Novel oral iron therapy for iron deficiency anaemia: How to value safety in a new drug?

Giovanna Culeddu,Dyfrig A Hughes,Dora I A Pereira,Jonathan J Powell,Li Su,Rupert A Payne,Yafeng Cheng

doi:10.1111/bcp.15078

Abstract

Novel oral iron supplements may be associated with a reduced incidence of adverse drug reactions compared to standard treatments of iron deficiency anaemia. The aim was to establish their value-based price under conditions of uncertainty surrounding their tolerability. A discrete-time Markov model was developed to assess the value-based price of oral iron preparations based on their incremental cost per quality-adjusted life year (QALY) gained from the perspective of the NHS in the UK. Primary and secondary care resource use and health state occupancy probabilities were estimated from routine electronic health records; and unit costs and health state utilities were derived from published sources. Patients were pre-menopausal women with iron deficiency anaemia who were prescribed oral iron supplementation between 2000 and 2014. The model reflecting current use of iron salts yielded a mean total cost to the NHS of £779, and 0.84 QALYs over 12 months. If a new iron preparation were to reduce the risk of adverse drug reactions by 30-40%, then its value-based price, based on a threshold of £20 000 per QALY, would be in the region of £10-£13 per month, or about 7-9 times the average price of basic iron salts. There are no adequate, direct comparisons of new oral iron supplements to ferrous iron salts, and therefore other approaches are needed to assess their value. Our modelling shows that they are potentially cost-effective at prices that are an order of magnitude higher than existing iron salts.

Highlights

| INTRODUCTIONThe true cost of prescribing “inexpensive” iron salts may be significantly higher than the cost of the product itself, owing to associated increases in the utilisation of health care services
Iron deficiency is the most common cause of anaemia worldwide[1,2] and, for uncomplicated cases, it is treated with oral iron supplementation
A decision analytic model was developed, with probabilities of transitioning among six health states relevant to the management of iron deficiency anaemia based on data obtained from the Clinical Practice Research Datalink (CPRD); utilities based on EuroQol (EQ)-5D-3L tariff scores; and direct medical costs of primary and secondary care services based on CPRD and linked Hospital Episode Statistics (HES) data

Summary

| INTRODUCTION

The true cost of prescribing “inexpensive” iron salts may be significantly higher than the cost of the product itself, owing to associated increases in the utilisation of health care services This has prompted the development of novel oral iron forms that seek to reduce ADRs or a switch away from oral iron to intravenous strategies.[5,6] Both alternative approaches are markedly more expensive than oral iron salts but the key issue concerns the pricing at which they would become cost-effective. A decision analytic model was developed, with probabilities of transitioning among six health states relevant to the management of iron deficiency anaemia based on data obtained from the Clinical Practice Research Datalink (CPRD); utilities based on EuroQol (EQ)-5D-3L tariff scores; and direct medical costs of primary and secondary care services based on CPRD and linked Hospital Episode Statistics (HES) data. The protocol for this study (reference number 14_201) was approved scientifically and ethically by the CPRD Independent Scientific Advisory Committee

| METHODS

| RESULTS

Findings

| DISCUSSION