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"Unmet need" in health care and economic value

"Unmet need" in health care and economic value



"Unmet need" generally indicates that a particular disease cannot be adequately treated, or perhaps treated at all. In this presentation, Koonal Shah notes the definitions and the approaches to ...

"Unmet need" generally indicates that a particular disease cannot be adequately treated, or perhaps treated at all. In this presentation, Koonal Shah notes the definitions and the approaches to measuring "need" that have appeared in the literature. A recent exploratory empirical study also is reviewed. This research focused on the extent to which member of the general public believe that "unmet need" should be ranked high in priority in decisions that allocate health care resources.



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    "Unmet need" in health care and economic value "Unmet need" in health care and economic value Presentation Transcript

    • "Unmet Need" as a Potential Source of Economic Value in Value-based Pricing Schemes Koonal Shah PharmAccess Leaders Forum Berlin • 16-18 October 2013
    • Background • Stated objective of the UK government’s proposed "value-based pricing" scheme: “…to include a wide assessment, alongside clinical effectiveness, of the range of factors through which medicines deliver benefits for patients and society...” • The consultation document suggests that higher prices will be granted to medicines that tackle diseases that are severe or are associated with unmet need PHARMACCESS LEADERS FORUM 16/10/2013 2
    • Background • Lack of clarity about: • • • How these components should be defined What evidence is needed to generate measures that can be applied in a "value-based pricing" scheme This presentation focuses on the definition of unmet need and the evidence supporting the use of unmet need as a source of value PHARMACCESS LEADERS FORUM 16/10/2013 3
    • Defining "unmet need" “Unmet need could reflect the degree to which there are existing treatments. A condition for which there is no effective treatment, and where there is, therefore, significant unmet need, could be characterised by a high QALY loss, and deemed to exhibit a high ‘Burden of Illness’. Conversely, conditions that were already well served with effective treatments would be scored at a lower level of this measure – even if the untreated condition was itself severe and life-threatening.” VBP consultation document para 4.18 PHARMACCESS LEADERS FORUM 16/10/2013 4
    • Defining unmet need • “…‘unmet medical needs’ means a condition for which there exists no satisfactory method of diagnosis, prevention or treatment authorised in the Community or, even if such a method exists, in relation to which the medicinal product concerned will be of major therapeutic advantage to those affected.” Commission Regulation (EC) No. 507/2006 (EC Regulation on the conditional marketing authorisation for medicinal products for human use) PHARMACCESS LEADERS FORUM 16/10/2013 5
    • Defining unmet need • Some definitions of unmet need are concerned with whether the services are being received rather than whether they exist – e.g. Carr and Wolfe (1976) • Unmet need due to individual budget constraint may arise for underserved individuals with low socioeconomic status – e.g. Kataoka et al. (2002) • Some researchers note that unmet need has two dimensions: the per-patient level and the number of patients with this level of need PHARMACCESS LEADERS FORUM 16/10/2013 6
    • Unmet need in VBP schemes Kanavos, P et al. (2009) The role of . funding and policies on innovation in cancer drug development. London: London School of Economics and Political Science PHARMACCESS LEADERS FORUM 16/10/2013 7
    • Understanding society’s preferences • UK VBP proposals focus on "what society values" • Whilst there is a growing body of evidence on the extent to which society supports the use of severity in health care priority setting, few published empirical studies examine people’s preferences regarding unmet need • Tappenden et al. (2007) – preferences of NICE appraisal committee members • Green and Gerard (2009) – public preferences • Linley and Hughes (2013) – public preferences PHARMACCESS LEADERS FORUM 16/10/2013 8
    • Preference study • Objective: to develop a greater understanding of the extent of societal support for prioritising of health care resources according to disease severity and unmet need, using preference data elicited from members of the UK general public • By no means a definitive assessment of society’s preferences PHARMACCESS LEADERS FORUM 16/10/2013 9
    • Methods • Respondents from the general public • Face-to-face interviews conducted in respondents’ homes • Survey on health care priority setting • Seven choice tasks • Involved choosing between treatments for conditions which differed in terms of unmet need and/or severity and/or health gain from treatment • General attitudinal questions about NHS priorities • One budget allocation question • Repeat of earlier choice task but with more response options • Open-ended comment regarding unmet need task PHARMACCESS LEADERS FORUM 16/10/2013 10
    • Excerpt from questionnaire PHARMACCESS LEADERS FORUM 16/10/2013 11
    • Questionnaire Patient group A Patient group B Testing what? Life expectancy Life extension Other treatments? Life expectancy Life extension Other treatments? Q1 5 2 Yes 5 1 Yes HG Q2 5 1 Yes 1 1 Yes SV Q3 5 2 Yes 1 1 Yes HG x SV Q4 5 1 No 5 1 Yes UN Q5 5 1 No 5 1 Yes UN+ Q6 5 1 No 1 1 Yes UN x SV Q7 5 1 No 5 2 Yes UN x HG - - - - - - - - Q10 5 1 No 5 1 Yes UN (but difficult to take) HG = health gain; SV = severity; UN = unmet need; UN+ = ‘partial’ unmet need PHARMACCESS LEADERS FORUM 16/10/2013 12
