Health Policy blog covering advocacy developments and health system initiatives around the world in the areas of:
Health Policy blog covering advocacy developments and health system initiatives around the world in the areas of:
The objective of patient involvement in drug (or medical device) development evolves over the lifecycle:
Early research: Understand unmet needs of patient community and assess ability of the medicine or device to meet those needs
Clinical development: Define evidence generation plans to capture impacts that are meaningful to patients
Submission and registration: Assemble submission dossiers that include patient-relevant evidence collected during clinical development
Launch & Post-Launch: Ensure that the patient value of a treatment or device is understood by access and reimbursement stakeholders; continue to build evidence, including real-world evidence, to highlight impacts on patients
Patient representatives include those diagnosed with a medical condition, family, caregivers, advocates and patient organizations. All can provide valuable insights through the lifecycle.
Regulatory review focuses on whether the medicine or device is safe and effective. Patient input is essential in judging whether the drug’s efficacy is meaningful to patients. The expectation for involving patients in the development of a drug to ensure it reflects their needs is only increasing.
The US Federal Drug Administration (FDA) published guidance for patient-focused drug development (PFDD).
Guidance 1 (2020, final): Overview of sampling methods and considerations for defining a target population to collect patient input
Guidance 2 (2022, final): Best practices for identifying what is important to patients
Guidance 3 (2022, draft): Selecting clinical outcome assessments to measure outcomes of interest to patients
Guidance 4 (in drafting): Collecting clinical outcome assessment data and incorporating into endpoints
Only patients, caregivers, or patient representatives can provide a non-diluted perspective of the lived experience with a health condition, a healthcare technology or using a healthcare system.
For example, they can provide insights into:
What it is like to live with the condition; impact on quality of life and caregivers
Community unmet needs, including what patients expect from a new treatment for the condition
Practical implications of treatment (uptake, adherence); acceptability by patient population
Financial implications for patients
Interpretation of evidence
Limitations of existing data
Inequalities and special needs of subpopulations
Adapted from:
INAHTA position paper | Date of Issue: June 2021 | https://www.inahta.org/download/inahta-position-statement-on-patient-involvement/
Patient Engagement in Early Dialogues: Tools and resources for HTA bodies https://imi-paradigm.eu/petoolbox/pe-in-ed-hta/
Patient involvement can be defined as the inclusion of evidence/research indicating the patient perspective and through the active engagement of individuals.
Here is an overview of the top methods of patient involvement (adapted from FDA guidance).
Lit review: Collects patient input on burden of disease and treatment preferences; e.g., scientific publications, medical records, patient journal, etc.; outputs can be used with regulators and HTAs
AV materials: Includes recordings of speech or actions to demonstrate the patient experience (generally not used for regulatory and HTA decision making)
Patient experience registries and disease registries that capture patient experience are useful in understanding the burden of disease and the functional impact of disease and treatments on daily life.
Can be useful in identifying top concerns or chatter among the patient community to supplement literature reviews and help formulate questions for further exploration during interviews or advisory groups. If robust methodologies are chosen, can be used as evidence to regulators and HTA bodies.
Quantitative method for capturing patient input; can be used to explore a specific topic further or gain general awareness about patients’ needs and expectations. If robust methodologies are chosen, can be used as evidence for regulators and HTA bodies.
Qualitative method for gathering patient input; ideal for deep diving into a specific topic, when topics are sensitive in nature or hard to communicate through writing.
Both types of meetings include a small group of patient representatives in moderated discussion. Group interactions may help additional issues surface that would not have otherwise during individual interviews. In focus groups, the company is traditionally not in the room. Advisory boards are meant to provide expert opinion on a topic; these can be done as a one-off activity or as a recurring meeting.
Chapter 12 in this manual provides an overview of these codes and regulations.
Independence and full editorial control
All interactions must be ethical and patient organizations must remain independent. An industry partner cannot seek to influence the content of an organization they sponsor to advance the company's own commercial interests. Patient organizations' opinions and actions should align with their missions and be formulated independently.
Written agreements and clear expectation
The level and nature of involvement must be clear to both parties from the outset. Industry partners should have a written agreement, which includes purpose of activity, when providing financial support, indirect support, or non-financial support to a patient group.
Transparency
Industry partners are encouraged or mandated to make publicly available a list of groups receiving financial and non-financial support from the company. This is particularly relevant for groups operating in Europe and Latin America.
Single-company funding
No company may require that it be the sole funder of a group or any of its programs. While there may be cases where only one company wishes to support a patient organization, no company can make its support conditional on being the sole funder. This is particularly relevant for companies operating in the rare disease space.
Events and hospitality
Companies can provide support for patient organization meetings if the primary purpose of the meeting is professional, educational, and scientific, or otherwise supports the mission of the organization. Companies holding meetings for patient organizations must ensure the venue and location are appropriate and conducive to the purpose of the event. Hospitality must be modest by local standards and limited to the scope of the event.
Use of logos and proprietary materials
Use of organizations' logos or proprietary material requires prior written permission from groups.
