FDA should protect the American people, and Pharma should pay!

The Food and Drug Administration (FDA) regulates drugs, and, I guess, food, although I don’t know much about  what they do in that area. It also does not approve certain drugs because they are not classified as drugs, but rather “nutritional supplements” or in new jargon “nutriceuticals”. This is odd, because such a classification does not make them either safe or effective. If they ARE effective, do the good that is claimed for them, then of course they could have other effects, which could be bad. If they are biologically active, they could be harmful. The only way they can be presumed safe is if they have no effect. Oh, well.

The FDA has been prominent for several things in recent years, most commonly regarding the approval (or not) of drugs to treat COVID, and often for demonstrating that proposed treatments, even those endorsed by high-level elected government officials, were not effective. It also made news (and this blog, FDA approves Alzheimer's drug against the recommendation of its scientific panel. Be very concerned, June 21, 2021) by its approval of the Alzheimer’s drug, Aduhelm, against the recommendation of its committee of scientific experts (eventually Medicare, the largest payer, refused to routinely pay for it, although it will in some situations).

A recent article in the New York Times, F.D.A.’s Drug Industry Fees Fuel Concerns Over Influence, discusses the controversy over “user fees” that the agency charges drug and medical device makers to help fund its work. Well, “help” may be incorrect, because such fees account for 75% of its budget.  This requires annual negotiation between the agency and the trade organizations for the industry, and those negotiations often lead to concessions to the manufacturers. At the least, it creates a situation in which it appears that the manufacturers, rather than the public, are the agency’s clients. The Times notes that ‘The user fee program traces its roots to 1992, when AIDS activists pressed the F.D.A. to hasten drug approvals. About a decade later, drugs moved through the pipeline more quickly, averaging about 10 months from roughly 19 months,’ seen at the time as a big victory for AIDS patients. Of course, it is important to note that speedier approval is only a good thing if the drugs being approved work for their intended purpose; speedier (or any) approval is not a good thing if the drugs do not work, no matter how much people with AIDS or any other disease, or their advocates, or physicians or scientists or drug manufacturers, wish they did.

With the pharmaceutical and medical device industry paying for the costs of running the FDA, which should be high enough to ensure that adequate staff and time are available for thorough review of drugs. The agency would otherwise be funded by general tax revenue, and it seems entirely just that the industry that makes money from those approvals (an INCREDIBLE amount of money; the drug industry is regularly by far the most profitable in the US) should pay for them rather than the rest of us. What is wrong is for those payers to have any influence on how the agency operates, what it does, or certainly what drugs are or are not approved. It is an insane idea to think that they should have influence because “they are paying for it” as if it were a business deal, and yet this seems to be the perspective of some influential politicians, such as Sen. Richard Burr (R-NC).  ‘Mr. Burr, a business-focused conservative, complained that the program burdens companies with negotiating with the agency over the fees, which he predicted would rise even higher.’ They should rise as high as they need to in order to fund the agency and the industry should have zero input into their policy decisions (as, indeed, the tobacco industry apparently does not over the 1200 FDA employees in its tobacco division, although the division is entirely funded by user fees).

This issue with the FDA is one (important) example of how, when industries are unsuccessful in “persuading” the government (though large cash donations) to entirely privatize a public function, they seek control of it anyway. In some cases this is a win-win for the industry and the government: the industry not only gets effective control of policy but very large influxes of money from the government to their business, and also gets to deny complete responsibility since it is a "government program”. (See, for example,  Medicare Advantage and the DCE/REACH program, "Private Equity": Profiteers in nursing homes, Medicare Advantage, DCEs, and all of healthcare, September 16, 2022.) Of course, there is a lose-lose part of the equation that involves the other two parties: the sick people who need treatments that are both effective and affordable, and the rest of us who are funding these donations to corporate coffers. Guess which group, winners or losers, has more people? Guess which gives more money to politicians?

It is tempting, when the nation’s people want something done right (like protecting them from unsafe and ineffective drugs) but do not want to pay more taxes to make it work, to enact things like “user fees”. This is certainly fairer; it is why, for example, semi-trailers pay higher highway taxes than cars --  because they travel so many more miles and are so much heavier they cause far more damage to the roads. (You used to see bumper stickers on them that announced how much, until, presumably, they realized, that the other folks driving on the highway had little sympathy and probably cheered and felt it wasn’t enough!) Thus charging the pharmaceutical companies who make so much money on drugs to pay for the FDA makes sense and is the way it should be, as long as they have no influence on the process. But that lack of influence is what irks Mr. Burr, and the drug makers who fund him.

