Therapies for advanced cancers pose a special challenge for health technology assessment organizations in many...

Health technology assessment organizations evaluate medical therapies and technologies to help inform coverage and reimbursement decisions for payers around the globe. Even as they establish strict review processes, these organizations--and the reimbursement authorities that use their assessments--have sometimes handled cancer interventions with special care. We found that some countries have created separate health technology assessment pathways for cancer treatment, while others have eased access to cancer treatments through end-of-life or disease-severity exceptions within health technology assessment policies. In the United States, although no separate evaluation pathways exist for cancer, cancer drugs receive special status by virtue of unique Medicare rules covering off-label indications. Worldwide, we demonstrate that health technology assessment organizations are struggling with cancer's "exceptionalism."