One Opinion on How To Form an Opinion

January 15, 2010

by Mark Dunning

My wife is a fan of the sniff test. The expiration date on a carton of milk doesn't matter. She still wants to stick it under my nose and ask "What do you think? Is this OK?" I usually offer my opinion by gagging or shrugging. The interesting thing is that she doesn't always act on my opinion. Sometimes I'll say it's good and she'll throw it out, sometimes I'll run screaming from the room and she'll pour it on her Cocoa Puffs. That's OK. I'm just offering my opinion. I don't have any particular proof that it's good or it's bad. The checkbook is a different story. I know what's in the bank and I know our weekly expenses. So when my wife asks if we can afford something, she (usually) heeds my advice because I have the facts.

I get asked my opinion on Usher syndrome treatments a lot. I find this surprising because as any regular reader of this blog knows, I'm generally light on facts. But that's the problem when it comes to Usher syndrome treatments; we're all light on facts. You see there are no widely accepted, scientifically proven treatments for the vision component of Usher syndrome. So just about everything at this point is an opinion. There are few statements of debits and credits and cold hard facts. That leaves families, like my wife with her spoiled milk, to decide for themselves if a treatment is good or bad. My advice? Line up a lot of noses before pouring anything on your Cocoa Puffs.

In my last post, I urged families to fully research any proposed treatment with a level head before embarking on it. All too often desperate families are willing to jump on the first positive advice they get without fully vetting it first. It's human nature to run to the light when you're stumbling around in the dark.

I find families take chances with unproven treatments for Usher syndrome that they would never even take with a proven treatment for other conditions. We're all familiar with the concept of getting a second opinion. A doctor says you need hernia surgery, you goes ask another doctor if he or she agrees with the first. Yet families with Usher are often so relieved that someone is offering something that might help, that they just jump in without question. In fact, families are often careful NOT to ask questions because that might expose some reason why the treatment is not appropriate causing the physician to withdraw the treatment offer and stifling that glimmer of hope. It's the exact opposite of how they should act.

Now no one sniffs around for Usher syndrome treatments more than I do. So here's what I do to determine if something passes the sniff test:

Governmental approval

Say what you will about our governmental bureaucracy, but it works pretty well when it comes to ensuring treatments are safe. There are no Usher syndrome specific treatments that are currently approved by the Food and Drug Administration or recommended by the National Eye Institute that I know (again, I might be light on the facts). There are approved treatments that might be appropriate for Usher syndrome such as Cochlear Implants and Vitamin A but these are not Usher specific treatments.

Still, that's the first thing I look for: Is this treatment or this study approved by the NEI or the FDA? If not, I'm skeptical. As a result, I tend to be more skeptical of any treatments outside the US. That may not be correct thinking since there are many reputable and talented researchers outside this country. In fact, my wife's cousin, who is British, is the first call we make when we need information. So while I'm more skeptical of international efforts, I obviously don't discount them completely.

Clinical trials are different from treatments. A clinical trial is a test of a potential yet unproven treatment. These are enticing to many families who have already heard over and over that there is no cure for Usher. So if there is no cure presently, something that holds promise might be worth trying.

I'll talk more about clinical trials in a later posting, but like treatments, I would want to know that a clinical trial met governmental requirements before I participated. You can find a registry of clinical trials maintained by the US government at this site developed by the FDA and NIH. The nice thing about this site is that it also includes most internationally and privately funded clinical trials as well as those funded in part by the US government. It's a pretty comprehensive list. I personally would be very wary of participating in any that is not on this site.

Get the Facts

Just because the government approved a treatment or a trial doesn't mean it's right for you or your family. So the next thing I do is judge the science for myself. Now I'm not a physician or a research doctorate. The details do go over my head at times. Jennifer wrote recently about how to spot good science which is a good lesson to follow. Personally, I look for several things in particular.

How long has this treatment been studied?
How many people have been part of the study?

Who are the people that participated?

How long have those individuals participated in the study?

I'll give you an example. I know of one particular treatment study that's been going on for nearly 20 years with hundreds of participants, many of whom have been participants for the entire life of the study. That's good science right there. Lots of data to back up the findings of the study. If it's been going on that long with that many people, they have probably seen most of the common side effects which makes me more confident it's not going to harm my daughter.

The caveat in this one particular study is that while Usher patients were included, they weren't the focus. So then the same questions come up. Exactly how many Usher patients are we talking about? What type were they?

I personally don't want to go first. If a bunch of people with the same type of Usher have tried it with no ill effects, then I'll consider it. You might be less cautious. That's your prerogative. As I said, this is just my opinion.

For a great list of questions to ask before participating in any clinical trial, check out The Center for Information and Study on Clinical Research Participation.

Deciding Who To Trust

Traditional hernia surgery is FDA approved and has been performed on thousands of patients with great success. However, if I'm due to have that surgery and the doctor comes in smelling of bourbon and slurring his speech, I'm not having it. Why? Because I don't trust him. Trust is the most important factor in deciding if a treatment is right for you.

For me, trust in a physician comes down to one thing (aside from sobriety, of course): Will he or she take the time to answer, thoughtfully, every one of my questions? In my case, this is my daughter we're talking about. I don't care how many degrees or plaudits a person has, they are not touching my daughter until I understand every single detail of what is going to happen. If they don't have the time to spend to make sure that I am completely comfortable with a treatment, then they don't care enough about my daughter and they aren't going near her.

Of course, there are other factors I consider as well. Degrees help. Obviously I weigh the opinion of someone who is medically trained more than the average guy on the street. But I have found that many physicians don't know the first thing about Usher syndrome and in those cases, I actually value the opinion of families with Usher over those of a particular physician. So degrees are important, but expertise in the field matters more to me.

Finally there is family. I mentioned earlier my wife's cousin in London. She's at the top of our trust list because she is medically trained, an expert in the field, family, and always willing to spend an hour explaining a treatment to us. Bella's godfather is also a physician. Nobody cares more about her well being than him, but he is not an expert on Usher syndrome. We talk to him about proposed treatments to see if they at least are logical medically, but I don't think I'd trust a treatment he proposed even if his heart was in the right place (not that he would do that). And of course there is Grandma and Grandpa and aunts and uncles and cousins and other people who care deeply about Bella. We consult them for the sniff test, but like my wife with the milk, they don't have a big influence on the final decision.

Finding Experts

One of the reasons we created the Coalition for Usher Syndrome Research was due to the frustration many of us, families and researchers alike, felt in trying to find purported experts. Families, physicians, and researchers alike wanted to be able to quickly find experts in a particular treatment or Usher sub-type who were willing to spend the time to speak to them. Or they wanted to find that expert to provide a second opinion. It was incredibly difficult.

I'd love to say it's easy now, but though it's gotten better, it's still pretty opaque. The best places to check are with the Coalition for Usher Syndrome Research or with the Foundation Fighting Blindness. The Coalition is aware of just about every researcher working on Usher focused projects. The Foundation Fighting Blindness knows just about every retinal related project going on in the world, including those that focus on Usher or might be appropriate for Usher. If neither of those organizations are aware of a particular researcher, I'd be skeptical. I wouldn't dismiss him or her, but I'd be more wary.

Decision Time

At the end of the day, however, there is only one opinion that matters. It's yours. Use the resources and process above to educate yourself then look in the mirror and ask yourself these questions:

Have I learned all that I can about this particular treatment?

Have I fully vetted that information with enough people that I trust?

Do I trust the folks administering the treatment?

Do I feel confident it won't harm me or my family in any way?

Do I truly believe it has the potential to help me or my family?

Then make your decision and trust it. No one is more expert on what is best for you and your family than you.

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