Obviously, Burr is wrong, and so is the current process. Of the two sets of interests – the health of the American people and the profits of Big Pharma, the first should be the sole responsibility of the FDA, and the money to fund it should come from the second. Pharma will still make an exorbitant amount, no matter how much they and Sen. Burr cry that they do not have enough clout in the process to make even more, and they will continue to spend far more on marketing than on research and development.

And this should be the process for all government agencies. Fund them to protect the people from the profits of the companies that benefit.

"Private Equity": Profiteers in nursing homes, Medicare Advantage, DCEs, and all of healthcare

What happens “When private equity takes over a nursing home” is the subject of a recent New Yorker article. What happens is not pretty, at least not if you are concerned about the care of the people housed in it. Presumably it is good for the private equity investors, if all they care about is making money (probably). Compared to the days when the particular home examined in the article was run by the Catholic order of Little Sisters of the Poor, it is more crowded, more poorly staffed, dirtier, and the people living there are sicker, get less care, and are more likely to die. Good thing it is making money for the investors!

Private equity, a bland-sounding term for ‘rapacious profit-seeking capitalist investors’, is taking over a lot of things these days, in healthcare and in other areas formerly run by non-profits or government, as well as traditional businesses. Sometimes this is occasioned by circumstances largely outside “the market” or government policy, which is true (to a degree) in this case -- fewer women are becoming nuns, and the “10 sisters to a home” ratio that they aimed for became impossible to keep up. Sometimes it is “the market”, without outside interference (hard to come up with these examples). Sometimes it is a result of government policy, driven by people (largely but not only Republicans) who believe anything run by the government is bad and should be run privately, and probably anything run by a non-profit would be run better by a profit-seeking company. They are starry-eyed idealists who actually believe this despite all the evidence to the contrary. Much or most of the time it is a combination of the last two, that is market forces created by government policies that give tremendous advantage to profit-seekers, including direct subsidies of public funds and freedom from the restrictions placed on non-profits and government agencies themselves. The most common form of the latter in government, of course, is to starve the budgets, underfunding public agencies so that the public gets legitimately upset by the lack of service, and “private equity” rides in like a “white knight” to save the day.

Of course, it almost never works. That is, if “works” means “performs the ostensible functions of the company or agency better”. That they almost never do, and they also almost never save money, and almost always end up costing more – paid either the taxpayer or ratepayer or public that purchases their product. If by “works”, however, we mean “makes money for the investors”, it frequently does. This scenario plays out across many industries and formerly public functions, but it has a particularly bad smell when the “product” that is being invested in and turned over to profiteers is people, and people’s health.

People in nursing homes are very vulnerable. That is why they are in nursing homes. Even the best ones face problems with staffing, a big issue in any industry owned by profiteers since the best way to fix this is to hire more staff and pay them better and the prescription for “success” by investors is to have fewer staff and pay them less. Such problems, however, are generally even greater for those nursing homes that care for patients on Medicaid, who include the always-poor and the even larger number of “never-were-poor-before-but-are-now-after-trying-to-pay-for-long-term-care”. While the large majority of people on Medicaid are young women and their children (Medicaid, a federal/state partnership, makes it exceedingly difficult for single adults or men to receive benefits in most states), the large majority of its spending is on the aged and disabled requiring long-term care including in nursing homes. (Medicare, the federal program for aged and disabled people, does not generally pay for nursing home care, except a few days after some hospitalizations.) The Kaiser Family Foundation notes that although only 5.5% of Medicaid recipients are in nursing homes, they account for 34% of its spending.

This lesson is a key one to keep in mind: everyone does not (and should not) use an equal share of health care, or account for an equal share of healthcare costs. Sick people cost more. Sicker people cost even more. Most people are not sick or costly in any one year. The aged, disabled, and those who have multiple chronic diseases are the sickest and among the costliest, and that is not most of us. The rest of us are not in that group until we are, either from gradually entering it as we age or suddenly falling into it from an accident or developing cancer. In general, not just nursing homes, about 3% of people account for 50% of health care costs and about 50% of the people account for 5% of costs. (see my post “Red, Blue, and Purple: The Math of Healthcare Spending”, Oct 20, 2009. It hasn’t changed much.)

