1. An Agency Perspective on Plain Language Summaries of Publications
Measuring the Impact of Patient Engagement Activities in Medicines R&D – A Way to Sustain Cultural Change?
– DIA 2019, Panel Presentation –
Nicholas Brooke
Executive Director PFMD & PARADIGM Partner
An Agency Perspective on Plain Language Summaries of Publications

3. Complex system Complex landscape Guidances Regulatory FDA Fair market3 8 7
Complex landscape 2 6 5
* for clinical trials
publications,
various reports etc. 4 1
Guidances
PFMD
Quality
Guidance
CIOMS
Capacity building /training
Envision
EFPIA | EPF | EFGCP
| Industry
Plain language summaries*
Clinical trials info access
Measurement
PFDD
NHC (US version)
Legal agreements
PARADIGM
on CAB
(on preferred agreements)
WECAN survey | guiding principles | reference contracts
Patient views
PFMD Industry training
FDA
Fair
market
value
WECAN
survey
extended survey
EFPIA principles
NHC calculator
Regulatory

5. Value of Patient Engagement in Clinical Trials
The analysis of ‘the patient-centric trials’ by the Economist indicated:
drugs developed using patient-centric designs had 20% increased chance to be launched compared to drugs developed without. This remained consistent across the different therapeutic areas covered (neurology, oncology and rare diseases).
patient-centric trials took less time to recruit 100 participants (4 months), compared to the other innovation types (6-11 months) and all trials (7 months).
20%
Chance to be launched compared to drugs developed without.
4 months
Compared to the other innovation types (6-11 months) and all trials (7 months)
Source: the Economist Intelligence Unit patient-centric trials based on PubMed, Data:

6. Research on the financial value of Patient Engagement
Net present value (NPV) = metric integrating key business drivers of cost, time, revenue, and risk
Study: assessing the impact of patient engagement on NPV for a typical oncology development program entering phase 2 or phase 3
Results:
Pre-phase 2 project
NPV increase of $62MM
Pre-phase 3 project
NPV increase of $65MM
An investment of $100,000 in patient engagement can generate NPV increase that exceeds 500-fold the investment.
Source: Therapeutic Innovation & Regulatory Science 2018, Vol. 52(2)

7. Stakeholders’ Expectations Matrix Conclusions
The table below shows stakeholders’ expectations of one another in patient focused medicines development.
Group being considered …….
Patients
HCPs
Policy/ regulators
Payers
Industry
Researchers
Research funders
Patient organisations promote opportunities for engagement.
Be informed and actively involved.
Advise and represent patients clinical and non-clinical needs.
Understand patient needs to inform policies.
Incorporate patient views in developing criteria for funding decisions and be transparent about cost and value.
Advocacy, collaborate with funders and advise researchers.
Clinical management
Patient education.
Take on more responsibility to drive patient engagement.
Ensure value for money in funding decisions and represent the patient.
Be involved and be active in connecting the wider patient community.
Promote engagement in drug development to patients.
Ensure engagement happens effectively.
Provide clear payment decisions with patient / public input to criteria.
Ensure end to end patient engagement .
Provide feedback about what works and their experiences of drugs.
Provide expertise and represent the patient. Provide advice and support to patients.
Create a framework and facilitate engagement. Ensure alignment of shared priorities.
Include patient voice in decisions.
Actively understand patient needs and outcomes. Provide information and resources.
Inform of unmet needs – continuous dialogues.
Provide individual and global experience.
Participate in clinical trials.
Represent and empower patients.
Assess and provide objective information.
Provider a balanced view of evidence.
Connect stakeholders
Set frameworks to involve patients.
Pay for access to drugs based on population need and effectiveness.
Build patient voice in end to end development.
Seek active patient input in all stages of development so drug meets holistic needs
Funding unmet needs
Need to become more involved.
Support other patients – emotionally and practically.
Patient support and advise on medicines.
Earlier engagement in priorities and research.
Facilitate development.
Ensure policies are in place.
Provide guidelines of best practice.
Engage in areas and priorities for research.
Understand outcomes to determine requirements.
Set framework for end to end patient engagement.
Understand patient priorities.
Ensure value of patient input.
Provide strategy around patient priorities in research.
Set criteria for patient engagement in research
Build stronger and broader relationships that enable collaboration and challenge.
Be the stakeholder between the patients and research and across stakeholder groups.
Listen to how patients can/want to be involved to guide the development process.
Build relationships to share priorities to reflect the needs o f patients.
Understand better ways to involve patients in the end to end drug development process.
Design research that is easy for a range of patients to participate in.
Ensure patient perspective has been taking into account when making funding decisions.
Involve patients at all stages of drug development. Ensure research addresses patient needs and takes a holistic view of requirements.
Engage with and incorporate patient views in all stages of drug development so the drugs address the issues important to the patients and public (unmet need).
From the perspective of…….
Ensure research subject and methodologies takes into account patient needs and preferences.
Ensure patients voice is incorporated into determining research priorities and patients are involved in the research.

8. The current perception of PE is low, yet ideal expectations are high
Latest survey
The current perception of PE is low, yet ideal expectations are high
Current impression:
Highest mean at CT: 33.82
Lowest mean at pricing and reimbursement decision process: 20.49
Ideal expectation:
Highest mean at Early Dialogues: 72.19
Lowest mean at Licensing of medicines: 62.74
Largest gap between current and ideal: ED at There is quite a lot of expectation in early dialogues to improve PE processes.

9. Previous challenges with stakeholders in PE activities
Of 13 listed challenges, the top 5 challenges are listed in the figure. The most reported challenge was ‘patient input was not part of decision making’ for the total responses. However, when broken down, the patient community reported their top challenges as ‘Communications were not clear and open’ and a ‘Lack of shared vision/goals with other stakeholders’.

10. Measuring the impact of PE
Respondent Group
Do NOT have metrics or methods to measure impact of PE (n/total)
Industry
103/130
Regulators, policymakers, HTA, research funders, payers and healthcare professionals
11/25
A majority of respondents in both groups stated they do not have metrics or methods to measure the impact of PE. Where respondents who said they do, we asked for more information to conduct further research about what type of tools might be used.

11. More effective PE
Top three outcomes in ED: When the voice of the patient is reflected in the decision’, ‘When patients’ needs are better met’, ‘When it results in improving transparency and openness in decisions’

12. Delphi processes for co-prioritisation
Development and validation of a framework for prioritisation questionnaires
Meeting in person to reach final consensus of the key criteria and their definition
Setting research priorities
Designing clinical trials
Early dialogues with regulatory and HTA
Expert panels go through prioritisation exercise first online
(2 rounds)
Survey & focus group results
Literature review
Criteria to assess how patient engagement expectations are met
Background data

13. Results: Criteria to assess patient engagement practices
Setting Research Priorities
Designing Clinical Trials
Early Dialogues with Regulators and HTA
CRITERIA
WEIGHT
Key Elements of Practice Design
19.5
Aim and objectives 14
17.9
Resources 16
Patient engagement impact
Target participants involved in patient engagement
15.3
Evaluation of the PE Practice in Setting Research Priority 12
Target patients involved
Involvement and participation
14.7
Capacity Building
Legal and ethical consideration 11
Code of conduct
11.3
Patient Engagement Impact
11.5
Capacity building
11.1
Involvement and Participation 10
10.9
Code of Conduct
Sustainability 8
Evaluation of the PE practice in the Design of Clinical Trials
Evaluation
8.8
TOTAL
100