Right to Try: My Latest Thoughts

So Paul Knoepfler has an excellent article on his take on Right to Try on his blog (find it right here.) I think that it’s balanced in a lot of ways, and yet I do have to say that I think there are more issues that need to be take into account. There’s a piece of advice that I’ve seen floating around the web for quite a while: if you write something online that you feel is significant, make sure that it’s preserved in a more permanent way than on Facebook, as a comment on someone else’s post, etc etc etc. That’s what I’ve done here. 🙂 So… here’s my comment on Paul Knoepfler’s post on Right to Try issues.

I have every reason to have a very personal interest in the Right to Try laws. Four years ago, I was diagnosed with a disease no doctor had ever seen before. All they could be sure of was that it behaved like age-related macular degeneration or Stargardt’s macular dystrophy, and that it would end in blindness. There was a drug in development that has now passed Phase I testing with flying colors, and my doctor told me that it would help me– if it weren’t many years away from approval. I had a remission that nobody could explain either, and I’m still in remission. But I have not been cured.

And when I first found out about Right to Try, I was not a fan. I saw all of the problems and limitations, and there are a lot of them. For one thing, if I had another flareup, the laws still wouldn’t help people like me, because they are limited to patients with an immediate terminal diagnosis. For another, they are not applicable to anyone right now because of the constitutional clause of federal supremacy. The list of problems goes on. After a lot of research, study, and talking to other patients, though, I have to say that I now support Right to Try.

The reason is this: the only way to support a theoretical Right to Try in the face of all the limitations is if these laws will provide pressure for change at a higher level, and if there is just no other realistic way for this pressure to occur. An excellent article in the Yale Law Review Journal pretty much laid it out.

Definitely check it out here– it shouldn’t be behind a paywall.

The only way *this* is justifiable is if there are huge obstacles blocking effective treatments for patients in ways that are simply not being addressed. This may not be the case for traditional drug therapies, but for cellular therapies, it is. Stem cell clinics have too many problems to count, but there are good reasons why people are going to them. Cellular therapies exist that are stuck in Phase I testing and ideally would have been on the market many years ago.

I don’t blame the FDA for everything, largely because the way that stem cell research was held back for over forty years is because of special interests rather than being the FDA’s fault. But whatever’s happened in the past, we must deal with the situation as it is now. And right now, it is a fact that other countries are handling the issue of these novel therapies very well, and we are not. The European Medicines Agency, for example, has the Adaptive Pathways Initiative, which provides for the early approval of drugs for restricted patient populations based on small initial clinical trials. Approval is then expanded progressively with input from health assessment bodies. The EU has certainly not somehow thrown out all regulation. But it’s effective regulation.

The RPE drug that could cure me (and the 180 million other people in the world with all types of macular degeneration) already has better results than most drugs used for any purpose that are out on the market right now, and the results have twice been published in a peer-reviewed medical journal (the Lancet.) That’s not even mentioning the supporting results from South Korea, which were also published in a peer-reviewed medical journal. There’s no other treatment for well over 90% of all types of macular degeneration. This drug should have been given breakthrough designation by the FDA long ago, and it still hasn’t been.

There’s also the issue of the FDA’s lack of transparency and evasiveness when it comes to the issue of who actually gets access to the compassionate/extended access program. The Right to Try bill recently passed the Oregon House, and I went to the legislature page to read the supporting and opposing material for it. There was a good article in the New England Journal of Medicine: Practical, Legal, and Ethical Issues In Expanded Access to Experimental Drugs.

A rare change to read this article, which would normally be behind a paywall. Courtesy of the Oregon State Legislature! 🙂

The claim was made that all of the work involved with filing documentation for expanded access for a patient would only take “eight hours” of a physician’s time. Well, the problem is that the FDA itself says that the actual figure is *one hundred hours*.

http://www.raps.org/Regulatory-Focus/News/2015/02/04/21243/From-100-Hours-to-1-FDA-Dramatically-Simplifies-its-Compassionate-Use-Process/

I went to the original FDA document in the federal register from 2009 (can’t find the link right now, but I have it,) and read through it. Not what you’d call fun reading, but it’s pretty important. The FDA did indeed claim that only eight hours would be required, and there were lots of facts and figures to back that claim up. So what happened? How did eight hours expand to one hundred? How can anyone think that this provides anything remotely in the realm of decent access for the average patient when HMO’s require doctors to schedule appointments every fifteen minutes? There has been absolutely no transparency here, and the FDA did it to themselves.
Add they say now that it will be 45 minutes, but last time, eight hours was the figure claimed in theory, and it somehow expanded to one hundred hours in reality. How do we know that the same thing won’t happen this time? And even if it doesn’t, even if 45 minutes really does turn out to be accurate, the last proposals were made in 2006, and the new laws weren’t passed until 2009. That’s three years. A lot of terminal patients can die in three years.

I’m working on quite a few articles about Right to Try, and this clearly could go on for many pages, so I’ll cut it off here. But I would really ask people to think deeply about all sides of this issue. No matter which way you look at it, Right to Try is not a simple thing. But it exists for a reason, and it’s largely that patients have been brushed aside and not listened to. It’s why we ended up here in the first place. And this has to change. Right now, Right to Try represents a realistic chance for this to happen.

Cathy Danielson
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Cathy Danielson

In 2011, my life was shattered when I was diagnosed with a mysterious, incurable disease that always ends in blindness. The only hope on the horizon was a drug that broke all barriers in early testing. This drug, which replaces damaged cells in the eye, comes from the new class of stem cell based treatments that could cure the incurable, providing hope for patients with cancer, heart failure, ALS, cerebral palsy, and many more fatal and disabling diseases.

I survived the disease, and I now have the only remission on record. But a remission is not a cure. The stem cell drug I need for a real cure is now rapidly moving towards approval in the rest of the world, but in the USA, it—and all other cellular therapies-- are still stuck behind prejudice, ignorance, and lack of funding. Hundreds of millions of desperate patients with incurable diseases need these drugs.

That’s why I’ve gone on to fight for greater public education on stem cell drugs, knowing that our laws must be changed so that all of us can get access to the best treatments instead of our health and our lives being held hostage by special interests.

I’m now a patient advocate whose work on stem cells and patients’ rights has been published in outlets such as the Oregonian. I am a manager at popular science and financial blog www.stemcellinvestor.com and a frequent speaker at many venues across the spectrum, including churches, scientific conferences, and atheist groups, and everything in between. I’m also an advocate for Right to Try laws that would allow access to experimental medication for terminal patients at the state level. Read the entire story in my upcoming book, And the Blind Shall See: A Skeptic Patient Surprised by Faith, Science, Family, and Miracle Cures.
Cathy Danielson
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