Above and Beyond: Assessing the Nature and Impact of Additional Considerations Above Clinical and Cost Effectiveness by 8 HTA Agencies for a Rare Disease Area


Harrison K1, O'Rourke D2, Jonsson P1
1National Institute for Health and Care Excellence (NICE), Manchester, UK, 2National Institute for Health and Care Excellence, Manchester, UK

Presentation Documents

OBJECTIVES: HTA agencies primarily evaluate the quantitative therapeutic value and cost-effectiveness of new health technologies to inform reimbursement recommendations. These decisions are influenced by social, ethical and equity factors which vary across jurisdictions due to differing political, budgetary, social, and values mandates. This is particularly important for drug treatment in rare conditions with novel mechanism of action or orphan designation, as in haematological malignancies (HM). These are often characterised by high levels of uncertainty and high incremental cost-effectiveness ratios due to difficulties in producing robust evidence in small and heterogenous patient populations, as well as their high prices. This study aimed to explore variance and potential influence of additional considerations (AC) on the assessment decision across HTA agencies.

METHODS : ACs were extracted from 8 national HTA agencies publically available source language reports (n=62) for 12 HM innovative drugs. The identified 170 “other considerations” were themed and classified into categories and sub-categories in line with previous findings from literature.

RESULTS : 81% (50/62) assessments included > one AC (mean 2.74 considerations/report). The inclusion of disease considerations (unmet need:RR1.61;CI1.01-2.58,p=0.04, rarity of disease:RR1.62;CI1.11-2.37,p=0.01, current treatment issues:RR1.62;CI1.17-2.17,p=0.003, patient impact(RR1.42;CI1.02-1.97,p=0.004, national priority:RR1.42;CI1.03-2.37,p=0.03) and treatment considerations (tolerance:RR1.86;CI 1.12-3.01,p=0.01, innovativeness:RR1.53;CI1.24-1.89,p=0.0001, indirect benefit:RR1.5;CI1.23-1.83,p=0.0001, end of life:RR1.5:CI1.23-1.83,p=0.0001) were associated with an increased likelihood of a positive recommendation. Patterns of reporting and consideration varied across different HM sub-indications, by mechanism of drug action and HTA agency.

CONCLUSIONS: Differences are seen in reporting and use of additional considerations across HTA agencies for HM disease and treatment characteristics. This is likely a consequence of agency-specific value preferences. Inclusion of these considerations in HTA assessment is associated with positive reimbursement recommendations and indicates that given the challenges in producing robust evidence for these rare HM diseases, scientific and social value judgments are an important part of the decision processes for these drugs.

Conference/Value in Health Info

2021-11, ISPOR Europe 2021, Copenhagen, Denmark

Value in Health, Volume 24, Issue 12, S2 (December 2021)




Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment

Topic Subcategory

Novel & Social Elements of Value, Reimbursement & Access Policy, Value Frameworks & Dossier Format


Oncology, Rare and Orphan Diseases

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