What price for "miracles," and who pays for them?

There are now five drugs on the market to treat genetic disorders that are each priced at over $2 million.

They have been approved by the FDA to treat illnesses that had no curative therapy. Some, such as spinal muscular atrophy, were fatal while others such as hemophilia and sickle cell anemia led to repeated crises and frequent hospitalizations.

Are these astronomical prices justified? The pharmaceutical companies justify the prices by citing two factors: the high cost of drug development and the long-term financial benefits.

The problem with using research costs as a justification for the prices is that much of the basic research is funded by NIH grants or academia rather than by industry.

In the case of Zolgensma, Novartis’ $2.1 million drug for spinal muscular atrophy, the bulk of the early funding came from a private charity founded by parents whose child had the disease. ProPublica documented how their efforts, both financial and emotional, were ignored when the commercial potential of the drug began to become apparent. They hoped for a cure for their daughter and others similarly afflicted, not a windfall for the executives of the start-up they helped fund. See: https://www.propublica.org/article/zolgensma-sma-novartis-drug-prices-gene-therapy-avexis

Research published in JAMA provided details of the sponsorship and funding of 341 trials of gene therapy. Fewer than half were industry-funded. Academic hospitals, universities and the NIH were more often the funders, and yet the financial rewards almost all go to the pharmaceutical industry.

One could make a case that the sky-high one-time cost of gene therapy pays for itself by preventing the numerous emergency room visits and hospitalizations that it will eliminate. The estimated lifetime cost of hemophilia is $20 million! This makes the one-time $3.5 million cost of Hemgenix seem a bargain.

The big if is that the only data we have is that the benefits last 3 years – hopeful, but only a short span in a life-long disease. If the benefits fade after 4-5 years will CSL Behring refund the cost?

Will health insurance companies be willing to pay for a drug that may benefit the patient for 50 years or more when they know that their average subscriber is probably going to change insurers in a few years?

We need a different way to pay for these “miracle” drugs. One option is to have them paid for on an annual basis, spreading the cost over the patient’s estimated lifespan, with payment to stop if the drug stops working. A plan must be devised soon, or scientific advances plus pharmaceutical greed will exceed society’s ability to pay.





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Osteoporosis: could I have it?

Osteoporosis, thin weak bones, is common in post-menopausal women and older men. While not fatal, it is a major risk for fractures and can severely impact your quality of life.

Estrogen in women and testosterone in men are needed to maintain healthy bones and the biggest risk factor for developing osteoporosis is aging. The precipitous drop in estrogen levels in women who go through “the change” accelerates bone loss, and some 80% of those with osteoporosis are female.

Other factors increasing your risk are smoking, alcohol consumption, poor intake of calcium and vitamin D, lack of weight-bearing exercise, taking cortisone-type drugs and being thin. (One of the few health benefits of obesity is that you are less likely to get osteoporosis.)

How do you know if you have it? Osteoporosis is a “silent disease,” with no symptoms until it is advanced, and may first be discovered when you suffer a fracture.

The best way to find it before a fracture is to have a modified X-ray called a DEXA scan, which measures the density of your bones at the hip, spine and/or wrist. This gives you two numbers, a T-score, which compares your bones to those of a young healthy adult, and a Z-score, which compares you to an average person of your age.

If your T-score is: (note that these numbers are a consensus, not “truth.”)

• –1 or higher, your bone is healthy.

• –1 to –2.5, you have osteopenia, a less severe form of low bone mineral density than osteoporosis.

• –2.5 or lower, you might have osteoporosis.

The risk of broken bones increases by 1.5 to 2 times with each 1-point drop in the T-score.

A DEXA should be done when a woman is 65, a man 75, unless they have many risk factors for osteoporosis. While there is no hard rule, it should be repeated in about 2 years. If the results are stable, you can probably wait 5 years before a third.

If you want a precise estimate of your risk of fractures, google “FRAX Score.” The first link that shows up (https://frax.shef.ac.uk/FRAX/tool.aspx?country=9) will take you to the validated tool developed at the University of Sheffield in England. In addition to your T score, it asks for information such as age, height and weight and will then tell you your risk of a major fracture in the coming decade.

How can you prevent osteoporosis? Don’t smoke; don’t drink much alcohol; do resistance exercise (weights); get adequate calcium in your diet (dairy, leafy greens, almonds) and get adequate Vitamin D. Note that dietary calcium is better than pills. We get D from sun exposure and fortified milk.

Since few adults are big milk drinkers, and most of us do not spend all day outdoors soaking up sun, a vitamin D supplement is a good idea, and the amount in a multivitamin is probably adequate for most.

If you are at high fracture risk, many different medicines are available that have been shown to reduce the likelihood of fractures – but are not a guarantee. The best-studied are the “bisphosphonates,” taken as a weekly or monthly pill, but there are many other classes of drugs highly marketed. All these remedies have the potential for serious side-effects, so the decision to start requires a careful dialog with your doctor.

