The “Right-to-Try” legislation and autism

It is said in various publications that autism does not affect life expectancy. I do not believe this to be the case. Seizures and accidents (e.g., traffic collisions, drowning) expose autistic individuals to increased mortality. According to a Danish study that followed 340 individuals from adolescent through adulthood, the death rate is twice as high among autism spectrum disorder individuals as in the general population (Mouridsen et al., 2008). In this particular study cause of death varied according to age. Epilepsy-related deaths were more common in younger individuals while diseases of the circulatory system were more common among older individuals. This and other studies (Bilder et al., 2013) have surveyed cases of autism of unknown cause (so-called idiopathic autism) but have not included those that are syndromic (those where the underlying cause/pathology is known). Many cases of syndromic autism are destined to progressively deteriorate and die during childhood. Although this is only a very small percentage of autistic patients, the significance of their plight can only be underscored by imagining that the child affected is your own.

There are drugs available in clinical trials that may be effective in the treatment of some syndromic forms of autism. However, the clinical trial process by which the FDA approves drugs is a lengthy one. In a phase I trial drugs are tested to determine whether they are safe and to establish appropriate dose-ranging. In a phase II trial the safety and efficacy of a drug is tested. A phase III trial involves testing a large population of individuals in order for the trial to gain statistical power thus allowing us to judge the efficacy of the therapeutic response. According to current FDA rules only patients enrolled in these trials can receive the medication. It may thus take a large number of years to get a promising drug approved. However, in many cases, time is a luxury that a dying patient can’t afford. There are many tragic cases of individuals dying several months before a lifesaving drug was approved by the FDA (http://delonline.us/1K13FqL ). In these instances the patients and relatives knew of the existence of these drugs but were stonewalled by various agencies in their effort to obtain them.

Last year 5 states passed Right-to-Try legislation (Arizona, Colorado, Louisiana, Missouri and Michigan). This year 27 states are already considering passing legislation on the subject. Most Right-to-Try advocates promote the use of drugs that have passed safety trials (phase I) for terminally ill individuals. However, instead of having to pass through a cumbersome process asking permission from the FDA to use the drugs for “compassionate care”, it is believed that they should be able to decide with their own physicians what avenues to pursue regarding their own health. It is difficult enough having to deal with somebody dear to you who is dying. Coping is a lot harder when government bureaucracy ties your hands and allows you only to watch the process.

It is likely that only a few individuals will benefit from the Right-to-Try legislation. In many other cases death is to be expected even if they receive the experimental drug or therapy. However, without legislation, the knowledge that you could have done something for your loved one and were thwarted in your attempt, will live forever.

References

Bilder D, et al. Excess mortality and causes of death in autism spectrum disorders: a follow up of the 1980s Utah/UCLA autism epidemiologic study. J Autism Dev Disord 43(5):1196-204, 2013.

Mouridsen SE, et al. Mortality and causes of death in autism spectrum disorders: an update. Autism 12(4):403-14, 2008

4 responses to “The “Right-to-Try” legislation and autism

  1. I don’t know if they mean severely autistic but I guess this explains why I’m always paranoid for my life.

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  2. Very interesting. You forgot to mention also there was a study done a few years ago at Brigham Young University showing that lack of social relationships can be as detrimental to health as smoking, alcohol consumption and obesity. I blogged about this study on autism’s gadfly http://autismgadfly.blogspot.com/2010/07/lack-of-social-relationships-may.html Also, you probably remember the study done By Lisa Croen at Kaiser Permanente showing that persons with autism had higher incidences of hypertension, diabetes, osteoporosis and obesity than the non-autistic controls who were enrolled at Kaiser. This was an IMFAR presentation but not sure if it was published as a study. These also bolster the argument for a shorter life expectancy for autistic people. Many years ago, I also read that there had been cost-benefit analysis studies on the FDA showing the dollars lost in lives not saved by their slow, bureacratic process exceeded lives saved by their testing standards but I don’t remember the specifics.

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  3. I think you are framing the argument incorrectly.The question,as I see it,is not a right to try.For most cases of syndromic autism,but one of a right to a diagnosis in the first place.

    I cannot read the Bilder article,because it is behind a paywall,but our friend Paul Whiteley provided this summary at the time.

    http://questioning-answers.blogspot.com/2012/12/mortality-and-autism.html

    Dr. Whiteley cites two specific diagnoses,Sanfilippo Syndrome and Cerebral Palsy.

    Diseases such as Sanfilippo Syndrome may be the type of obvious cause of syndromic autism you write about,but they are not the most common ones.Systemic autoimmune and metabolic/mitochondrial disorders with autism probably are.But these disorders are still out there on the fringes,struggling for mainstream acceptance and awareness,especially among the medical profession.Perhaps a big reason for this lack of acceptance and awareness was the fact that the research into these types of autism was embraced early on by the antivaccine movement,giving them a taint of quackery.I see it as merely a monumental case of bad coinicdence,that such research was emerging in the early 2000s,at the same time Andrew Wakefield,and Jenny McCarthy were gaining momentum.

    But there is also the fact that an autism diagnosis is a stigma,and a tremendous obstacle in trying to get proper medical care.This is something even those in the neurodiversity movement are willing to admit.These autoimmune and metabolic disorders are diseases that rarely kill early.For the most part,autistics with diagnosed mitochondrial disorders,do not have acute diseases like Leigh’s Syndrome.But both types of disease cause significant chronic disability,and directly impact the severity of the autism itself.It is really long past time for doctors to get past the idea that all syndromic and systemic forms of autism are the handful of classical genetic ones.

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