Case from a Pharma Company (“Company”)
Company modified and developed new tools to include direct patient perspectives and drive patient centered decisions:
- Patient Insights Playbook and Integrated Patient Journey Template
Patients from US, UK, Australia, Taiwan and Korea provided feedback on the PE Playbook. This occurred via in depth interviews. Some modifications have been made and further modifications will be made, especially to terminology, and also to ensure that interactions are mutually positive.
- Updated Target Product Profile (TPP): includes direct patient insights
To validate insights from patients, various qualitative and quantitative approaches have been utilized, depending on the project. For example, during a patient advisory board meeting, patients consistently expressed and described the impact of fatigue on their daily lives. Fatigue is well documented in the literature, however hearing directly from patients influenced the R&D team to take action and study fatigue.
Although the Target Product Profile (TPP) is proprietary, it includes primary and secondary endpoints, patient reported outcomes, patient benefits, safety, tolerability, and dosing/presentation. Insights should be provided directly by patients based on descriptions of disease and treatment burden and unmet needs. Please see Table of Contents of the PE Playbook and Dashboard examples. For representativeness, it is important to specify at the beginning what is the target population.
- Customer Value patient insight into formulation/supply chain
- Patient Voice Plans in R&D: patient insight into compound and clinical strategies and protocol design
- Internal Compliance Guidebook: best practices on how to implement patient engagement
Practical examples: modifying a device design based on patient insights, resulting in more correct dosing of medication, re-ordering tests in a clinical trial, reducing length of visits in a clinical trial, decision to measure fatigue and sleep patterns based on patient insights, working with patients to develop a new symptom diary, and involving patient groups in development of web site content. These examples are from multiple disease areas within the Company.
The assessment of the quality of PE practice
Driving change via Patient Engagement Dashboard
Objective: drive behaviour to incorporate direct patient voice early and understanding barriers.
Activation: Company is measuring the amount of organizational change.
Enablers: Company is seeking to understand roadblocks to organizational change.
Sustainability: Company is measuring the impact of these Patient Engagement activities.
1- Shared purpose
Company’s Patient Engagement Mission Statement: “We will systematically interact with patients, obtaining direct and inferred insights, so we can effectively act on their perspectives early”.
In one example, the Patient Reported Outcomes team worked with patients to understand their perspectives, ultimately resulting in the development of a new tool to measure what matters to patients, as reported directly by patients. Direct patient interviews were used to develop the questionnaire, and patients participated in cognitive interviews to ensure that the questionnaire’s instructions, questions, recall periods and response criteria were clear and easily understood. Validity and reliability were assessed in an observational study.
Patient Reported Outcomes team worked with patient groups to create and validate the mission statement.
2- Respect and accessibility
Existing tools and processes have been updated to include Patients as an additional stakeholder with equal respect in Company’s decision making (Target Product Profile, Customer Value/Formulation Decisions and Integrated Patient Journey). Patient Reported Outcomes team also created a Patient Voice Plans for R&D at a compound level, to strategically assess and determine what were the best ways to engage with patients in Company’s clinical plans. Patient Reported Outcomes team knows that there is not a one size fits all methodology for patient engagement so they recognize that the unique needs of all stakeholders should be taken into account upfront.
In working with patients on the PE Playbook, Patient Reported Outcomes team worked with facilitators who are based in the local countries and understand the local cultures, and a discussion guide was used.
3- Representativeness of stakeholders
The Patient Voice Plans, at a compound level, will strategically assess and determine which sample groups should be involved in the project. The Internal Compliance Guidebook and the Patient Insights Playbook also provide guidance and recommendations on selecting representative groups.
One example in France: a patient group provided input into a trial protocol, changes were made, and the group was notified about which changes were implemented and some which were not. Later, Company decided not to further develop the medicine and the same patient group was notified.
4- Roles and responsibilities
In collaboration with patient groups, a video was produced to help patients manage a new diagnosis with IBD. This was a truly collaborative effort, with roles and responsibilities outlined at the beginning, which were modified as the project was underway based on real time learnings. In the UK, the operating company has partnered with a patient organization for a three year period to be measured by the patient group against a set of criteria evaluating the level of patient centricity. A baseline has been identified, and plans have been agreed upon.
