Often, drug manufacturers are faced with limited study data when developing therapies for rare diseases. To identify challenges in creating economic models, researchers at RTI Health Solutions coauthored a study that reviewed health technology assessment submissions for rare disease treatments.
Although the definition varies by country, over 7,000 known diseases are classified as rare. Economic modeling in this area is a new and developing field. Limited data from short-term, single-arm trials, coupled with limited natural history and data can make generating robust estimates of cost-effectiveness difficult.
The paper also considers other aspects of value that may be important to patients and decision-makers, including the potential to improve patient independence and increase their ability to work.
Health technology assessment (HTA) of rare diseases has been an area of much debate and has evolved substantially in recent years. The National Institute for Health and Care Excellence (NICE) may now accept rare drug products for reimbursement up to a cost-effectiveness threshold of £300,000 per QALY. The Institute for Clinical and Economic Review (ICER) recently produced a new value assessment framework to be used in assessing products for ultra-rare diseases.
This review will help to inform high-quality economic modeling of drugs for rare diseases. It will allow researchers to understand better the difficulties imposed by limited data when developing models for the treatment and provides advice on methods of mitigating such challenges.
Given the rapidly evolving health technology assessment landscape in rare diseases and the need for rigorous assessment of the cost-effectiveness of orphan drugs, a firm understanding of how best to develop economic models is crucial. This paper will act as a useful resource for researchers who are aiming to create robust economic models that accurately reflect a new rare disease product’s clinical benefits in relation to its costs.
Read the full paper here:
Economic modeling considerations for rare diseases. Pearson I, Rothwell B, Olaye A, Knight C. Value Health. 2018 May;21(5):515-24.