How benefit risk data can improve
certainty and the value proposition of
new cancer treatments
Eric Low
Commissioning Chemotherapy Services Conference
Tuesday 29 November 2016
London
www.myeloma.org.uk
Overview of talk
• Current patient input mechanisms into benefit‐risk decision‐making on new treatments
• The changing context for patient input and involvement mechanisms
• The potential of patient preference methodologies in different decision‐making contexts
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Why do patient perspectives on benefit-risk matter?
What is measured in clinical trials:
What this doesn’t tell us:
• Efficacy
• Is this treatment something that patients want? • Safety • Is the treatment acceptable to patients?
• The trial endpoints e.g. effect on survival • Patient‐reported quality of life (used to translate into a quality‐adjusted life year measure)
• Observed risks e.g. prevalence and magnitude of side‐effects
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‐ to all? to the majority? to a few?
• What do patients think about the wider pros and cons e.g. oral vs IV, continuous or short‐
term treatment, impact on work and family etc.?
• How does it compare to what is already available (not just the trial comparator drug) to Usuall
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treat the condition?
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What role does ‘the patient perspective’ have
on benefits-risk decision-making today?
Regulators (FDA, EMA) and health technology assessment bodies routinely seek patient input on new treatments through consulting patient groups and/or inviting views from patients directly e.g. as ‘patient experts’ or open consultation.
Myeloma UK supports this process by:
providing written and oral evidence
helping find patient representatives to speak at committee meetings
conducting surveys and interviews about the drugs in question using research and organisational knowledge on what it is like to live with myeloma
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putting researchers in touch with myeloma patients on our research panel
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Challenges with current patient input processes
1. Can be resource‐intensive
2. Limited number of patients with ‘lived experience’ of as yet unapproved treatments
3. Representativeness and generalisability of views
4. Capturing the wide range of patient experiences in a systematic and robust way
5. Making the process user‐friendly for patients
6. Reducing benefits and risks to a common metric as the only output considered in a decision can sometimes lead to oversimplifying complex decisions
7. Do we have the right type of evidence to answer the question?
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Does patient input make a difference to decisions?
• Patient input is highly valued by regulators and HTA
• Lends legitimacy and context to decisions
• However, it does not have as much influence as other ‘less subjective’ factors
‐ Trial data
‐ Cost (HTA)
• Patient input often has a stronger influence in borderline cases or where there is uncertainty over benefit or value
 How can we make sure patient input has a more central role in every value decision not just in borderline cases?
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Moves towards a ‘science of patient input’
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Clear international direction towards more formalised incorporation of patient input
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Innovative Medicines Initiative (IMI2) ‐ Patient perspective elicitation on benefits and risks of medicinal products
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FDA, EMA, NICE and others trying new approaches
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identify, and work to minimise, legal, regulatory, financial and procedural barriers to patient involvement (EMA, 2013)
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Myeloma UK is collaborating with the EMA and NICE on a myeloma pilot studies
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Future benefit‐risk and HTA evaluations likely to require patient preference data
www.myeloma.org.uk
No consensus yet on:
• Best practice – working on developing evidence
• Responsibilities for conducting studies
• Pharmaceutical industry
• EMA/NICE
• Patient groups
• Which methods to use and when in the process?
• Quantitative vs qualitative
• Different quantitative approaches
What are patient preference research methodologies?
• 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
• Quantitative patient preference studies commonly use trade‐off techniques which measure the relative value to patients of specific components of a treatment such as:
• The way a treatment is administered e.g. oral administration vs. injection
• Outcomes e.g. survival, control of disease vs. risk of side‐effects
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• They enable large numbers of patient preferences/values to be elicited in a y
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systematic way
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Myeloma UK patient preference studies - aims
• How much do myeloma patients value different attributes of treatment?
• How much relative gain in outcomes is necessary for the potential benefits to outweigh the risks? • What is the majority view and what is the distribution?
• How do different patient characteristics affect preferences?
• Use the evidence to inform our patient input in upcoming EMA and NICE evaluations
• Evaluate how useful the data is – does it help reduce uncertainty on benefits, risks and value?
• Understand the potential for the data to influence clinical trial decisions
• Learn about how we can adapt and build on the methodologies, including
how can we best support patients to take part in this type of study
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About the myeloma patient preference studies
1. Multi‐criteria Decision Analysis (MCDA) study
2. Discrete Choice Experiment (DCE) study
• Part‐supported by Bristol Myers Squibb
• Steering group involves NICE, Myeloma Patients Europe, UCL and MHRA
• Study focus is on assessing preferences related to upcoming EMA evaluations of myeloma treatments
• Builds on a EMA successful pilot with Myeloma Patients Europe in 2015
• CaPPRe is a research group in Australia specialising in community and patient preference research
• UK‐based study
• Study focus is on exploring myeloma treatment preferences generally using trade‐off decision Usuall
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scenarios
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• Initially UK‐based, with a view to then rolling out across Europe
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• Potential for use in wide range of decision contexts Discrete Choice Experiment
(DCE) study: methodology
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Multi – Criteria Decision Analysis Study: findings
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Over 500 myeloma patients took part
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On average, the weight given to progression‐free survival was higher than the weight given to the toxicity attributes
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Participants giving a higher weight to severe toxicity were more likely to be younger, working, looking after dependent family members, and to have previously experienced severe or life‐threatening side‐effects •
This collaborative project is an example of how Myeloma UK are generating evidence through the systemic collection of patient preferences
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NICE: study to determine how patient
preferences could be captured quantitatively
and included in Health Technology Assessment (HTA)
• Will review the literature and current research activity relating to patient preferences, with a focus on the methodological literature in HTA and related disciplines as well as sectors other than health • Gather insights from patients, carers and the public about what aspects of patient experience and types of preferences should be captured • Critically assess potential methods in terms of their applicability and suitability to the HTA context and consult widely on the findings
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The goal in sight: the right treatments
for the right patient at the right time
Ensure access to drugs that help patients achieve outcomes they value
Reduce uncertainty over the benefit‐risk‐cost question
Make sure that only the most valuable drugs are developed in the first place
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“If we expectUsually
patients to comment on the benefits at the
evaluation phase we need to ensure the endpoints to
be meaningful to them in the first place”.
Potential considerations
for patient preference research
• Patient understanding and comfort with language of trade‐offs and likelihood of risk
• Hypothetical treatments but real patients with very personal goals – need to respect and treat benefit‐
risk issues sensitively • Need to prioritise and limit what can be explored in these studies to keep them manageable for patients
• Huge gap between clinical trial endpoints and broader factors that matter to patients – should we focus on what counts or what is normally counted?
• Important that preferences are described using patients' own words so that they have psychological meaning and are not imposed on the patient by the researcher (Frewer, 2001)
• We need to identify current best practice and learn from it (EMA, 2013)
• Will studies like these stand up to scrutiny?
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Preference Study Focus Group, Feb 2016. Photo supplied by EMA
Conclusions
• Newer forms of patient involvement in treatment evaluation place greater emphasis on every patient is an expert and recognising the differences as well as similarities in what matters to patients
• Patient preference research methodologies have the potential to add significant value to benefit‐risk assessment and put the patient voice closer to the centre
• There will always be a need for qualitative and other forms of patient input – to ensure both breadth and depth of perspective
• The process of eliciting patient preferences can increase transparency in regulatory decision‐making, provide formal opportunities for input from stakeholders, and delineate the sources of disagreements among participants (Institute of Medicine, 2012)
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• Further research and cross‐stakeholder work is needed to identify best practice and y
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[email protected]
0131 557 3332
Myeloma Infoline 0800 980 3332
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