Nursing homes are not the only healthcare institutions being turned over to “private equity”. Medicare, the most important health care funding advance of the last century, has been especially under attack. Traditional Medicare (TM) has two components; Part A, which pays for hospitalizations, is paid for by the Medicare Trust fund (your paycheck deductions), and Part B, which pays for outpatient care, by monthly premiums paid by recipients. Part C, now known as Medicare Advantage, is a privatized version of Medicare which, if you choose it, puts you essentially in an HMO, for good and bad. The good, the “advantage” is that it usually pays for all your hospitalization (TM only pays 80% of its approved charges, requiring most TM recipients to have a “Medicare supplement” insurance plan), and gives other services, including (sometimes) hearing and vision care, and even gym memberships! The bad is that, as in an HMO, you have a limited choice of doctors and hospitals, and except in an emergency they are usually in a limited geographic area. The worse is that these plans, mostly owned by insurance companies and increasingly by private equity, are only required to spend 80% of their premiums on actually delivering health care (and they pad even this with non-healthcare expenses, including executive salaries). The worst is that they don’t want you if you are sick (and thus will cost them money); those gym memberships cost little but hospitalizations cost a lot! They have many ways of encouraging healthy seniors to sign up (“cherry picking”) including lots of TV commercials, and even more inventive ways of discouraging sick people from staying in (“lemon dropping”). The money from this and other policies that funnel your taxpayer dollars to private companies is addressed in detail by former CIGNA executive Wendell Potter in his Substack where he documents how 72% of United Health’s income is from public funds!

The newest wrinkle, particularly good for private equity (and bad for Medicare recipients) is called Direct Contracting Entities (DCEs). These were created to address the problem investors had with Medicare Advantage identified above, that is, the ‘if you choose it’ part. Because with DCE you don’t have to! The DCE contracts with your primary care physician (or usually the company or health system that employs them; the physician often doesn’t even know about it!) and then all those physician’s patients are enrolled in the DCE even though they may not know it! This allows the private equity company to collect your Medicare $$ without your permission! And they have even addressed another ‘problem’: the DCE can keep 40% of your Medicare money as profit! What a deal! (BTW, this program will be renamed REACH in 2023, so don’t be confused. And Accountable Care Organizations, ACOs, have many of the same characteristics.)

So, look at how good this is! We get the government out of providing efficient cost-effective care by both starving the agencies for funds and encouraging (by $$) private investments. It works great! Well, for the investors. For you, probably not so much. But there is also a good chance it is working for your senators and representatives who may not only be getting campaign contributions, but even have stock in these companies.

We NEED a single-payer program that covers EVERYONE, such as an improved (covers every necessary service 100%, no 20% copay). We do NOT need scams such as Medicare Advantage and DCE/REACH that move us in the wrong direction!

People, patients, polypills, primary care and POEMs: Making your health better in the real world

A recent article in the New England Journal of Medicine, ”Polypill Strategy in Secondary Cardiovascular Prevention”, also covered by the New York Times, demonstrated that people who had previous cardiovascular events, (ie., myocardial infarction -- MI, heart attack, stroke or urgent need for bypass for impending MI), had fewer recurrent heart attacks, including fatal ones, strokes, and urgent needs for bypass if they were treated with one pill a day (“polypill”) that combined their recommended medications than they did if they took multiple pills multiple times a day. The conclusion (from the Abstract) summarizes it as:

Treatment with a polypill containing aspirin, ramipril, and atorvastatin within 6 months after myocardial infarction resulted in a significantly lower risk of major adverse cardiovascular events than usual care.

This is good news, if not entirely surprising. The effectiveness of medications in preventing recurrent disease is a combination of both how effective the medication is if “taken as directed” and how difficult it is for a person (or, from a doctor-centered perspective, how “compliant” a “patient” is) to take the medication as directed. For an example of what this means, consider birth control, where “theoretical effectiveness” (how effective a method of pregnancy prevention would be if "used as directed" 100% of the time) is contrasted to “use effectiveness” (how likely a person who is ostensibly using a particular method of birth control is to get pregnant). Obviously, what matters to a person who does not want to get pregnant is the latter, and this is greatly impacted by how easy it is to not use it. Condoms and diaphragms have to be used every time and oral contraceptives have to be taken every day, which can contribute to a lower use effectiveness than forms of contraception that do not require this, such as IUDs and implants, known as LARC, long-acting reversible contraception.

