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The patient voice – the value of patient-reported outcomes in HTA

The patient’s voice is increasingly important in securing access for therapies in markets and demonstrating how they deliver and radiate value across healthcare systems.

Conveying the nuances of patient experience as a result of their condition and the effects of therapy – and developing methodologies for assessing and rating these by appraisal authorities – is one of the greatest current challenges in pharma as missteps and misunderstandings
can delay and even derail approvals.

Sudesh Basra and Harrison Davis, of PRMA Consulting, a market access consultancy and member of Fishawack Health, believe that insights-driven strategies to utilise patient- reported outcomes (PROs) and navigate healthcare technology appraisals (HTAs) are a critical element of reimbursement potential.

Why is the patient voice so important?
The importance of taking account of the patient experience of a therapy has been elevated, as healthcare systems around the world seek to understand how new therapies will bring an incremental benefit to patients and obtain the best value from new therapies. Decisions about the extent to which a therapy is granted market access are multifactorial and every part of the decision process needs to be considered, particularly when there is finite budget.

Patients are now more involved in their treatment decisions, in partnership with their clinician. This means that understanding which aspects of their condition have the greatest impact on their lives and how a therapy confers benefits are important to patients and clinicians alike. How well a new therapy addresses those patient needs in health technology assessments is vital to its future success and profitability. Pharmaceutical companies need to incorporate, increasingly, the influence of the patient voice in regulatory and health technology assessment (HTA) activities, and the measures that can more tangibly assess the impact of a therapy on the patient population.

Is enough being done to engage with patients?
Current PROs capture some of the evidence needed but there is further opportunity to capture patients’ perspectives about what is meaningful to them on a practical level, living day-to-day with the illness, and to discern what specific parameters associated with the condition need to be considered. Patient-relevant measures may include, for example, how much longer can they continue to work with the new therapy or, in certain conditions, how far they can walk. The challenge is how to measure benefits which are relevant to patients and relate it to the clinical efficacy of the therapy.

For instance, a recent pre-competitive collaboration in Duchenne muscular dystrophy put the patient’s voice first and examined quality of life themes that resonated with patients. It resulted in a condition-specific instrument that could be used in a cost utility analysis. Feedback from patients and Action Duchenne/Muscular Dystrophy UK was that staying independently mobile and delaying the time until wheelchair use is actually more reflective of the impact on health-related quality of life (HRQL), and of longer term prognosis than measures previously used to capture impact on HRQL or the six-minute walking distance parameter.

Manufacturers that understand which symptoms have the greatest impact on patients can advance PROs to better capture the HRQL impact of therapies, such as in the development of PROs with greater focus on itch, pain and symptom severity as measured by the patient in the treatment of psoriasis.

Quantifying that type of benefit is not easy but these were great examples of incorporating the patient voice in the instruments used to assess product efficacy for the purposes of health technology assessments.

Are HTA authorities looking for more patient content when assessing products?
Yes, certainly – it is the direction of travel, although it does vary from country to country. The National Institute for Health and Care Excellence (NICE) in the UK, for instance, has further evolved its methods for health technology assessment through a ‘severity modifier’ to provide a broader definition of severity by including quality of life as well as quantity of life in its assessment and better reflect society’s opinion that the value of one quality-adjusted life year (QALYs) provided to patients with rare diseases should be more than those with less severe diseases. More broadly, we are hearing from clinicians about the importance of the patient voice on posology aspects, for example, incorporation of ‘treatment holidays’.

The questions being asked are about parameters that benefit patients and their families.

What are the challenges in this rapidly evolving environment?
In essence, the challenge is to provide a broader definition of value for a therapy and asking how the therapeutic effect relates to patient- relevant outcomes. This is where PROs should be considered in more detail. Next steps in the evolution may be to develop methodologies to enable consideration of qualitative data from PROs with the quantitative data from clinical trials – understanding how HTA authorities are evolving their evaluation methodologies is critically important for market access.

What is the benefit for pharma from investing in deeper patient insights?
A deeper understanding of PROs allows us to assess the areas of risk or challenge to a company’s HTA submission and consider how that risk might be mitigated by supplying supplementary evidence to make the submission more robust. HTA outcomes can have an impact on the negotiated acquisition price of drugs, and maximising the contribution of PROs is important.

How does PRMA Consulting help their clients with these issues?
We have international experience in this sector. Our work looks at patient-centricity and HTA methodologies, and we bring practical experience of the likely perspectives of HTA agencies and payers towards PROs and the implications in specific markets around the globe.

PRMA Consulting offers its Policy, Access, Value and Evidence capabilities to clients from the US to Asia-Pacific, and across Europe.

Read more insights about the relevance of patient perspectives at

Sudesh Basra is Director and Harrison Davis is Senior Consultant, both at PRMA Consulting, a member of Fishawack Health

Sudesh Basra is Director and Harrison Davis is Senior Consultant, both at PRMA Consulting, a member of Fishawack Health

4th August 2022

From: Marketing, Healthcare


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