Perform Patient Preference Studies

Overview

Capturing data on how patients perceive potential risks and benefits of new treatments can inform therapy development and the U.S. Food and Drug Administration (FDA) marketing review process. Patient preference studies, also known as patient-centered benefit-risk studies, can help identify what is most important to patients, particularly when assessing benefits and risks are challenging.

Advantages

A patient preference study is a way to collect and analyze patient and caregiver preferences concerning risks they are willing to accept for a specified benefit provided by a treatment. In fact, patient and caregiver preferences can differ greatly from what clinicians and researchers might expect, especially in the rare disease community. Although performing patient preference studies during each stage of therapy development has become more common, the FDA does not require Sponsors to include these studies. Therefore, you may wish to educate research and industry partners about the advantages of patient preference studies. The data from patient preference studies can:

  • Characterize the burden of disease, unmet medical needs, patient perspective on benefit-risk, and diversity of preferences in the patient population:
    • Identify what is most important to patients.
    • Understand the maximum risk and/or burden patients will accept for different benefits.
    • Learn how patients may compare benefits and risks between 2 therapy options.
    • Determine whether preferences differ based on demographics or geographical location.
    • Investigate other, differentiating factors that may affect patient preferences, such as severity of symptoms or stage of disease.
  • Influence the design of clinical trials
    • Develop endpoints that matter to patients.
    • Select target populations within the patient group.
    • Reveal confounding factors that may require stratification of participants during analysis, for example, participants may be stratified by age or disease stage in order to best determine effectiveness and safety of treatment.
    • Develop meaningful effect sizes for endpoints based on patient and caregiver preferences. You can learn more about Clinical Outcome Assessments (COAs) in NCATS Toolkit section on Help Industry With Clinical Trial Design.
    • Determine how to promote patient participation to make certain enough participants enroll. 
    • Uncover factors that may affect a participant’s decision to discontinue participation in a clinical trial.
  • Impact the FDA marketing review process:
    • Qualified COAs developed through the inclusion of patient preferences can be presented to FDA for consideration during the clinical trial and marketing review process. 
    • Benefit-risk of therapies targeting unmet medical needs may be assessed more tolerantly by FDA.
    • Patient perspective of benefit-risk determined through scientifically performed patient preference studies will be considered by FDA during the marketing review process.
  • Provide patients with information important to their treatment decision-making process after a therapy receives FDA marketing approval.
    • COAs may be used in labeling the therapy to allow better understanding of benefits and risks of use.

Lead or Support

Patient preference studies can be performed by your group, other patient groups, industry, academia, and even the FDA, as is the case with Patient-Focused Drug Development (PFDD) Public Meetings. Depending on the timing and reasoning behind a patient preference study, it may be most helpful to form a collaboration. Remember that the objective of a patient preference study is to gain perspectives that are representative of the entire patient population, so working with other groups and reaching out to patients that may not belong to any group can increase the impact of the study. When you are deciding whether to perform the study alone, or take the lead or play a supporting role in a collaborative effort, you may wish to consider a number of factors.

  • Cost of performing a study:
    • Study costs may range from $100,000 to $400,000 and up.
  • Staff time needed for the duration of the study:
    • Studies may take from 6 to 9 months from start to finish.
  • Requirement of external expertise to design and analyze study:
    • Large industry partners may have expertise among their staff.
    • Academic partners can offer experience, scientific rigor, and credentials, as well as a knowledge within a specific disease area.
  • Outreach experience to include patients outside group membership.
  • Historical participation of membership in similar surveys.

Timing

The timing of patient preference studies depends on the stage of therapy development you are hoping to influence. 

  • Early patient preference studies focused on understanding unmet medical needs, burden of disease, and general patient benefit-risk perspectives can provide crucial information during the therapy discovery phase to academic and industry researchers.
    • Influence symptoms to be targeted for treatment development.
    • Attract interest of academic and industry researchers.
    • Determine priority of therapies in early development from a patient perspective.
  • In the preclinical phase of therapy development, when researchers have completed initial analyses of the possible benefit-risk of a potential therapy, patient preference studies can obtain information about more specific preferences and can use this data to develop clear COAs.
    • Ask about more specific benefit-risk scenarios that may influence clinical trial design and patient subpopulations to target for enrollment.
    • Develop a COA or choose one that already exists. You can learn more about COAs in NCATS Toolkit section on Help Industry with Clinical Trial Design.
    • Determine information that may be important to patients during their decision process of whether or not to enter a specific clinical trial. 
  • During Phase 2 and 3 clinical trials, patient preference studies can be focused on actual benefits and risks seen among trial participants.
    •  The data from studies performed now can be submitted to the FDA to be included in the marketing review process.
  • After therapy approval, patient preference studies can be used to detect any difference between stated preferences (what a person believes they will choose) and revealed preferences (what a person actually chooses).