There is also good evidence that taking a bisphosphonate for a couple of years and then stopping is a better idea than taking it forever.

Remember: prevention is better than treatment, so start early. The bones you preserve will be the ones to keep you upright and moving.



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Measles: do I need a booster?

It may be hard for younger people to accept this, but when I was in medical school, we talked casually of patients having had “the usual childhood diseases,” referring to measles, mumps, chicken pox and rubella (German measles).

Measles is a highly contagious disease that spreads easily to others with near 100% transmission. While generally a flu-like illness with a rash from which children recover, about 5% of measles patients develop pneumonia, 1 in 1000 develop brain swelling with deafness and/or intellectual disability resulting and 3 per 1000 die.

A vaccine to prevent measles was introduced in 1963 with dramatic results. Prior to 1963, nearly every child got measles by age 15. There were 3-4 million cases a year, with 48,000 hospitalizations and 500 deaths annually. This fell dramatically after the vaccine was available. Reported cases fell 97% between 1965 and 1968 and measles was declared “eliminated” in the U.S. in 2000.

Unfortunately, this very success has led to complacency, and the disinformation by “anti-vaxxers” has contributed to a falling off of vaccination rates.

Not surprisingly, measles has recurred. In 2024, the U.S. saw 16 outbreaks (3 or more cases) involving a total of 285 cases. As of the end of February, 2025 has seen 9 outbreaks, with a total of 164 cases. The best known is the Texas outbreak, but there have been others around the country. 95% of the cases involve people who were unvaccinated.

Do you need to worry?

If you were born before 1957, you almost certainly had measles, whether you remember this or not, and if you were born before 1963, you probably did. Natural infection gives virtually 100% life-long immunity, so there is no need to get a booster.

If you were vaccinated between 1963 and 1968, you may have received a less-effective vaccine and may want to have your antibody levels checked with a blood test.

If you received two doses of the standard MMR (measles/mumps/rubella), you are 97%+ protected unless you have an immune deficiency.

So, for most of us, protected by childhood infection or vaccination, no worries. For our children and grandchildren: GET VACCINATED. The MMR does NOT cause autism, and there have been no deaths from the vaccine in healthy people. Children with immune deficiency, a very rare condition, cannot get the vaccine, and depend on the other 99% of us preventing outbreaks by getting vaccinated.



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Influenza - NOT just a "bad cold"

Unless you are Chinese, you have probably never heard of Barbie Hsu, but in the Chinese-speaking world this Taiwanese actress is famous. That world was shocked to learn of her death from influenza while on a family vacation in Japan.

Ms. Hsu was young (48), healthy, rich and treated in a country with an excellent health care system. Why did she die?

People tend to dismiss “the flu” as just another cold, and only a minority of people around the world get their annual flu shot. As of Feb 1, about 45% of Americans have received their vaccination. For younger adults, it is well under 40%.

Influenza is much worse than the run-of-the-mill winter respiratory virus. While it is not usually fatal except in infants and frail elders, Ms. Hsu’s death reminds us that even healthy people do die from influenza.

Even if you rarely die, influenza is a very nasty illness. You have a terrible cough, ache all over, and are usually sick for a week or more. In the U.S., for the 2023-24 flu season, there were 18 million flu-related medical visits, 470,000 flu-related hospitalizations, and 28,000 flu-related deaths.

This year, there have been a larger than average number of influenza cases, office visits and hospitalizations. It looks as if this will be the worst flu season since 2009-2010.

What should you do?

Get your flu shot! While far from perfect, the immunization does reduce your chance of catching influenza by about 40% and reduces your risk of being sick enough to require hospitalization by 60%. It is not too late to get the shot.

Given the surge in cases, you should also consider wearing a mask when you are in crowded indoor environments.

If you are sick, please stay home! Sharing life’s bounties is good. Sharing your respiratory virus is not.

Good hand hygiene is also important.

Remember – the life you save could be that of your best friend or favorite aunt.



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Cannabis and health

Marijuana, now most often called cannabis, use is growing. In recent years, almost 20% of the U.S. population have used cannabis, with 4% using it daily or almost daily. What is the impact on the health of regular users?

Advocates describe better sleep, less pain and less anxiety. Unfortunately, these benefits come at a price.

Multiple studies have examined the association of regular cannabis use with symptoms and illness.

I can get the good news out of the way quickly. Regular users are less likely to report nasal congestion than non-users, unlike tobacco users who report more nasal and sinus issues.

Unfortunately, on the key major illness side, the news is not good. Regular cannabis users have more heart attacks and strokes than non-users, with even higher risk among those who use it daily or near-daily.

Regular cannabis smokers also have dramatically more (3-4 times) head and neck cancers than non-users. The highest risk was for laryngeal cancer, but higher rates of mouth and tongue cancer are also seen.

