Ron and Ed welcomed to the show James Geraghty. As a biotechnology executive, Jim has been a passionate participant in the orphan drug revolution since its inception. We talked about his book, Inside the Orphan Drug Revolution, The Promise of Patient-Centered Biotechnology, which is in part a history, with eye-witness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies.
Use these show notes to follow along with the audio…(scroll a bit further to see what a bot came up with)
Segment one:
Jim’s book is a labor of love. He wrote it because there is a mission that people should know about. It has transformed society and needs to be understood. It is the orphan drug revolution. https://www.amazon.com/Inside-Orphan-Drug-Revolution-Patient-Centered/dp/1621824683
Austrian economics has nothing to do with Austria much like an orphan drug has nothing to do with orphans. The drugs treat orphan diseases. They were “orphaned” by pharmaceutical drug companies in favor of quick wins as opposed to the much more difficult.
Today there are over 7,000 monogenic disorders and over 6,000 with no therapy available to them. More information is here: https://www.uptodate.com/contents/inheritance-patterns-of-monogenic-disorders-mendelian-and-non-mendelian
The Orphan Drug Act of 1983 addressed the need for treatments and research for diseases such as ALS and muscular dystrophy that are considered rare in the United States. https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-act-relevant-excerpts
There are misguided campaigns to cap or reduce drug prices. Whenever there is a movement in this direction, innovation in the segment of the market being targeted dries up.
The explanation of benefits is one of the most confusing things you can receive as an American healthcare consumer. Ed wrote about this recently here: https://www.edkless.com/blog/2022/10/on-medical-billing-and-payments
Segment two:
Abbey Meyers is considered the mother of the orphan drug revolution. She is referenced in Jim’s book and also on the FDA website at this link: https://www.fda.gov/industry/fdas-rare-disease-day/story-behind-orphan-drug-act
The 1983 Orphan Drug Act completely changed the face of therapeutics for rare disorders. By 1990 FDA had designated 370 products for orphan status, and of these 49 were approved for orphan indications.
Initially, President Reagan was going to veto the Orphan Drug Act of 1983. He was not in favor of government intervention. Mothers who knew Nancy Reagan worked in the background to help President Regan see the benefits. Ultimately, he signed.
Every year there are hundreds of drugs passed for orphan diseases. Patients today who benefit are in the millions. This has been a global revolution with patients who benefit all around the world.
Segment three:
Ceradase was the initial orphan drug developed by Genzyme. In the initial trial of 7 patients, 6 showed no improvement. “That’s an anecdotal case and you can’t build a company on it.” Well, they did. And it worked.
Our understanding of genetics continues to explode in large part due to the human genome project. Science has come to understand DNA and RNA and much of our understanding about mRNA was developed in the pursuit of treating rare diseases.
In the context of orphan drug development, “N of 1” is an important concept. An N of 1 trial is a clinical trial in which a single patient is the entire trial. They are a single case study.
Can we get to a point where the FDA can approve a broad platform and then patients can be treated as individuals who have ultra-orphan diseases? Jim certainly hopes so although we are not there yet.
Segment four:
International rare disease day is on February 28th. This is closely tied to Jim’s book, “Inside the Orphan Drug Revolution”. Learn more at this link: https://www.rarediseaseday.org/ @rarediseaseday
Even in countries with universal health insurance, they have other ways to block orphan drug treatments and make it difficult to obtain for those in need.
A big THANK YOU to Jim Geraghty for joining us today to discuss his book! “Inside the Orphan Drug Revolution” is available at this link: https://www.amazon.com/Inside-Orphan-Drug-Revolution-Patient-Centered/dp/1621824683
The bots are coming for us!
For a few weeks now, we have been experimenting with a new feature from Otter.ai. Many of you will know that some of the current AI solutions have caught Ron and Ed’s attention during the bonus episodes. We let Otter.ai loose on the audio version of this show and here is what it (he? she? they?) came up with:
Casa Bonita updates. 0:00
Why didn’t Trey Parker invest in that? 2:21
Cannibal the Musical –. 3:20
Introduction to James Garrity and his book. 8:19
What is an “orphan drug”? 13:00
Drug pricing as a problem for patients. 18:30
Jim’s favorite story from the book. 21:40
The Orphan Drug Act of 83. 27:03
What is the term “n=1”? 31:44
How the FDA is understaffed and underfunded. 36:33
N equals one technology and pandemic preparedness. 41:25
What is an ultra-orphan disease? 45:51
How do we make these things affordable for all of these patients? 49:09
Jim talks about the challenges of orphan drug access in the developing world. 52:53
What is the full-loaded cost of gene therapy? 57:04
Bonus Content is Available As Well
Did you know that each week after our live show, Ron and Ed take to the microphone for a bonus show? Typically, this bonus show is an extension of the live show topic (sometimes even with the same guest) and a few other pieces of news, current events, or things that have caught our attention.
This past week was bonus episode 416 - It's all about payments. Here are some links discussed:
Click the “FANATIC” image to learn more about pricing and member benefits.