ABC, the AAP, and Autism

I've been thinking a lot about the American Academy of Pediatrics’ recent protests of the January 31 pilot episode of the ABC television show “Eli Stone.” In the lawyer drama, a mother receives a $5.2 million settlement after she charges that the mercury-laden preservative thimerosol that was used in a vaccine caused her child’s autism. Since many children are diagnosed with autism around the time that they receive a number of vaccinations, some people have linked these vaccinations with autism, especially the MMR (measles, mumps, and rubella) vaccination.

A small but influential 1998 study suggesting a link between thimerosol and autism was later retracted by its authors, and researchers have since found no link between vaccinations and autism. Furthermore, thimerosol was dropped as a preservative in standard vaccines in the U.S. in 2001.

Some parents still refuse vaccinations for their children, though, due to concerns about vaccine contents and the possibility of rare complications. Their choices have lead to regional, sometimes fatal outbreaks of measles and whooping cough (pertussis) among unvaccinated children. Right now, for example, there is a measles outbreak in San Diego, with ten unvaccinated children and infants infected and over fifty quarantined.

The AAP only fueled the vaccination debate, however, with an emotional press release in late January, calling the “Eli Stone” pilot “the height of reckless irresponsibility.” In the letter, AAP President Dr. Renee R. Jenkins says that “if parents watch this program and choose to deny their children immunizations, ABC will share in the responsibility for the suffering and deaths that occur as a result. The consequences of a decline in immunization rates could be devastating to the health of our nation’s children.” The AAP went on to authorize the early release of a February Pediatrics journal article showing that infants expel the type of mercury used in thimerosol over ten times faster than they expel the type of mercury often found in fish. Some researchers believe that if heavy metals such as mercury build up in the body, they might cause autism.

But what this debate really comes down to is parents’ fear of autism, a fear that is especially acute in the San Francisco Bay Area where I live. A CDC study released in 2007 that looked at autism spectrum disorders in selected regions of the country found an average rate of autism of about 1 in every 150 eight-year-old children. Earlier estimates had suggested autism was much less prevalent, as low as 1 in 500 children. The CDC pointed out that it’s unclear whether the autism rates are increasing over time, or whether it is simply being diagnosed and reported more accurately.

Still, no one knows what causes autism, and a quick glance at the numbers makes it look like an epidemic. When I had my first child, I, too, was told by well-meaning acquaintances that mercury-laden vaccinations might cause autism, one of a long list of things to panic about as a sleep-deprived new Mom. If I had my child vaccinated and she developed autism soon thereafter, would it be my fault if the light in her mind dimmed? Was I willing to take that chance?

By the time my children were born, though, thimerosol had been pulled from the vaccines, and pertussis was making an alarming comeback in San Francisco among unvaccinated infants and adults whose pertussis vaccinations had worn off. My children were vaccinated.

There is a frightening childhood illness for every generation, it seems. At one time it was polio, until Jonas Salk developed an effective vaccine for it in the 1950s. My great aunt had polio as a young woman (luckily she recovered fully). My mother, who grew up in the Bay Area, remembers driving by San Francisco’s Shriner’s Children’s Hospital on 19^th Avenue with her family, where many of the young polio patients were dependent on iron lung machines, wondering if that would happen to her. Today, the old Shriner’s Hospital is a retirement community next to a row of modern townhouses, and thanks to the vaccine polio is almost completely eradicated worldwide.

Big Bucks and Orphan Drugs

I had the opportunity a while ago to cover a pharmaceutical company’s drug development meeting. The meeting took place at a lovely and incredibly expensive hotel, and the salmon and petit fours for lunch were a far cry from my usual refrigerator-browsing when I work at home. I knew that the pharmaceutical industry was exceedingly wealthy, but I hadn’t seen it up close before.

