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.
Friday, January 25, 2008
Thursday, November 29, 2007
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?
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?
Monday, October 22, 2007
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.
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.
Sunday, September 30, 2007
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.
Thursday, September 13, 2007
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.
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.
Thursday, August 30, 2007
Uninsured Rates Creeping Up
Earlier this week, the U.S. Census Bureau released the latest statistics on income, poverty, and health insurance coverage (gathered in 2006). The message: more people had health insurance in 2005 than in 2006.
Although the poverty rate fell .3% from 2005 to 2006, the number of people without health care coverage increased .5% to 47 million in 2006. This may be due to a decline in employment-based health insurance, the type of coverage that most Americans have. The number of people receiving employment-based health insurance fell .5% from 2005 to 2006, while the number receiving government health insurance, such as Medicare and CHAMPVA, fell .3%.
Children and certain minorities are especially likely to be uninsured. The number of uninsured children increased 9%, from 8 million to 8.7 million, between 2005 and 2006. Among the entire population, Hispanics are most likely to be uninsured (34.1%), followed by American Indians/Alaska Natives (31.4%), Native Hawaiians/Other Pacific Islanders (21.7%) and African-Americans (20.5%).
Mark Twain famously wrote that "there are three kinds of lies: lies, damned lies, and statistics" (inaccurately attributing Disraeli as the source of the quote), so these statistics need some context. The Census Bureau notes, for example, that they surveyed previously unrepresented populations in 2006, including prison and college dorm residents, which could skew comparisons between 2005 and 2006 data.
Still, I think that children, the middle class, and the largest minority group in the country (Hispanics), among others, deserve some better health care options. A pediatric ER doctor told me recently that there is a tacit understanding between parents and ER physicians that the ER has, by necessity, replaced primary care for uninsured pediatric patients. These parents know that many of their visits are not medical emergencies, and the physicians understand that the parents have no other option for health care. That is what health care has become for many people: these silent bargains and unspoken apologies.
Although the poverty rate fell .3% from 2005 to 2006, the number of people without health care coverage increased .5% to 47 million in 2006. This may be due to a decline in employment-based health insurance, the type of coverage that most Americans have. The number of people receiving employment-based health insurance fell .5% from 2005 to 2006, while the number receiving government health insurance, such as Medicare and CHAMPVA, fell .3%.
Children and certain minorities are especially likely to be uninsured. The number of uninsured children increased 9%, from 8 million to 8.7 million, between 2005 and 2006. Among the entire population, Hispanics are most likely to be uninsured (34.1%), followed by American Indians/Alaska Natives (31.4%), Native Hawaiians/Other Pacific Islanders (21.7%) and African-Americans (20.5%).
Mark Twain famously wrote that "there are three kinds of lies: lies, damned lies, and statistics" (inaccurately attributing Disraeli as the source of the quote), so these statistics need some context. The Census Bureau notes, for example, that they surveyed previously unrepresented populations in 2006, including prison and college dorm residents, which could skew comparisons between 2005 and 2006 data.
Still, I think that children, the middle class, and the largest minority group in the country (Hispanics), among others, deserve some better health care options. A pediatric ER doctor told me recently that there is a tacit understanding between parents and ER physicians that the ER has, by necessity, replaced primary care for uninsured pediatric patients. These parents know that many of their visits are not medical emergencies, and the physicians understand that the parents have no other option for health care. That is what health care has become for many people: these silent bargains and unspoken apologies.
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