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The Most Expensive Medicines are Worth Every Penny

Posted by | 5:03pm on Tuesday, February 23, 2010

First, it’s simple economics: fewer patients means higher drug prices. 

A Forbes article came out this week about the most expensive medicines on the market, naming nine drugs that cost more than $200,000 per year.  The author singled out Alexion Pharmaceutical’s Soliris, Shire Pharmaceutical’s Elaprase, and BioMarin Pharmaceutical’s Naglazyme. These are treatments for patients with rare disorders.

Simple Economics
I have several issues with the sensational nature of this piece. First, it’s simple economics: fewer patients means higher drug prices. From the article: “In the inverted world of drug pricing, the fewer patients a drug helps, the more it costs.”  How is this inverted?  Isn’t that how all economics in a capitalist system works?

The bar set by the FDA is not lower, in terms of safety and efficacy, for drugs that address rare diseases. In fact, it’s much harder and sometimes impossible to field a multi-arm clinical trial because there are so few patients and virtually no natural history for many rare disorders.  The Orphan Drug Act offers incentives for companies to have extended monopolies on the orphan drugs they bring to market, and since this act was put in place in 1983 more than 200 orphan drugs have been approved.  Before the Orphan Drug Act there were none.  With our current drug development system, the only chance a patient with a rare disorder has is if a company takes on a risky drug development process through the FDA process designed for bigger market drug trials.

The NIH and FDA are working with the National Organization for Rare Disorders (NORD) to develop a better process for bringing drugs to market.  Six months ago at the NORD Rare Disease Summit, I heard Francis Collins’ plan to use the NIH to address the three main areas of drug development.  He likened the drug development process to a football game with drug companies currently having to pick up the ball from the 5 yard line and run it all the way down the field.  He would like to have the government move the starting point to the “50 yard line” to speed up process for drugs coming to market.

Collins discussed:

  • Building on the experience mapping the human genome so the government can systematically map basic molecules, instead of having drug companies use trial and error.
  • Training more scientists to develop assays that can be used at government facilities that offer high speed testing of 100’s of thousands of drug possibilities, and
  • Creating a national registry for collecting the natural histories of rare disorder patients to test efficacy against.

Marketing is Different for Rare Disease Therapies

The Forbes article notes: “There are so few patients that companies don’t have to invest as heavily in marketing.” Not necessarily, since marketing for a rare disorder is more about supporting the patient all the way through the patient journey.  Most rare disease patients are the primary driver of their diagnosis and treatment, so if a pharma company wants people to know about their therapy and adhere and live longer, they have to do this much more directly than with in a bigger market.  Doctor’s don’t have the bandwidth to keep up and pharma must step more into the support role for these patients and families.

It’s in the Public Interest
Innovation happens at the margins.  As Dr. Tim Cote, who oversees the FDA department for orphan drugs, said at the Rare Disease Summit:

“Most of our medical science comes from people with rare diseases. We learn what aging is through Progeria. We learn that the DNA can repair itself through xeroderma pigmentosa. We learn what normal hemoglobin that we all have is through people with hemoglobinopathies… These people with rare disorders form the very fundamental basis of our materia medica. They’re what we studied in school. They’re what we understand in treating all disease that we ever reach. So somehow we need to communicate that – that these are not just odd things that happened to some people, but this collection of rare disorders really are the essence of what medicine is for all people.”

To learn more, read the transcript from the Rare Disease Summit.

About Wendy White

Since founding Siren Interactive in 1999, Wendy has been recognized as a thought leader at the intersection of niche pharma brands, patient empowerment and online marketing. Her vision for how the internet can facilitate interactions and provide crucial information that patients, caregivers and their healthcare providers previously struggled to find has propelled Siren to the forefront of relationship marketing for rare disorder therapies.

View other posts from Wendy

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    23 February 2010 at 11:02pm
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