Health technology assessments of devices drawn on experiences from orphan medicines

Health technology assessment (HTA) is important when launching a new drug or medical device.
This thesis is initially focused on orphan medicines, with the burden of nephropathic cystinosis as an example. In the second part, the focus was on medical devices. Orphan drugs and medical devices have the following in common:
• Pricing and other factors that influence the cost of care
• Levels of evidence in HTA
• Use of quality of life data
This thesis demonstrated that the price of orphan drugs is linked to the rarity of the disease. However, there is a lack of high-quality evidence to support premium pricing.
Key focus of both industries for HTA assessments should be on increasing the levels of clinico-economic evidence both in terms of quality and number of publications. Real-world evidence should also contribute to the growing evidence base, especially where it is difficult to generate enough evidence in a clinical study.
Payers and HTA bodies should give more weight in decision-making to patients’ quality of life. An important recommendation for HTAs is to look at all stakeholders in health care, therefore taking a broad societal perspective.
In summary, substantially more clinico-economic evidence and experience in HTA assessments of orphan drugs and medical devices will help decision-makers so that these technologies may become more widely and quickly available to patient populations.