Hearing the Patient Voice: How 4 Countries are Utilizing Patient Input

By Xcenda

While health technology assessments (HTAs) aim to produce policies that balance clinical benefit alongside the increased expense, the entity ultimately most affected by these decisions is the patient. Patient and patient advocacy groups are increasingly laying pressure on regulatory and HTA agencies to have more input into the decision-making process. We examine how 4 countries are utilizing patient reported input. 


Hearing the Patient Voice Loud and Clear: A Look at 4 Countries and Their Use of Input From Patients and Patient Organizations

While health technology assessments (HTAs) aim to produce policies that balance clinical benefit alongside the increased expense, the entity ultimately most affected by these decisions is the patient. As such, patients and patient advocacy groups are increasingly laying pressure on regulatory and HTA agencies to have more input into the decision-making process. Patients, particularly when well organized, can be a powerful force that produces change by asserting their influence on lawmakers, agencies, and manufacturers. In a recent example illustrating the power patients can have over a regulatory decision in the United States, the product EXONDYS 51 (eteplirsen) was approved to treat Duchenne’s muscular dystrophy despite an initial vote against approval by a Food and Drug Administration (FDA) advisory committee. Although the FDA was originally skeptical of the clinical trial evidence which included only 12 patients, due to continued support from patient advocacy groups, the FDA ultimately approved the drug with orphan status and fast track designation.1 Patients also have a role in US value-based frameworks. The Institute for Clinical and Economic Review (ICER) has, on multiple occasions, directly leveraged patient perspectives in its final policy and is currently looking to update its framework to better clarify how patients can become involved.2,3 The United States is certainly not the only country where the patient voice can be heard within the regulatory or HTA procedures. This article will provide a brief snapshot as to how Australia, Canada, England, and Scotland involve patient or patient groups in product assessment decision making.


Australia is an example of a country that is actively pursuing mechanisms to better include patient and patient groups. The Pharmaceutical Benefits Advisory Committee (PBAC) includes a “consumer representative,” and patients can provide written input during the PBAC assessment process. But the inclusion of patient feedback is not standardized, and how the input is incorporated into decision making is not transparent.4 To combat this, in 2015, the Patient Voice Initiative was launched in an effort to improve and integrate patient involvement into its HTA methods before a decision has been effectively rendered. PBAC hopes the initiative will signal to the government, clinicians, and industry the importance of the patient voice and will ultimately strengthen the Pharmaceutical Benefits Scheme (PBS) by involving patients and patient groups in the regulatory and HTA decision-making framework.

While concrete changes to the PBAC procedures as a result of the Patient Voice Initiative are limited, several meetings with patients and patient advocates were held in September and October of 2016, at which a number of ideas were proposed to improve patient involvement in HTA processes.


While there have not been many updates to how England incorporates the patient voice within technology appraisals, it has remained a prime example for other countries in how patients might be integrated into decision making. The National Institute for Health and Care Excellence (NICE) not only includes patient input within the individual technology appraisal process but also utilizes a Citizens Council to address overarching moral and ethical issues NICE should take into account.
When considering the individual single technology appraisals (STAs), NICE seeks recommendations from consultees and commentators to nominate patient experts to be included in the assessment.5 Once selected by the Chair of the Appraisal Committee, the 2 patient experts submit a short personal statement on the technology and how it should be used in England, as well as attend the Appraisal Committee meeting.
Looking more broadly than individual HTA decisions, NICE utilizes the Citizens Council to provide a pathway for patients to have a larger role on what therapies are ultimately recommended to the National Health Service (NHS).6 The Citizens Council, a panel of 30 members of the public who reflect the demographic characteristics of the UK, is responsible for producing social value judgements and occasionally changes to NICE’s methodology. While many are familiar with NICE’s scientific value judgements which are about interpreting the “quality and significance of the [scientific] evidence available,” social value judgements relate to society instead of science.7 The Citizens Council meets once a year for 2 days to discuss topics that typically arise from the NICE guidance development procedure, after which a report is generated. To date, 18 reports covering a range of topics have been published.


In addition to involving patient groups in typical HTA submissions, Scotland also employs a unique alternative pathway for orphan, ultra-orphan, or end-of-life treatments in which patients have significant influence over the final outcome.
The Scottish Medicines Consortium (SMC), responsible for providing recommendations to the NHS of Scotland on clinical and cost-effectiveness of new medicines, leads patient advocacy efforts by sharing additional evidence from patient groups. In 2015, the SMC began formally registering patient groups for partnerships to gain information from the patient and caregiver perspective through patient group submissions. These submissions assist the SMC committee to capture relevant patient information as part of its assessment for new medicines. The SMC has partnered with over 80 groups since the launch of its patient group registration.8
Beginning in 2014, for therapies treating orphan, ultra-orphan, or end-of-life indications, pharmaceutical companies can tap into the Patient and Physician Engagement (PACE) pathway, which gives patient groups (and clinicians) a stronger voice in SMC decision making.9 To be eligible for this process, the manufacturer must provide supporting evidence that its product falls into one of the eligible categories. If the New Drugs Committee (NDC) arrives at a “not recommended” conclusion, then the manufacturer can request the drug undergo the PACE pathway, which adds an additional 1–3 months to the assessment timelines. Representatives are chosen from patient groups and asked to describe the added benefits of the medicine that are not fully captured within the typical drug assessment. These may include:


Canada, like England, has a well-established framework for including the patient voice in its HTAs. As part of the standard common drug review (CDR) procedure, the Canadian Agency for Drugs and Technologies and Health (CADTH) patient group provides input on the following10:

Patient group input, along with clinical and economic data, is reviewed by the Canadian Drug Expert Committee (CDEC), an independent CADTH advisory committee composed of appointed healthcare professionals and public members. Unlike Australia, CADTH specifically outlines how patient group information is used. Major steps include11: 

A summary (approximately 2 pages in length) is created based on all patient group information received by CADTH
The summary is sent to each of the patient groups, and they comment on whether the summary reflects the main issues and outcomes of importance to them
The patient group summary and patient group comments are incorporated into an individual section in the CADTH CDR Clinical Review Report
CDEC public members present the patient input prior to CDEC deliberations on the drug
A summary of the patient input deliberated by the CDEC is included in the recommendation document
Patient group input submissions in their entirety are shared with drug plans and posted on the CADTH website; they are also kept on file and may be referred to in future CDR reviews
CADTH and CDEC are also working with drug plans to improve their decision making by sharing patient input. Drug plans are able to access patient group submissions, CDR review reports compiled by CDATH, and CDEC recommendations to aid in decision making.

Overall, outside of clinical trials, the patient voice has been and remains a critical consideration for a new product seeking global market access. Each country varies in how patients are involved in HTAs and the relative influence that they hold. Furthermore, product characteristics (such as an orphan designation or being an oncology therapy) may impact how patients are integrated into the assessment pathway. As countries continue to reassess their HTA methods and how healthcare stakeholders (including patients and patient advocates) fit into these processes, it is important that manufacturers seek local knowledge to remain abreast of changes that may impact a product’s submission.