Patients helped develop the Patient Engagement Mission, Patient Engagement Playbook, and have provided insights into Target Product Profiles as well as other tools. Patients provided feedback and insights through various interactions including advisory boards, interviews with patients and patient groups, focus groups, surveys, market research and other means. Patients also helped one R&D team develop a new survey to measure symptoms for a disease. The process began with members of an online patient community providing feedback on a draft which was then further modified, and further tested.
Elements of study designs modified include reordering tasks/tests and reducing length of visits. A new Director, Patient Insights has joined the team and is currently conducting studies in over 13 countries with more than 1,300 patients across all 11 disease areas. The purpose is to gather patient feedback on protocols in order to make modifications before protocols are finalized.
A recent example of modifying study design was reordering tasks based on patient feedback. The team originally planned to start with more difficult tasks and end with simpler tasks as a "reward". However, based on direct patient insights, a decision was made to start with simpler tasks and build up to more difficult, so that patients could build confidence. This change will apply to multiple studies for the same disease area.
In both cases (IBD project and patient centric culture partnership), contracts were mutually agreed upon and entered into up front – before the start of the projects - and modified as appropriate along the way. One lesson learned is collaborating on a project slowed down timelines, but it was the right thing to do.
5- Capacity and capability for engagement
The Patient Voice Plans, at a compound level, will strategically assess and determine what support is needed for meaningful engagement in the project(s). The Patient Insights Playbook also provide guidance and recommendations meaningful collaborations.
Training and tool kits have been developed for internal personnel, and external moderators are selected based on experience working with patients. For a patient advisory board recently put in place for a 1.5 – 2 year period, patients went through an application process. The application was written in a manner appropriate for lay persons, and mutual expectations are being set for engagement throughout the project.
6- Transparency in communication and documentation
Within the Company, tools and processes have been standardized to ensure a harmonized approach to patient engagement. Anonymized project specific insights are stored and shared so that they can be leveraged in the future. The dashboard is attached here.
A new internal system was developed and put in place to store market research and other appropriate documents so that patient insights can be referred to and acted on regularly. Patients and patient groups have provided consent and privacy rules and agreements have been followed. Before a project begins, materials are produced which are clear and written in lay language.
7- Continuity and sustainability
Company has policies on patient engagement, inclusive of transparency (Global, Regional and National regulations).
Company’s policies are set according to global/local laws and regulations and are independently monitored and audited for business adherence to the set policies.
Outcomes and results include, but are not limited to:
1) Product formulation/design - modification of a drug/device combination product in development based on patient insights, resulting in more correct dosing.
2) Modification of multiple clinical trials based on direct patient insights, including reversing the order of tests/tasks (protocol modification), providing transportation to the site, and reducing length of visits. In one clinical trial, there were no dropouts for 7 months.
3) Developing medicines that better meet needs: decision to modify a target product profile to pursue a biomarker or faster acting medicine, based on feedback from a patient advisory board.
4) Measuring what matters to patients: decision to measure fatigue and sleep patterns, working with patients to develop a new symptom diary.
5) Co-developing disease educational materials. These examples are from multiple disease areas within the Company.
Positive impact for specific medicines development phases: Better recruitment and retention in clinical trials Better correct use leads to better outcomes A more aligned product to patient’s needs and wants.
Direct or indirect positive impact for patients: A better experience in Clinical Trials Better correct use of a product Products that better align to their needs and wants.
Learning is that patient engagement requires time and commitment. It is important to have the support of senior leaders, to have tools and expectations and to embed patient engagement into the various departments, such as R&D, supply chain, commercial etc. It is also helpful to hold employees accountable by measuring progress over time. The Dashboard has been in place for 1.5 years. Significant progress has been made and there is still more to do. To operationalize patient engagement, it is important to get support from all functions of Company to embed patient engagement as a way of working. It was found helpful to start with “service models” whereby a dedicated resource or department in Company initially conducts the engagements – this can accelerate progress. It was also learned that budget constraints and time can be limiting factors for patient engagement, but as long as everyone is asking what does the patient want and need and as long as patients are involved directly in development of solutions, ultimately needs should be better met and research should be accelerated.