A Rare Breed

I have been doing a lot of reading lately after a period last year when I did not touch a book. Mostly these books are related to science and published in the last year and I am devouring them at a rate of one per week or two. A recent one was very compelling and was eagerly read over breakfast, lunch and in the gym over a few days this week. The book is “A Rare Breed” by Daniel S. Levine and Daniel P. Maher. This book is different in that its published by Biomarin. Yes that’s right, a drug company published a book and apparently there is a tie in movie too. The book chronicles the 20 year story of what is primarily a rare disease drug company. Surprisingly it does go into some uncomfortable territory. It reveals that some CEOs and others at the company may not have been perfect. No surprise, especially for those driven by getting drugs approved. But more importantly the book demonstrated the Biomarin perseverence, overcoming the ups and downs, the bad and the good deals. The book also shares a few patient stories. As someone that has worked in rare diseases for a relatively short time I found the book spoke directly to me. It gave me useful advice and it definitely provided a shot of reality in how much effort and how big of a team you need to get a treatment approved (in many cases here they are biologics).

There were parts were I felt like a fly on the wall, at least I think some of the meetings described would have been fascinating to attend. Skipping through 20 years and a lot of success and failure in 220 pages is some accomplishment.  There were periods of repetition of a few rare diseases but not too much. What was missing for me was literature, references to go along with some of the background. Yes it felt like a booster for the company at times but rightly so they have done so much for so many with relatively little money in comparison to the truly big companies in the same space.

The book has to be required reading for any parent / patient advocate, scientist, VC, regulator. I found myself texting snippets to rare disease parents and scientists to get their take. I think this sets the standard for a book on rare disease R&D and for me it may become something of a touchstone. I want people to read this book because they will learn what it took to get the treatments approved. They will learn that it takes exceptional people to drive this.  By the end of the book it is apparent that the company has always been fighting with what it wants to be. Fighting to remain independent. Fighting for the rare disease patient…  Now I need to watch their movie.





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