Incorporating the patient perspective on the value of medicines into health technology assessments (HTA) 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 oncology, has a formalised 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 can 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.
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. Patient preference research extends beyond health outcomes (endpoints) measured in clinical trials to include all aspects of treatment that are important to the patient.
Patient preference research methods are interested in measuring the values (needs / views) of patients with a 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).
The Patient Voice Initiative began in 2015 when 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. Following these meetings, a conference was organised aimed at increasing patient engagement in HTA in Australia. Following the conference, a steering committee was formed to action items generated from the workshops.
The steering committee is currently made up of a patient advocate, CaPPRe, three pharmaceutical companies and a patient advocacy organisation. The meetings have been sponsored by nine different pharmaceutical companies.
There were three meetings in 2016. One in Sydney; the objective of the day was to discuss ways of improving patient involvement in HTA processes that are used in Australia by bodies such as the PBAC (Pharmaceutical Benefits Advisory Committee), MSAC (Medical Services Advisory Committee) and PLAC (Prostheses List Advisory Committee) for the reimbursement of new drugs, devices, procedures and prostheses. This included discussion on specific elements of the existing system e.g. patient experience in PBAC hearings. There was a follow up meeting in Melbourne to make it easier for patients and patient groups to be involved and another in Canberra.
The first meeting was divided into 2 parts:
Firstly, a download of information, including:
• Top line review of HTA in Australia and PBS processes and the PBAC
• Presentation on consumer engagement in PBAC processes
• Direct experiences from a patient with multiple myeloma and a patient with Cystic Fibrosis
Secondly, a workshop facilitated by two patients on patient views of the current system and ideas on how best to improve patient involvement in the HTA process for listing of new drugs on the PBS. The ideas generated were then compared to ideas generated at the earlier meeting in Sydney, which took place in February 2016.
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.
An educated consumer voice will help signal to Government, clinicians and industry the importance of consumer preferences Bringing the two worlds together will help strengthen the PBS for Australia.
Benefits to different stakeholders include:
• 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
• 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
• 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? Preference information can be used to guide the conversation between the physician and the patient.
Problems addressed by Initiative
Often, PBAC submissions are already done deals by the time that consumers are aware of them. The Department of Health also does not systematically seek out consumer views – currently it is just a website and a form. Up until now, the committee has been dominated by clinicians.
There is a need for a more formalised framework for eliciting meaningful patient input and a more transparent process for how that input is incorporated into the decision-making process.
Involving patients in the lifecycle of medicine development
Director of Research and Innovation