Tag: orphan drugs

Economies of Rare

Economies of Rare

Economies of scale are the efficiencies unlocked by large numbers. I’ve come to realize that the efficiencies of scale can emerge at the opposite extreme of rare – especially ultra-rare – genetic disease communities, which can number as few as dozens to thousands of...
A vision for Perlstein Lab

A vision for Perlstein Lab

Today’s post will focus on the vision for PLab. Let’s start with a basic statistical concept- long tail distribution. While this form of frequency distribution has been around since the 1940’s, it gained prominence in fields outside of insurance and...
Biologics and you

Biologics and you

In our last post we talked specifically about orphan drugs that are small molecules. In this post I’ll address the orphan drugs that are biologics. They behave differently: There’s a possible increase in cost/year around 2006 but it doesn’t seem to remain...
Orphan drug prices and approvals

Orphan drug prices and approvals

This post kicks off a series about drug prices in the orphan drug space. We initially wanted to get a sense of orphan drug price trends over the last 30 years — since the passage of the Orphan Drug Act of 1983. There were a few published industry reports...
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