    • Methods of analysis • Distribution of responses to each question • Comparing respondents’ responses from one question to another • Variety of face validity tests • Independent selection of open-ended comments that are of relevance to unmet need PHARMACCESS LEADERS FORUM 16/10/2013 13
    • Results – sample 60 respondents (30 in London; 30 in Kent) Characteristic 40 48 60 52 18-34 26 29 51 50 65+ Social grade Male 35-64 Age General pop. Female Gender % 21 21 A 0 3 B 8 20 C1 48 28 C2 22 21 DE 22 28 PHARMACCESS LEADERS FORUM 16/10/2013 14
    • Results – aggregate response data Option A Q1 Option B Strongly A Slightly A No pref Slightly B Strongly B Larger gain Smaller gain 58% 30% 7% 2% 3% 88% Q2 Moderately ill Severely ill 38% 7% 15% 53% Q3 Q4 Q5 Q6 Q7 Larger gain + moderately ill Smaller gain + severely ill 33% Unmet need No unmet need 20% Partial unmet need 10% Unmet need + moderately ill No unmet need + severely ill 20% Unmet need + smaller gain No unmet need + larger gain 10% Unmet need 20% 10% 23% 57% 18% 40% 20% 20% 37% 15% 23% 42% 15% 43% 22% 22% 5% 48% 33% 15% 15% 17% 40% 15% 22% 5% 25% 20% 27% 17% 37% 18% 20% 25% 10% 5% 10% 43% 23% 30% 53% PHARMACCESS LEADERS FORUM 16/10/2013 15
    • Results – aggregate response data Q8 I think that the NHS should give priority to treating patients for whom there are no other treatments available 43% I think that the NHS should give priority to treating patients who will get the largest health gain from treatment 57% PHARMACCESS LEADERS FORUM 16/10/2013 16
    • Results – Q10 0% to A; 100% to B 10% to A; 90% to B 20% to A; 80% to B 30% to A; 70% to B 40% to A; 60% to B 50% to A; 50% to B 60% to A; 40% to B 70% to A; 30% to B 80% to A; 20% to B 90% to A; 10% to B 100% to A; 0% to B 0% 10% 20% 30% 40% PHARMACCESS LEADERS FORUM 16/10/2013 17 50%
    • Results – Q4 vs. Q10 Q10 Q4 100% A Strongly A Slightly A No pref. Slightly B 1 Strongly B Total 1 90% A 1 1 80% A 2 70% A 4 3 1 60% A 1 3 3 50 : 50 2 6 7 60% B 1 2 1 4 8 4 23 2 2 2 80% B 2 12 12 12 1 2 5 1 2 1 90% B Total 9 1 1 70% B 100% B 2 1 7 14 10 60 PHARMACCESS LEADERS FORUM 16/10/2013 18
    • Results – focus on unmet need • Five of the choice tasks examine unmet need preferences • Q4 – unmet need vs. no unmet need • Q5 – unmet need vs. partial unmet need – Existing treatments are difficult to take and cause disruption to patients’ lives • Q6 – unmet need + less severity vs. no unmet need vs. more severity • Q7 – unmet need + smaller gain vs. no unmet need + larger gain • Q10 – unmet need vs. no unmet need (budget allocation framing) • Whilst distribution of responses differs from task to task, none suggests that, on average, society supports giving higher priority to treatments that address unmet need • But Q8 (attitudinal/opinion question) tells a different story • (although there remains strong evidence of a relationship between Q7 and Q8; p=0.99) PHARMACCESS LEADERS FORUM 16/10/2013 19
    • Face validity of the data • We conducted checks of consistency across questions • Interviewers also reported their assessments of respondent understand and effort • Interviewer assessments predicted fairly well how likely respondents were to give inconsistent answers (as judged by us) • Excluding respondents who we suspect were not answering "properly" makes little/no difference to results • Both respondents who understood/concentrated and those who did not tended to go for a 50:50 split in Q10 • Survey administered by trained, experienced interviewers • Questions no more complex than those used elsewhere in the empirical ethics literature PHARMACCESS LEADERS FORUM 16/10/2013 20
    • Comments left by respondents • 18 of the 60 respondents left comments that we considered to be relevant in terms of unmet need • Examples: • “Strong preference that if no treatments are available for condition A – then this should be prioritised.” • “Being a new breakthrough treatment I thought it worth giving slightly more of the budget.” • “Condition B has an alternative to choose from there is still hope for alternative B” • “There should be a higher focus on finding new treatments for patients and then on looking at treatments to replace old ones.” • Both conditions patients die after 5 yrs. A no treatment 5 yrs. B Treatment 5yrs. As far as I can see the overall scenario for both A+B is the same.” PHARMACCESS LEADERS FORUM 16/10/2013 21
    • Concluding remarks • Unmet need typically described in terms of the availability of alternative treatment options, though other definitions exist • (Sparse) literature suggests that at least some members of the society consider unmet need to be a valid health care priority setting criterion • Estimating the strength of society’s preferences is challenging PHARMACCESS LEADERS FORUM 16/10/2013 22
    • About OHE To enquire about additional information and analyses, please contact Koonal Shah at kshah@ohe.org To keep up with the latest news and research, subscribe to our blog, OHE News Follow us on Twitter @OHENews, LinkedIn and SlideShare The Office of Health Economics is a research and consulting organisation that has been providing specialised research, analysis and expertise on a range of health care and life sciences issues and topics for over 50 years. OHE’s publications may be downloaded free of charge for registered users of its website. Office of Health Economics Southside, 7th Floor 105 Victoria Street London SW1E 6QT United Kingdom +44 20 7747 8850 www.ohe.org ©2013 OHE