Dec 20, 2019

A lottery for the most expensive drug in the world

Novartis is giving away about 100 doses a year of the most expensive drug in the world, Zolgensma — a gene therapy that cures children of a deadly disease, the Wall Street Journal reports.

Between the lines: The free drug will be offered via a lottery system, which some patient groups say is inappropriate and unfair, as it fails to account for need.

The program is designed to make the drug accessible to a limited number of patients outside of the U.S. There's a high demand for the $2.1 million drug in countries where it hasn't yet been approved.

My thought bubble: This is great news for a lucky few patients, but it's not a long-term solution to the world's issues with access to pricey drugs.

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Axios

A new multimillion dollar drug

A lab tech thaws a blood bag before genetically modifying a patient's immune cells. Photo: Gerard Julien/AFP via Getty Images

BioMarin Pharmaceutical is eyeing a $2 million–$3 million price tag for its hemophilia gene therapy if it's approved, which could make it the world's most expensive drug, the Wall Street Journal reports.

Why it matters: It's a good reminder that today's pipeline is likely to cause a giant shock to the health care system over the next few years.

Go deeperJan 17, 2020

Caitlin Owens

Health care industry grapples with staggering gene therapy costs

Data: CVS Health; Table: Axios Visuals

The gene therapy pipeline contains several drugs that are likely to cost the health care system billions of dollars in the near future, according to an a new CVS white paper.

The big picture: Drugmakers are already having to come up with creative ways to get paid for high-cost drugs, the Wall Street Journal reported yesterday, and that's before these new gene therapies hit the market.

Go deeperJan 14, 2020

Caitlin Owens

A venture capitalist wants to mimic blockbuster drugs at a lower price

A venture capitalist is launching a company today to create new drugs that mimic the effects of blockbuster drugs, and then sell them to insurers and hospital systems at a lower price, STAT reports.

Between the lines: The key question is whether insurers and hospitals will buy these new drugs over their competitors.

Go deeperJan 13, 2020