Orphan drugs: expensive yet necessary

Orphan drugs: expensive yet necessary.

Whether the prices of certain orphan treatments are justified is highly controversial. One argument is that such therapies should not be funded through the public purse or private health plans because a patient with a rare disease requires more than their 'fair share' of a limited health care budget. Orphan medications can also be denied because they fare poorly in the cost-effectiveness assess...

Rare diseases and expensive drugs

Immunologists/allergists are a physician group that is familiar with caring for patients with rare diseases. Conditions that come under the umbrella of our specialty include various primary immunodeficiency diseases, mast cell disorders, various autoinflammatory diseases and diseases of the complement cascade including Hereditary Angioedema (HAE). A rare or orphan disease is defined by the Euro...

Orphan drugs revisited.

Commentary Orphan drugs revisited

C. MCCABE, A. TSUCHIYA, K. CLAXTON and J. RAFTERY From the Section of Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Department of Economics, University of Sheffield, Sheffield, Department of Economics and Related Studies, University of York, York, and Wessex Institute for Health Research and Development, University of Southampton, Southam...

Ethics roundtable: Using new, expensive drugs

Costly genetically engineered therapies, which threaten to cripple the health care industry economy and undermine the common good if applied indiscriminately, loom on the horizon. The spectrum of applicable candidates include moribund nursing home patients at the end of life. They will be fair game for therapy that will ultimately send them back to nursing homes to return later with the same co...