Engage with the FDA

Groups that have successfully traveled the therapy development path advise engaging with the FDA early in the process of developing and conducting patient preference studies. Establishing an ongoing conversation with the FDA can make certain you are asking your disease community the questions that are significant to the FDA throughout the clinical trial and marketing review process. 

  • Patient Affairs, available at +1-301-796-8460, works with patients and patient advocates to involve patients more effectively in regulatory decisions related to therapy safety and approval. The Patient Affairs Staff are the best resource to understand who to contact at the FDA and when. 
    • For Patients, focuses on topics that can help you learn more about the different programs and processes of the FDA, as well as upcoming public workshops and meetings. 
  • Medical Device Innovation Consortium (MDIC) Patient Centered Benefit-Risk Project Report (2015) emphasizes the opportunity to work with FDA Center for Devices and Radiological Health (CDRH) staff from early stages of medical device development. The report covers the value of patient-preference information and considerations that should be made when using this information.  The Report has elements that cross over to drug and biologic development. 
  • FDA-led PFDD Public Meetings ran from 2012 to 2017 as an effort to collect the patient perspective on the burden of their specific disease and unmet medical needs. Meetings continue to be held periodically but tend to be focused on wider public health issues, such as chronic pain and opioid use disorder. You can learn more about the FDA-led PFDD Meetings using the following resources:

Tips for Success

  • Know what decisions you want to influence and whether the questions you are asking will help you reach that goal. 
  • Explore whether there are other ways that are less demanding on time and resources to achieve your goals, such as focus groups or interviews.
  • Develop collaborations with a shared understanding of the purpose and use of information generated.
  • Understand the significance of using sound science to develop and analyze patient preference studies.
  • A clear understanding of objectives translates into choosing the appropriate methodologies to achieve those objectives.
  • Engage the FDA throughout the process.
  • Remember patient and caregiver preferences may change over time as new therapies become available.
  • Read Key Considerations in Developing & Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study (2016), a report providing information to help you decide if and when your group wants to conduct a patient preference study. The report also provides more details about the different types of studies and analyses.
  • Review Benefit Risk Bootcamp (2014), an overview of sessions from an FasterCures event that describes effective participation in benefit-risk assessment. Descriptions and slide presentations from each session are available. Topics discussed include understanding benefit-risk assessment, common methods used, and key FDA programs.

Resources

Engage with the FDA
FDA Patient Affairs U.S. Food and Drug Administration (FDA) (link)
FDA For Patients U.S. Food and Drug Administration (FDA) (link)
Tips for Success
Key Considerations in Developing & Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) (link)

Capturing data on how patients perceive potential risks and benefits of new treatments can inform therapy development and the U.S. Food and Drug Administration (FDA) marketing review process. Patient preference studies, also known as patient-centered benefit-risk studies, can help identify what is most important to patients, particularly when assessing benefits and risks are challenging.

A patient preference study is a way to collect and analyze patient and caregiver preferences concerning risks they are willing to accept for a specified benefit provided by a treatment. In fact, patient and caregiver preferences can differ greatly from what clinicians and researchers might expect, especially in the rare disease community. Although performing patient preference studies during each stage of therapy development has become more common, the FDA does not require Sponsors to include these studies. Therefore, you may wish to educate research and industry partners about the advantages of patient preference studies. The data from patient preference studies can:

  • Characterize the burden of disease, unmet medical needs, patient perspective on benefit-risk, and diversity of preferences in the patient population:
    • Identify what is most important to patients.
    • Understand the maximum risk and/or burden patients will accept for different benefits.
    • Learn how patients may compare benefits and risks between 2 therapy options.
    • Determine whether preferences differ based on demographics or geographical location.
    • Investigate other, differentiating factors that may affect patient preferences, such as severity of symptoms or stage of disease.
  • Influence the design of clinical trials
    • Develop endpoints that matter to patients.
    • Select target populations within the patient group.
    • Reveal confounding factors that may require stratification of participants during analysis, for example, participants may be stratified by age or disease stage in order to best determine effectiveness and safety of treatment.
    • Develop meaningful effect sizes for endpoints based on patient and caregiver preferences. You can learn more about Clinical Outcome Assessments (COAs) in NCATS Toolkit section on Help Industry With Clinical Trial Design.
    • Determine how to promote patient participation to make certain enough participants enroll. 
    • Uncover factors that may affect a participant’s decision to discontinue participation in a clinical trial.
  • Impact the FDA marketing review process:
    • Qualified COAs developed through the inclusion of patient preferences can be presented to FDA for consideration during the clinical trial and marketing review process. 
    • Benefit-risk of therapies targeting unmet medical needs may be assessed more tolerantly by FDA.
    • Patient perspective of benefit-risk determined through scientifically performed patient preference studies will be considered by FDA during the marketing review process.
  • Provide patients with information important to their treatment decision-making process after a therapy receives FDA marketing approval.
    • COAs may be used in labeling the therapy to allow better understanding of benefits and risks of use.

