1. Approaches to Patient Input for BR-PV and Risk Minimization Stephen Heaton

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3. Regulatory Authorities
Have begun to appraise the value of patient perspectives and preferences for benefit-risk management
US FDA is working on several programs to ensure patient perspectives have an effect on determining which products are developed and approved
FDA has launched the Patient Preference Initiative
EMA also is working to implement more structured medicine benefit-risk methods in evaluations that take patients’ values and their willingness to accept risks into account

4. CIOMS IX
CIOMS Working Group IX stated, “In the context of risk minimisation planning, input from patients regarding the relevance, acceptability, and feasibility of a proposed initiative is critical in order to optimize the design of the risk minimization plan, broaden support and gain cooperation for implementation.”
Incorporating patient perspective and preference information into benefit-risk acceptability, assuming risk can be minimized to offset its negative impact on the benefit-risk balance

5. PV Patient Engagement Roadmap
Goals:
PV as a key stakeholder in patient engagement initiatives from early development through launch and lifecycle
Achieve broad internal recognition that PV has a unique opportunity to generate patient insights worldwide through the ability to interact directly with patients through the AE reporting and follow-up process, under safe harbor
Achieve broad internal recognition that PV provides unique expertise in patient focused communications on risk as well as benefit risk balance

6. PV Patient Engagement Roadmap
Why PV as a key stakeholder?
Primary focus on patient – safety and benefit-risk
Unique opportunity for two-way communication under safe harbor
Patient AE self-reporting and follow-up activities
Unique opportunities for non-brand/product focused collaborations
Academia, Regulatory Agency consultants, other (eg PSCAP)
Patient advocacy groups
External environment expectations
Regulatory agencies (eg RM communication tools, PIL, REMS)
Lay public
Corporate Brand expectations and reputation

7. PV Patient Engagement Roadmap
Opportunities for Increase Patient Insights
Development & BU
Pharmacovigilance
Discovery and Development
Advocacy groups
Study subject insights
Focus Groups
Preference studies
Market insights
Regulatory Agency Insights
Development
Risk management tools
Risk minimization tools
F/U Questionnaires
Patient Preference Studies (BR)
Patient
Insights
Medical Affairs & BU
Launch / Lifecycle Management
Advocacy groups
Patient Insights
Focus Groups
Preference Studies
Medical Information Queries
Social Media
PSP-PSDMP
Regulatory Agency Patient Insights
Post-Marketing Surveillance
AE self-report
F-U Questionnaires
PSP-PSDMP
Social media monitoring
AE Reporting
Other

8. PV Patient Engagement Roadmap
Opportunities for Enhance Patient Communication
Development & BU
Pharmacovigilance
Development
Risk management tools )
Risk minimization tools
Other
Discovery and Development
Recruitment tools
Informed consent document
Retention tools
Other
Enhance Patient Communication
Medical Affairs & BU
Launch / Lifecycle Management
Education and Counseling materials
Advocacy groups
Medical Information Queries
PSP-PSDMP
External communications programs
Post-Marketing Surveillance
Additional RMin materials
PIL
Other

9. What patient input can we consider?
Patient perspectives1 : wide range of information including anecdotal comments in correspondence to e.g., FDA or testimony at Advisory Committee Panel meetings, patient opinions expressed publicly including social media, patient responses to qualitative ad hoc surveys, quantitative measurements of PROs, and more
This can be applied when patient views, experiences, or feelings on some topics are examined.
Patient preference1 information is defined as qualitative or quantitative assessments of relative desirability or acceptability of attributes that differ among alternative diagnostic or therapeutic strategies.
This can be applied:
- when multiple options exist without an option clearly superior
- when evidence supporting one over others is more uncertain;
- when pts’ views about the most important benefits / acceptable risks of a product vary considerably within a population or differ considerably from those of HCPs.
Patient-reported outcome (PRO)2 measure that reports status of patient’s health condition that comes directly from the patient, without interpretation
of the patient’s response by a clinician or anyone else. 
PRO instruments fall under a broader caption of perspective /engagement;do not really fall under preference methodology.
1. FDA draft guidance on patient preference information (May 2015)
2. FDA Guidance on patient-reported outcome measures: use in medical product development to support labeling claims (Dec 2009)

10. Patient Preference
Patient preference and perspective methodologies can be applied during benefit risk assessment and risk minimization tool design
Helps ensure that patients use them in least burdensome and most effective manner
RMin tools need to be designed with more specific patient input from given target population who will use the medication
Patient input during tool selection may help determine feasibility as well as potential effectiveness
Potentially can save substantial time and cost, especially if performed during development
Providing results of patient input to regulatory authorities also may increase benefit-risk acceptability

11. Patients and additional risk minimization tools
Patients are medication end users
Patients and caregivers who live with and endure a disease on a daily basis for which they receive medical treatments may have developed their own insights regarding perspectives and preferences on their medical treatments’ benefits and risks
As such, patients can contribute in managing risks

12. Termination of Market Supply
How can PPP risk minimization information be applied in the product lifecycle? …… A
PoC
Phase B C D E …
Start
Phase I
PoC
Start
Phase II b
Start
Phase III
Release for
Submission
Launch
Variation/Change
Termination of Market Supply

13. Process

14. Conclusion
Patient insights for BR-PV requires a cross functional partnership
Focus is on BRM, RM - including risk minimization
Applied methodology overall that appears to fit the requirements for these activities is patient preference
Regulators are not yet aligned and at speed as to the right approach

15. Thank you