AND IMPLEMENTATION EVALUATING EFFECTIVENESS OF RISK MINIMISATION STAKEHOLDERS CURRENT TRENDS AND FUTURE DIRECTIONS The Working Group included expertise from regulators, academia, and industry 6

7 What is Burden in Context of Risk Minimisation? Increased load from additional risk minimisation intervention(s) on stakeholders: Patients Carers Healthcare systems: healthcare professionals, regulatory authorities pharmaceutical companies, and others Burden can impact processes: Patient access to prescribed medicines and/or needed healthcare services, daily activities Healthcare providers by adding steps or services not normally required in day-to-day management Health care systems by requiring extra human and/or financial resources Evaluation of risk minimisation programmes Risk minimisation planning should follow stepwise approach, and start with least burdensome measures needed to accomplish goals 7

8 Steps in Risk Minimisation Tool Selection I. Risk identification: Basic starting point for risk minimisation strategy decisions II. Goal setting: Risk minimisation goals, specific objectives, targets: expressed in terms of SMART criteria (i.e., specific, measurable, achievable, relevant, timebound) III. Healthcare system integration: Risk minimisation tools should be designed to fit within existing applicable regulatory, legal framework, patient care environment, and healthcare system IV. Burden: Consideration of impact on users and healthcare system in proportion to expected reduction of frequency and/or severity of targeted risks V. Evidence-based: Risk minimisation tool should be evidence based re effectiveness in achieving goals and desired objectives if possible VI. Proportionality: Risk minimisation should reflect level of risk and consideration of public health and benefit risk balance (BRB) acceptability impact 8

9 Risk Minimisation Induced Burden Consider if risk minimisation tool may place impractical, unrealistic, or undue burden Risks often absorbed and managed through usual healthcare delivery in given system ( routine risk minimisation) Additional risk minimisation considered when usual healthcare system cannot manage a particular important risk appropriately New burden may be induced by imposition of additional risk minimisation: healthcare system may not adapt to, and deliver, which could ultimately defeat goal of additional risk minimisation Can be viewed conceptually as Risk Minimisation - Burden Balance 9

10 Burden Considerations and Integration of Risk Minimisation in Healthcare Systems 10

11 Examples Risk minimisation imposed burden may include disruption of normal, or usual, workflow of a healthcare system already overtaxed in certain regions Risk minimisation tool such as frequent blood test not readily available at patient s usual healthcare facility: requires ill patient to travel long distances = burden on patient If frequent specified blood test required in this scenario, then why not arrange home visits to bedridden patient from mobile healthcare personnel as alternative? Not an alternative if healthcare system is not equipped to deliver this service, e.g., due to time, disruption of routine care, staffing costs, etc. = burden on HCS Result: risk minimisation programme may not be effective 11

12 General Considerations of Burden (1) Risk minimisation should balance optimisation of benefits and risks: prevention/mitigation of risks, and preserve access to needed medicines While strong measures to prevent or mitigate risks might be successful in achieving those goals, they may be so burdensome that they inappropriately limit access to a medicine In some cases, persons for whom benefits outweigh risks would not receive a medicine because of access limits, result that would have negative public health impact 12

13 General Considerations of Burden (2) To achieve proper balance, risk minimisation planning should consider burden of a plan Methodology to identify and quantify risk minimisation burden not fully elaborated-requires further development Despite methodology limitation, important that sources of burden be identified both during planning and assessment of risk minimisation 13

14 General Considerations of Burden (3) Burden of risk minimisation plans should be justified by benefits of a medicine (compared to next best alternative treatments), and effectiveness of plan Risk minimisation plans can vary from minimally burdensome to extremely burdensome Burden may be on practitioners, on patients, on other parts of healthcare system, or on several parts of system 14

15 General Considerations of Burden (4) Burdens on any part of system, regardless of magnitude, should only be accepted when considered necessary to ensure that goals of Risk Minimisation Plan are met Burdens that do not contribute to attaining goals are not justified Burden of Risk Minimisation Plan and its evaluation should be proportionate to level of risk Burden may vary according to country and healthcare system 15

16 Stakeholders and Burden (1) Risk minimisation burden may not only impact effectiveness of Risk Minimisation programme but may also prevent or reduce access to needed medicine Patient and HCP perspectives of a potential risk minimisation burden impact should be considered in early phase of risk minimisation planning Selecting risk minimisation tool with higher burden requirements should be reserved for medicinal products that have greatest potential for favourable benefit-risk balance, as well as achievable objectives Consultation of target stakeholder focus groups could be considered as part of validation of proposed approach 16

17 Stakeholders and Burden (2) Principle of proportionality between measures proposed and frequency and severity of risk being minimised has to be considered in development of Risk Minimisation Plan In many circumstances ability of regulatory authority (RA) to require additional risk minimisation activities by sponsor for new medicinal product being evaluated for registration requires identification of most efficient risk minimisation tool one that minimises risk without imposing such burden on sponsor and other stakeholders that sponsor may want to withdraw product from market Nevertheless RA needs to consider whether benefits of product outweigh associated risks and whether these can be managed in their jurisdiction 17

18 Potential burden factors (1) Individual vs. population burden of tool application on relevant stakeholders (physicians, pharmacists, patients, industry, healthcare centres, regulators, and others) Burden tolerance depends on particular medicinal product s benefit, other available therapeutic alternatives, and level of risk involved Larger burden may be acceptable for potentially lifesaving medication for unmet medical need 18

19 Potential burden factors (2) Even in non-life-threatening indications, e.g., acne, BRB may only be positive if potentially burdensome risk minimisation tool(s) are to manage severe teratogenic risk of a medicine Prior stakeholder experience with specific tools and combinations of tools will impact their fidelity to given risk minimisation programme If Risk Minimisation Plan results in overly burdensome risk minimisation programmes, prescribers may avoid medicine in question, or avoid risk minimisation programme, and prescribe potentially therapeutically less effective alternatives 19

20 Measuring Effectiveness of Risk Minimisation and Burden (1) Effectiveness of risk minimisation programmes essential and should be planned prospectively Evaluation of effectiveness also imposes burdens on healthcare system Evaluation may include measurement of programme implementation and patient outcomes BUT also measurement of programme s negative consequences, such as undue burden and unnecessary limitations on access Burden factors on patients and healthcare system need to be considered for any risk minimisation programme as they can potentially impact effectiveness as well as access to medicines. 20

21 Measuring Effectiveness of Risk Minimisation and Burden (2) Measuring effectiveness of public health initiatives is an evolving field Novel, robust, more appropriate evaluation designs and methodologies to evaluate effectiveness of risk minimisation programmes, including impact, if any, of burden on patients and healthcare system needed These methods should aim at further maximising safe and effective medication use while minimising burden on healthcare systems Exchange of factual experience and constructive interactions between patients, healthcare professionals, marketing authorisation holders and regulators 21

22 Thank you! Questions? 22