Patient preference studies can be performed by your group, other patient groups, industry, academia, and even the FDA, as is the case with Patient-Focused Drug Development (PFDD) Public Meetings. Depending on the timing and reasoning behind a patient preference study, it may be most helpful to form a collaboration. Remember that the objective of a patient preference study is to gain perspectives that are representative of the entire patient population, so working with other groups and reaching out to patients that may not belong to any group can increase the impact of the study. When you are deciding whether to perform the study alone, or take the lead or play a supporting role in a collaborative effort, you may wish to consider a number of factors.

  • Cost of performing a study:
    • Study costs may range from $100,000 to $400,000 and up.
  • Staff time needed for the duration of the study:
    • Studies may take from 6 to 9 months from start to finish.
  • Requirement of external expertise to design and analyze study:
    • Large industry partners may have expertise among their staff.
    • Academic partners can offer experience, scientific rigor, and credentials, as well as a knowledge within a specific disease area.
  • Outreach experience to include patients outside group membership.
  • Historical participation of membership in similar surveys.

The timing of patient preference studies depends on the stage of therapy development you are hoping to influence. 

  • Early patient preference studies focused on understanding unmet medical needs, burden of disease, and general patient benefit-risk perspectives can provide crucial information during the therapy discovery phase to academic and industry researchers.
    • Influence symptoms to be targeted for treatment development.
    • Attract interest of academic and industry researchers.
    • Determine priority of therapies in early development from a patient perspective.
  • In the preclinical phase of therapy development, when researchers have completed initial analyses of the possible benefit-risk of a potential therapy, patient preference studies can obtain information about more specific preferences and can use this data to develop clear COAs.
    • Ask about more specific benefit-risk scenarios that may influence clinical trial design and patient subpopulations to target for enrollment.
    • Develop a COA or choose one that already exists. You can learn more about COAs in NCATS Toolkit section on Help Industry with Clinical Trial Design.
    • Determine information that may be important to patients during their decision process of whether or not to enter a specific clinical trial. 
  • During Phase 2 and 3 clinical trials, patient preference studies can be focused on actual benefits and risks seen among trial participants.
    •  The data from studies performed now can be submitted to the FDA to be included in the marketing review process.
  • After therapy approval, patient preference studies can be used to detect any difference between stated preferences (what a person believes they will choose) and revealed preferences (what a person actually chooses).

Groups that have successfully traveled the therapy development path advise engaging with the FDA early in the process of developing and conducting patient preference studies. Establishing an ongoing conversation with the FDA can make certain you are asking your disease community the questions that are significant to the FDA throughout the clinical trial and marketing review process. 

  • Patient Affairs, available at +1-301-796-8460, works with patients and patient advocates to involve patients more effectively in regulatory decisions related to therapy safety and approval. The Patient Affairs Staff are the best resource to understand who to contact at the FDA and when. 
    • For Patients, focuses on topics that can help you learn more about the different programs and processes of the FDA, as well as upcoming public workshops and meetings. 
  • Medical Device Innovation Consortium (MDIC) Patient Centered Benefit-Risk Project Report (2015) emphasizes the opportunity to work with FDA Center for Devices and Radiological Health (CDRH) staff from early stages of medical device development. The report covers the value of patient-preference information and considerations that should be made when using this information.  The Report has elements that cross over to drug and biologic development. 
  • FDA-led PFDD Public Meetings ran from 2012 to 2017 as an effort to collect the patient perspective on the burden of their specific disease and unmet medical needs. Meetings continue to be held periodically but tend to be focused on wider public health issues, such as chronic pain and opioid use disorder. You can learn more about the FDA-led PFDD Meetings using the following resources:
  • Know what decisions you want to influence and whether the questions you are asking will help you reach that goal. 
  • Explore whether there are other ways that are less demanding on time and resources to achieve your goals, such as focus groups or interviews.
  • Develop collaborations with a shared understanding of the purpose and use of information generated.
  • Understand the significance of using sound science to develop and analyze patient preference studies.
  • A clear understanding of objectives translates into choosing the appropriate methodologies to achieve those objectives.
  • Engage the FDA throughout the process.
  • Remember patient and caregiver preferences may change over time as new therapies become available.
  • Read Key Considerations in Developing & Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study (2016), a report providing information to help you decide if and when your group wants to conduct a patient preference study. The report also provides more details about the different types of studies and analyses.
  • Review Benefit Risk Bootcamp (2014), an overview of sessions from an FasterCures event that describes effective participation in benefit-risk assessment. Descriptions and slide presentations from each session are available. Topics discussed include understanding benefit-risk assessment, common methods used, and key FDA programs.

Resources

Engage with the FDA
FDA Patient Affairs U.S. Food and Drug Administration (FDA) (link)
FDA For Patients U.S. Food and Drug Administration (FDA) (link)
Tips for Success
Key Considerations in Developing & Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) (link)