So back to prevention of heart attack: Who is surprised that taking one pill a day results in better compliance than taking more pills more often? Hands? No one? Maybe cardiologists, since the alternative was “usual care”, which must have been “multiple pills multiple times a day”. Any sentient person would know, without needing to be a doctor, scientist, statistician, or epidemiologist, that taking one pill a day is not only easier but much more likely to happen than taking multiple pills, and especially taking pills multiple times. Remember, the medication only works if you take it, and the harder you make it to take, the less likely people will and the less likely it is to work. Even drug companies know this; you probably have noticed that when a drug is about to lose its patent one of the first “new drugs” that the company comes out with is a long-acting version of it that you have to take only once a day! I cannot prove that they would have been able to release this in the first place but instead held it in reserve just for this reason, but I would not be surprised. One pill once a day is also likely to be cheaper (except while under patent) and this is a big issue for people who have difficulty affording their medications (ie., most of us, but especially those with lower incomes. In the US, of course).

Though it seems obvious that one pill a day is more likely to be taken, this research study is not without importance. For starters, it showed that it worked to take one pill combining 3 drugs once a day. After all, if it didn’t help prevent disease, it wouldn’t be desirable. That it worked better than “usual care” is almost certainly related to it being one pill once a day, and how difficult it is to remember to take pills more often. [Even those of you who do not have chronic disease: have you ever been given antibiotics to take 4 times a day for 10 days? How often have you actually taken all 40 within the 10 days, even after you’ve started feeling better sooner?] We may presume that taking the multiple pills multiple times a day would have worked as well if people had done it, but because they don’t, it didn’t work as well. The contribution of the study is to show that the one pill once a day works, and because it is one pill once a day, works better.

Another important thing that the study did  is to look at meaningful outcomes: death, repeat heart attack or stroke, need for bypass. This also seems like a “duh”, if you are a regular person, but it has been common in the literature to not measure these but often “intervening variables” such as changes in cholesterol level (or blood sugar, or whatever you are studying) because this is easier to do and requires less follow-up. But having lower cholesterol, for example, only matters to the extent that you are less likely to have a heart attack or stroke!

When this study first came out, I commented on FB and Twitter that

‘Family physicians and other primary care clinicians know this better than subspecialists, since they are focused on "what works best for the disease?" and we on "what is going to work for this person?"!’

Of course, this is, probably, not completely true for all family physicians and all subspecialists, but it is certainly true that it is more likely for primary care clinicians to think about and be aware of how a treatment affects the whole person. Subspecialists are usually concerned with treating one condition, “their” disease”, and at how the treatment they prescribe ameliorates that, while primary care clinicians are looking at how it affects the person’s life. One simple example is drug interactions and “side effects” (which are actually just effects, but not the effects we want). Primary care clinicians care for a person with all the conditions that they have and have to not only see how, for example, their heart disease medicine works for the heart disease but if it is bad for another disease they have, or if its side effects (or difficult regimen, multiple pills multiple times a day) means that they don’t take it.

In research, family physicians and other primary care clinicians have looked for Patient-Oriented Evidence (POE) as opposed to Disease-Oriented Evidence (DOE). DOE looks at whether a treatment, usually in an experimental setting (which often has many differences from real life, such as free medicine and people to remind you to take it!) makes a disease better, while POE looks at whether it makes the person’s life, as a whole, better. This is important, especially if you are a person. (Or probably if you are an animal!) Indeed, family physicians have taken this a step farther to Patient-Oriented Evidence that Matters (POEMs), with sections reviewing recent research that does featured in several family medicine journals. POEMs is, in addition to being a cute acronym, has meaning; not all evidence, disease or patient oriented, actually matters. For example, the study cited above: showing that using a polypill decreases your risk of cardiovascular events and improves your life matters, while simply showing a change in a lab value might well not.

It is really good that there are treatments for diseases, whether common (like heart disease) or rare (like, ironically, I just discovered a rare blood condition also called POEMS!) that can make you better. It is also really good that there are subspecialists who know about them and can make recommendations for treatment (especially for the rarer ones) and who keep up on the literature. But it is also important that there is someone keeping an eye on the person, the patient, with all their diseases and medicines and treatment regimens and side effects, and, oh yeah, the stressors of their everyday life with money, and family, and work (or not having work) and how in heck, in this country, they are going to pay for their treatment. These are the real components of real life, sometimes called the “social determinants of health”, that are poorly addressed by US healthcare.

Polypills are good, as are POEMs. And so are primary care clinicians, especially when their employers allow them sufficient time and encouragement to actually provide comprehensive care for their patients.