The people at the meeting included deferential twenty-something sales representatives and assistants; opinionated mid-career researchers and doctors, who occasionally cracked incomprehensible jokes involving biochemical formulas; and a polite and self-deprecating high-level executive. I’m sure there were plenty of office politics and power plays behind the scenes, but as an observer what I saw was a well-oiled machine. The real politics were external: a corporation versus the FDA (“the Agency,” they called it). The pharmaceutical company was starting human trials of a new drug that they hoped to bring to market if all went well.

For all the people at the meeting, and the expense and effort of bringing everyone together (many from out of town), the new drug was not a potential blockbuster. It was an orphan drug. Orphan drugs are developed to treat rare (orphan) diseases, defined as: diseases that affect 200,000 or fewer Americans, and/or diseases that are uncommon in the developed world (although they may be common elsewhere).

In the past, orphan drugs were too unprofitable for pharmaceutical companies to develop. In 1983, however, the federal government passed the Orphan Drug Act (ODA) to provide tax incentives, research grant money, and marketing benefits to companies that develop orphan drugs. The ODA has had a huge impact on orphan drug development. According to the FDA, from 1973 to 1983, about one orphan drug was brought to market per year. Since 1983, about 250 orphan drugs and products have been brought to market – averaging about ten each year.

The drug in question at the pharmaceutical meeting was designed to replace the current drug treatment, which has several miserable side effects that inhibit patient compliance, for a disease so rare that there might be more people involved in developing and regulating this new drug than there are potential patients for it. But if it survives the trials and the FDA and is brought to market, the drug could vastly improve the quality of life of these patients. It was good to see such altruism from a pharmaceutical company, even if it was prompted by a big carrot from the federal government.

Not-So-Rare Rare Diseases

I’ve been writing about epilepsy recently, a neurological disorder that affects about 1% of the population (especially infants, young children, and the elderly). With the roughly 80 million Baby Boomers starting to retire now, the number of people with epilepsy will increase even more, since the incidence increases in people 65 and over.

Despite these statistics, however, epilepsy research is underfunded for its incidence rate. This is due in part to stigmas that still surround epilepsy, such as the misconceptions that epilepsy is a sign of mental illness or low intelligence, or that it is contagious. These stigmas make some epilepsy patients and families reluctant to speak out about the disease, and consequently there is less patient advocacy for research funding. In medicine as in life, the loudest voices often get the most attention.

It doesn’t help that the National Institute of Neurological Disorders and Stroke (NINDS) lists the National Organization for Rare Diseases (NORD) website as a helpful organization for epilepsy patients, thus implying that epilepsy is a rare disorder. Perhaps NINDS refers readers to NORD because epilepsy is an umbrella term for a group of benign and malignant neurological syndromes, and some of these syndromes are less common than others. Since the incidence of epilepsy is bound to increase in the future, however, I think it’s important to rethink how to categorize epilepsy. It is complicated? Yes. Is it rare? No.

Semantics, stigmas, politics, money: how is medical research funded?

Emergency!

It happened so quickly, I hardly had time to understand it. At my daughter’s swim lesson this week, as I sat across the pool and waved at her, one of the other children in the class lost her grip on the side of the pool and began bobbing up and down in the water. Just as I thought, “wait a minute - what’s that kid doing?” the lifeguard ran over and pulled her out. The instructor was just a few feet away in the water; the girl’s mother was about 10 feet away on the side of the pool, and a posse of parents were watching their children swim in different classes – and yet the girl lost her grip and went under, almost unnoticed.

The speed at which emergencies happen still shocks me. The day my daughter fell on the playground several years ago, I was standing right next to her but had looked away for a moment. When I looked back, I found her crying and bleeding, the skin split under her chin. How quickly can I stop what I’m doing and thinking, understand what just happened, and react the right way when I need to? Smoke pours out of a house window, a woman collapses on a train, a car lies upside down on the road, its wheels still spinning – I have witnessed all of these.

