Appraising Ultra-Orphan Drugs: Is Cost-per-QALY Appropriate?

A new OHE report discusses NICE’s introduction of a cost-effectiveness threshold to appraise ultra-orphan drugs.

A new OHE Consulting Report has been published that discusses the use of a cost-per-QALY gained decision rule to appraise treatments for very rare conditions, as part of the NICE Highly Specialised Technologies (HSTs) programme.

The NICE HST programme started in 2013 and its interim method and process guide embraced a wide range of decision-making criteria to consider factors beyond direct health benefits captured by quality-adjusted life years (QALYs). Key changes to the HST method introduced from April 2017 are:

• The use of a standard cost-effectiveness methodology involving estimation of cost-per-QALY gained of new interventions for very rare conditions;
• The application of a £100,000 cost-per-QALY threshold over which HSTs will not generally be recommended by NICE;
• A weighting system for QALYs produced by new HST which generate more than 10 incremental QALYs over the time horizon of the disease, so that HST producing total incremental QALYs of 11-29 will be weighted between 1 and 3 (applying equal increments) and HSTs producing 30 QALYs or more will be weighted 3 times.

The OHE Report argues that:

• Given the importance of non-QALY elements in the assessment of HST, such as treatment impact on the process of care and on the patients’ or their carers’ ability to go to school or to work, and issues in measuring quality of life when the population affected are infants or young children, it is inappropriate to focus the HST appraisal only on a cost-per-QALY measure;
• Given the lack of empirical basis, the new £100,000 cost-per-QALY threshold and its further possible uplift by a factor of three seem arbitrary.

Click here to download the full report.

For more information, contact Martina Garau.

Related OHE publications:

Zamora, B., Maignen, F., O’Neill, P., Mestre-Ferrandiz, J. and Garau, M. (2017). Comparing Access to Orphan Medicinal Products (OMPs) in the United Kingdom and other European countries. OHE Consulting report. London: Office of Health Economics.