Patient Voice Initiative


Incorporating the patient perspective on the value of medicines into health technology assessments is becoming increasingly more important as acknowledged by several prominent HTA agencies. NICE in the UK has employed several measures, including a Citizen’s Council. Canada, particularly in the area of oncology, has a formalized process through which patient input on drug reviews and feedback on recommendations is obtained to ensure patients’ experiences (both good and bad) of living with cancer and undergoing treatment are routinely considered. In Australia, there is a consumer representative on the PBAC and patients have the opportunity to provide written input during the assessment process, although the process of how PBAC consider and incorporate this information is not transparent. There is a need for a more formalized framework for eliciting meaningful patient input and a more transparent process for how that input is incorporated into the decision making process.

What is traditionally done: Patient Reported Outcomes

In the current process health outcomes are traditionally measured using patient reported outcomes (e.g.,quality of life measures) and incorporated into a benefit assessment to determine the value of a health intervention.

For example, Quality Adjusted Life Year (QALY) which is a measure of the state of health of a person or group in which the benefits, in terms of length of life, are adjusted to reflect the quality of life.

Moving beyond the clinical trials

Patient preference research extends beyond health outcomes (endpoints) measured in clinical trials to include all aspects of treatment that are important to the patient.

Looking at patient preferences: How is this approach different?

Patient preference research methods are interested in measuring the values (needs / views) of patients with a particular condition. The goal is to explore how patients perceive treatments (both current and new treatments) and understand what is most important to these patients when evaluating treatments. Patient preference studies commonly use trade-off technique (such as discrete choice experiments / conjoint analysis) which directly measure the relative value of specific components of a treatment (e.g. Oral administration vs. Injection). Output is displayed visually using an interactive dashboard tool (example dashboard for CLL patients is displayed below):

How does the method work?

DCEs  are a trade-off technique. Participants are given hypothetical situations (tasks) and asked to choose which options they would prefer (e.g., treatment A or treatment B……).  By varying the components shown over repeated tasks and observing the choices made, the researcher is able to determine what is most important to participants in choosing between the alternatives.

Key outputs

Treatment Index: Allows the user to input the characteristic of any treatment and determine a score for that treatment as to how well it aligns with patient values.

Treatment comparison: Allows the user to input the characteristic of any treatment and compare to another treatment (e.g., new treatment entering the market compared to an existing treatment) and see what proportion of patients would prefer each treatment.

Relative attribute importance: A graph which show the relative attribute importance of each treatment attribute.

Patient preferences and the treatment life cycle

Patient engagement / input is not only limited to the regulatory or reimbursement stage. This approach can be applied across the drug development life-cycle, including eliciting patient preferences from the earliest stages of drug development and clinical trials that will drive treatment enhancement and alignment with patient values. The diagram below shows how patient derived preferences can impact drug development throughout the treatment life cycle (source FDA).

How does this information benefit stakeholders?


  • Gives patients a meaningful and transparent voice!
  • Patient feedback (not all patients are the same): Provides patients with feedback about their own preferences and how they compare to other patients with their condition (could be displayed as a dashboard or summary report)
  • Preferences are dynamic: show how preferences develop / change over time with experience, knowledge and at different stages of the illness


  • Provides a formal platform to allow patient input to assist in regulatory and reimbursement decisions

Pharmaceutical companies

  • Treatment alignment - measures how well treatments align with patient values
  • R & D - discover what new aspects of treatment patients would value the most

Physician / Patient alignment

  • Do physicians and patients have the same preferences?
  • Potential to use as a consultation decision tool - e.g., quick survey in waiting room which displays instant preference feedback which can be used to guide the conversation between the physician and the patient.

Where to from here .....Patient forum background

In 2015 a group of stakeholders from industry, academia and patient groups came together to discuss methodologies and approaches for eliciting the patient perspective on the value of medicines. As a result of these meetings a conference was organised aimed at increasing patient engagement in health technology assessment (HTA) in Australia. 

Following the conference, a committee was formed to action items generated from the workshops.

If you would like to be involved moving forward please click on the link to register interest: Register

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