An emergency requires you to stop and focus, and that can be hard to do because we’re so unfocused much of the time. We’re often multitasking, visually bombarded with print and video images, and thinking of the next thing we have to do – not what’s in front of us. I think that we might be worse at handling emergencies now than we were in the past, when it was easier to see if something seemed out of place, easier to hear if someone cried out.

Hat Trouble

Years ago, I was invited to observe a craniotomy. I was writing a patient education booklet about brain aneurysms at the time, and one of the doctors I had worked with extended the invitation to the medical illustrator, who was busy and passed it on to me. I was curious to see it, but I had never observed any surgeries before, and thought a craniotomy would not be a good place for a former English major to start. I had visions of fainting as the surgeon cut and peeled the skin back to reach the patient’s skull, knocking over trays of instruments with such a clatter that the surgeon’s hand would slip, with gruesome consequences.

Although I find medicine fascinating, and I’ve been known to page through pictures of diabetic foot ulcers over lunch, I am not now nor will probably ever be a clinician. I know that hands-on experience is completely different from book knowledge and PubMed articles. Since I write about medicine, though, when my husband took a business trip to Boston he brought back a Harvard Medical School baseball cap from the Coop in Harvard Square.

I’m a hat person who constantly loses hats, and I wear that hat because often I can’t find any other hats to wear as I rush out the door. Besides, the quality is quite good: cloth with a metal buckle to adjust it, rather than a cheap plastic fastener. But I knew the hat might have some unwanted consequences. I have been asked by other Moms at various playgrounds whether I went to “HMS” (to which I replied “what?” the first time someone asked). One Mom turned away in a huff when I said no.

My greatest concern about the hat is that some day I might be wearing it at a playground when a child falls and is critically injured. As the parents swarmed around to help, a Mom or Dad might turn to me and say, “You! Harvard doctor! Save this child!” I know first aid and CPR, but beyond that (ideally before I needed these skills) I would call in the professionals. Perhaps if a child is injured and a parent calls out for help, the first thing I should do is staunch the wound with my hat as I elevate it above heart level, so no one can see what it says and expect miracles.

Lessons from Cesarean Section Rates

Evidence-based (data-driven) medicine, or EBM, is gaining momentum as an antidote to the perils of of groupthink in medicine. Just because something has "always been done this way" doesn't mean it is right; EBM argues that research data should better inform how medicine is practiced day-to-day.

It's interesting to apply EBM principles to cesarean surgery rates in this country. Far more women deliver by cesarean section now than in the past. Why? Some researchers, such as Dr. Frederic Frigoletto, Jr. at Harvard Medical School, argue that the increased rate is primarily due to complications caused by increased obesity rates and advanced maternal age. Some women also choose elective cesareans in order to control the timing and nature of the birth, he explained in a 2006 WebMD article.

Although cesarean rates have increased across the country, not every area has the same rate. Rates are generally higher in more conservative areas of the country and lower in more liberal areas. A study of California cesarean rates presented at a health policy meeting in 2000 found that the cesarean rate was up to 2.5 times higher in some regions of the state than in others. These facts have made cesarean rates a political and feminist issue as well as a medical one.

Cesarean sections are also extremely profitable for hospitals. The California study noted that cesareans are more common among for-profit hospitals than not-for-profit and teaching hospitals, which suggests a profit motive behind the surgeries as well.

On the other hand, are obesity rates (and/or maternal age) simply higher than average in some areas, potentially leading to a local increase in medically-necessary cesarean sections? If this is true, does a hospital with high cesarean rates have an ethical obligation to put some of these surgical revenues toward programs to decrease the obesity rates in its community? (Trying to decrease maternal age in a community is a thornier issue, I think). And how would this ethical obligation undermine a hospital's bottom line? A decrease in cesareans, after all, means a decrease in revenue.

Medical data is a starting point for these discussions, not an end point. All data needs some context. It's good to remember this as the presidential elections approach and the candidates try to summarize their views on the health care system into marketable sound bites. A few sentences, or a single anecdote, rarely tell the whole story.