Middle-aged and older adults are increasingly using cannabis-based therapies (CBT) for symptoms, and this population suffers from many CBT-related side effects, including dry mouth, dizziness, balance problems, drowsiness and confusion.

Motor vehicle accident rates rose an average of 15% in states after recreational cannabis use was legalized, and the best evidence we have suggests that regular cannabis use is associated with a roughly 50% greater chance of having an accident and a 30% increase in fatal accidents.

Finally, there is the concern about cannabis’ effects on the developing brain. Cannabis use by mothers during pregnancy has been linked to problems with attention, memory, problem-solving skills, and behavior in their children.

Using cannabis before age 18 may affect how the brain builds connections for functions like attention, memory, and learning.

So, no, cannabis is not harmless. If you use it, do so with the knowledge that you may pay a price beyond the cost of the drug.



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What do we know about bird flu?

There are many influenza viruses. A and B are the main causes of seasonal flu outbreaks and while B only infects humans, influenza A can infect many bird and animal species.

A given influenza virus is identified by two proteins found on its surface, H and N. The common viruses causing winter influenzas are H1N1 and H3N2. The “bird flu” is H5N1.

When bird flu first appeared, it was thought to be a risk primarily to birds and was felt unlikely to infect people. Infecting birds is bad enough: the current outbreak among chickens has been a major cause of diminishing supply and increasing price of eggs. The infected flock must be slaughtered, so chicken also becomes scarcer and more expensive.

The story has gotten worse. H5N1 can clearly spread readily to dairy cattle; there have been major herd outbreaks around the U.S. It can also spread to cats, both wild and domestic and other animals.

From cattle, there has been spread to humans, so far rare and sporadic. Since 1997, there have been about 1000 proven human cases world-wide, reported from 23 countries. More than half of these have died, and this number is clearly an undercount, since testing for H5N1 is not routinely done.

In the U.S. since the start of 2024 there have been 66 proven cases of bird flu, most in dairy workers, and one death. One 13-year-old girl required life support but recovered.

So far, no human-to-human spread has been seen, but as we have learned, viruses mutate, and it would not be a shock for a mutation to appear that would allow this to happen.

Oseltamivir (“Tamiflu”) has been approved to treat H5N1 based on very little data. This drug has been available since 1999 to treat seasonal flu but is not that effective for most patients. Studies have shown that it shortens symptoms of influenza by a day. It does have benefit in very sick influenza patients, reducing the death rate by almost half.

A vaccine is available, but there are only a few million doses stockpiled and if the H5N1 mutates to spread human-to-human, the vaccine may need to be modified.

What should you do?

Avoid raw milk, which transmits H5N1 (as well as many other nasty infections!) and raw meat, and do not feed raw meat to your pets. Pasteurized milk is safe, as is properly-cooked meat.

Do not touch sick birds; call your animal control officer, who will know how to safely handle them.



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I heard about this new drug....

Among developed countries, only the U.S. and New Zealand allow direct-to-consumer advertising by pharmaceutical companies and boy, do they take advantage of this opportunity! You cannot watch television without seeing ads for pharmaceutical products.

The FDA relaxed its guidelines on radio and television advertising in 1997, and the boom took off. In 2012, spending on traditional media (mostly TV) advertising by the pharmaceutical industry was $3.2 billion and by 2024 it hit $7.5 billion.

As eyeballs turned from broadcast TV to social media, pharmaceutical advertising followed. In addition to the $7.5 billion spent on traditional broadcast media, pharma ads on social media hit $19.5 billion in 2024.

Why do they spend so much money on these ads? Because they work. People hear about new “wonder drugs” and ask their doctors about them.

Product claim ads, the majority, give the drug’s brand name (in large print) along with its generic name, the condition it treats and its benefits and risks. The benefits are up front and emphasized while the risks typically go by rapidly at the end. Cost is never mentioned.

Not surprisingly, there is no obligation for the ad to specify how the drug works or if there are competing drugs for the same condition that are safer or cheaper.

A fascinating study published in the Journal of the American Medical Association two years ago found that advertising expenses were much higher for drugs of limited benefit than for those with greater clinical benefit.

What should you do? If you think you have the condition the drug is supposed to help, it is perfectly OK for you to ask your doctor about it. They are in the best position to know if the drug might benefit you, if it is safe for you given any other medical conditions you have and medicines you are taking, whether there are better and/or cheaper drugs to use instead and if you can avoid the need for any drug by making lifestyle changes.

I must note that doctors are also heavily marketed by big pharma and not immune to the siren call of glossy ads and free lunches brought by pharma’s marketing representatives.

Remember, the purpose of any advertisement is to sell products. This is as true for pharmaceutical ads as it is for ads for cars or vacation time-shares. The primary goal of pharmaceutical advertising is not to help you but to sell more drugs, so take the claims with a large grain of salt.



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