Reimbursed Price of Orphan Drugs: A Value Based Framework

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

BACKGROUND The rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates. OBJECTI...

Orphan drugs. Relating price determination to disease prevalence.

Cancer drugs: high price, uncertain value

Most cancer drugs recently entering the European market do so without clear evidence of extending or improving quality of life, new research published in The BMJ has found. The findings raise serious questions about why the current regulatory environment supports the approval of cancer drugs that may leave patients at risk of experiencing toxicity and reduced quality of life without deriving me...

Developing a Patient-Directed Policy Framework for Managing Orphan and Ultra-Orphan Drugs Throughout Their Lifecycle

INTRODUCTION Policy decisions related to orphan and ultra-orphan drugs challenge traditional decision-making processes and often frustrate those affected by them. In general, these drugs are associated with significant uncertainties around clinical benefit, 'value for money', affordability, and 'adoption/diffusion', all of which arise from a lack of available high-quality evidence. Increasingly...

Orphan drugs revisited.