Policy

On June 7, 2003, in Uncategorized

Pharmacogenetics–the study of individual, genetic-based reactions to drugs, leading to personalized medicine–is making progress, and posing deep and serious questions about the future of healthcare practice and policy.
To the extent that drug ads contain genuine health educational information, the finding that viewers don’t pay much attention is not to be welcomed, argues at least one doctor.
Some drugs are very expensive, yet sometimes the cost is justified in economic as well as in human terms.
Belize is competing with Canada in offering lower-cost drugs to U.S. citizens.
Technology and healthcare economics may combine to expand the patient base for quack doctors, with serious implications for public health.
Stem cells collected from newborns’ discarded umbilical cords are being sold and used for both FDA-approved and unapproved uses, prompting ethical, quality, and regulatory concerns.

Pharmacogenetics–the study of individual, genetic-based reactions to drugs–is making progress, and posing deep and serious questions about the future of healthcare practice and policy.

Tests now exist for gene-based reactions to drugs and dosages for numerous diseases, and they may soon become pervasive. They include:

A test that predicts which children with leukemia will benefit from chemotherapy and which could be devastated by a normal dose.
A personalized vaccine made from the DNA of the tumor of a non-Hodgkin’s lymphoma patient.
A test for inherited differences in drug responses, and (soon, from the same company) a test for diet-based predispositions to disease.

Pharmacogenetics research continues on:

Why glaucoma treatments don’t work for 20 percent of patients.
Why Coumadin, a blood thinner, causes more adverse reactions than any other drug.
Why codeine works in 93 percent of the people who take it, but not in the remaining 7 percent.
Why blacks and whites respond differently to some asthma drugs.

Instead of the trial and error approach we have to use now, genotype-based tests for Coumadin, codeine, and cholesterol-treating statins will be common within three to five years, says one researcher. Another strongly disagrees, saying it is at least 20 and probably 50 years away–an untenably pessimistic view given the accelerating developments reported in this article in particular, as well as in the article about the HapMap project* and in HFD in general.

But knowing genetic predispositions is one thing; the availability of treatments is quite another. Should people be screened for diseases if no treatment is available? Sometimes, perhaps: Genetic testing has helped reduce the incidence of a devastating and incurable disease among infant Ashkenazi Jews by more than 90 percent. Also unknown is the differential between genetic factors and environmental factors (such as junk food and smoking) in disease.

Personalized medicine not only fundamentally changes the practice of medicine from being largely reactive to largely proactive, but also opens a Pandora’s box of ethical, legal, and financial questions. Health and life insurance companies will not want to insure someone known to have a predisposition for a debilitating or deadly disease. HFD’s prime directive is to predict the arrival of radical and (for good or ill) disruptive impacts on healthcare caused by accelerating trends in technology. Policymakers, practitioners, and society as a whole need to prepare.

Reference: Auge, Karen (2003). “Genetics poised to drive medicine: DNA profiles may transform health care into prevention.” Denver Post, May 13.

*See “Population Genetics” in the Research section of this issue.

Recent findings that TV viewers are less likely to remember prescription drugs ads on television than ads for over-the-counter medicines or nutritional supplements may cast some doubt on the hypothesis that TV ads sell patients medicines they don’t need or that are no better than cheaper alternatives. Soda, food, lingerie, and beer are apparently more memorable than prescription drugs.

One reason might be the obligatory litany of vomit, diarrhea, and other side effects few people want to hear, let alone think about for very long. A related reason is that the ads have to be very careful, by law, about what they do say. To the extent that drug ads contain genuine health educational information, the finding is perhaps not to be welcomed, argues at least one doctor.

Reference: Herper, Matthew (2003). “Prescription Drug Ads Are No Blockbusters.” Forbes, May 5.

Consider:

The multiple sclerosis drug Avonexcosts an average of $14,000 for a year’s supply. Two competing drugs cost even more; athird–Copaxone–is cheaper, at $13,000 per year.
The new AIDS drug Fuzeon will cost $19,990 for a year’s supply.
Gamimune N for “bubble-boy” and other immune deficiency diseases can cost more than $30,000 a year.
Cerezyme, used to treat Gaucher disease, costs an average of $170,000 annually.

Dozens of drugs cost $10,000 or more per year. Most are biotechnology drugs, or biologics. And so far, most are for rare diseases, but not all–rheumatoid arthritis drugs costing more than $10,000 a year are on the market now,* and biotech drugs for heart disease and diabetes are in the works.

Why are they so expensive? Traditional drugs are made by combining chemicals and are easily packaged in pills or liquid medicines, but biologics involve complex bioengineering and often must be administered intravenously by professional caregivers. They require very expensive R&D and production facilities–the
Cerezyme plant, for example, cost $150 million to build, and the facility that produces the psoriasis drug Amevive cost $173 million. And in the case of rare diseases such as Gaucher’s (fewer than 10,000 sufferers worldwide), the reason for the high cost is obvious.

Biologics are in demand because they are usually more effective than alternative treatments, if those are even available; and despite the high cost they actually lower the cost of patient care overall by replacing the need for hospital stays (average cost $10,000), and for that reason health insurers generally cover them.

Some drug companies provide drugs free to uninsured patients who can’t afford them, but insured patients with 20 percent copayment clauses may have to pay thousands of dollars out of pocket annually.

Reference: Ranii, David (2003). “High cost of living well.”
News & Observer, May 13./a>

* See also…

Belize is competing with Canada in offering lower-cost drugs to U.S. citizens. A Florida entrepreneur collects orders over the Internet and ships them with a prescription to a pharmacy in Belize. The drugs are then shipped from Belize to the U.S. customer. Importing medicines into the United States is illegal, but the FDA tolerates it if the drugs for personal use.

Reference: Galewitz, Phil (2003). “Belize could be new spot for prescriptions.” Palm Beach Post, May 12.

Cases have surfaced recently in the Boston area of crude and often botched back-room cosmetic surgeries performed by unlicensed practitioners on transvestites and Cambodian immigrant women seeking Westernized eyes, fuller lips, and larger bosoms. The amateur surgeons have access to medical supplies such as silicone for breast and buttock augmentation.

Illicit surgery is nothing new, but in light of the many indications that:

medical technology grows increasingly sophisticated yet less expensive and more readily available,
demand for dermatological procedures may be outstripping the supply of licensed dermatologists,*
the trend to superhealth grows while insurance benefits recede and licensed professionals continue to command high fees,

then amateur medicine has the potential to become much more mainstream, with serious implications for public health.

Reference: Mishra, Raja (2003). “Back-room plastic surgeries raise alarm: Transvestite, immigrant victims reluctant to inform authorities.” Boston Globe, May 19, p. B1.

*See also…

A Florida company is collecting and selling stem cells from newborns’ discarded umbilical cords, with the mothers’ prior permission and under agreement with a growing number of hospitals. Cord blood stem cells are FDA-approved for use as a substitute for bone marrow stem cells in transplants for people with leukemia and immune and metabolic diseases, and have great potential against other diseases. An Atlanta physician treated 43 ALS patients with stem cell transfusions (and apparently made over a million dollars) before the FDA ordered him to stop.

Some critics are concerned about the ethics of this business, others about the lack of quality control over the collection of the cord blood; while yet others are frustrated that regulation slows progress in fighting disease.

Reference: Hundley, Kris (2003). “Mining medical waste.” St. Petersburg Times, May 12.