rare diseases

Patient advocates speak out on partnering with life science companies

What does patient centricity mean from a patient’s point of view, and what kind of support do patient advocates need from life science companies? Three prominent patient advocates shared their perspectives. In 2010, Nick Sireau, a London-based father of two boys with the rare progressive genetic disease alkaptonuria (or AKU, also known as “Black bone […]

The unmet need for precision medicine in Lyme disease

Lyme disease cases and complications are rising, yet current treatment regimens are proving to be insufficient. Clarivate Senior Science Editor Shyama Ghosh and Managing Editor Stephen DuPraw collaborated with Principal Epidemiologist Swarali Tadwalkar to review the current and emerging treatment landscape and what’s needed to better serve patients.   In the U.S., about 30 million […]

Cystic fibrosis: Market shake-up to come?

This article, a Cortellis Market Insight report, is adapted from the author’s comprehensive Cystic Fibrosis Disease Report. Publication of the report coincides with Cystic Fibrosis Awareness Month in May in the U.S. and Canada, which aims to raise awareness and improve understanding of the disease. Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies have transformed […]

Orphan drug access varies across multiple markets

European countries differ in their value assessment of new orphan drugs, but steps can be taken to create a more harmonized, comparable and rational evidence-based decision-making system in Europe, two experts from the London School of Economics Health reported at the Ninth Orphan Drug Congress 2018. The presentation by Aris Angelis and Panos Kanavos covered […]

Shire’s Takhzyro expected to dominate in hereditary angioedema

The 2018 edition of Drugs to Watch, the annual industry forecast and analysis from Clarivate Analytics,  anticipated the approval and market entry of Shire’s Takhzyro (lanadelumab).  The drug was approved in the U.S. under accelerated review, with Orphan Drug, Fast Track and Breakthrough Therapy status, as expected in August 2018, for the prevention of hereditary […]