Sunday, October 30, 2022

Should the FDA Approve More Drugs?

Life can be vexing.   Life is not a math problem that has one indisputable correct answer.   We are constantly weighing options as we make decisions.  How much risk would we tolerate in order to hope to capture a reward?  Does an NBA star go for the three pointer or drive to the rim?  Does a defense attorney put his client on the stand or leave him mute hoping that the prosecution hasn’t met the required burden of proof?   Does a surgeon recommend an operation today or should the patient wait another 24 hours to see if his condition improves without surgery?

All of us struggle where to draw the line.   Look at the ongoing debates in the public square regarding national security.   While some government officials deny this, most of us acknowledge that there is a tension between guarding our civil liberties and protecting our security.  Civil libertarians claim that we can do both, but I believe that trimming civil liberties would provide our intelligence community with more tools to protect us.  I am not advocating this, but simply acknowledging what I believe to be a truth.  There will never be consensus on where to establish this boundary. 

Weighing the Options

The Food and Drug Administration (FDA) also struggles with an analogous issue.  They must balance the public’s need for new medicines while protecting us from unsafe medicines.   How much testing should a pharmaceutical company have to pursue to satisfy the FDA of the drug’s safety and efficacy?  This query is not easily answered.  More testing would likely enhance the safety of drugs that the FDA approves for our use, but would result in fewer medicines becoming available to us.  Would the public want medicines released sooner that show real promise for conditions such as Alzheimer’s disease, cancer, depression and autism?  Would the public tolerate greater risk of unknown side effects of drugs that are released on an expedited track?  What would these companies’ legal exposure be here?  Conversely, should the FDA’s pathway toward drug approval be lengthened in order to increase the margin of safety?

Desperate patients and their families may demand drugs that have minimal safety and efficacy data.  We all understand this.  But short circuiting the process means that there won't be high quality clinical trials, often with a placebo arm, to vet the drugs properly.  This does not serve the public at large as well as ill patients who deserve effective and safe drugs.

These are tough calls to make.  When the FDA deviates from established protocols, the results can be disastrous,  as occurred with their botched accelerated approval of Aduhelm in 2021 for Alzheimer's disease.  

So, do you want more drugs or more safety?  

Sunday, October 23, 2022

Can Pepto Bismol Relieve Indigestion and Upset Stomach?

Pepto Bismol remains one of the most popular over-the-counter medicines that my patients swallow.

They take it for all kinds of digestive distress.  Does it work?  Hard to say.  The elixir does have anti-bacterial and anti-diarrheal properties, but I suspect that there is a potent placebo effect at play also.  I personally think that these sales are largely the result of decades of brilliant marketing by the company.  We can all remember their television commercials in the days of yore when we would watch the pink liquid oozing down the esophagus and then gently coating the stomach creating a blanket of healing and protection.  What a graphic!  Many patients have internalized this marketing believing that this medicine is the fire extinguisher that can quiet their internal flames.  



Right out of the doctor's 'black bag'.

Patients are generally unaware of two important properties of this product. First, Pepto Bismol can turn the stool black.  Three or four times a year I receive frantic calls from patients with black stools who are scared that they are experiencing internal bleeding.  At times, a medical colleague refers such a patient to me wondering if a bleeding ulcer might be present, when the culprit is actually the pink drink. 

Secondly, Pepto Bismol is actually an aspirin product, which is not a medicine that most gastroenterologists recommend for stomach distress.  I have not yet met a single Pepto Bismol consumer who was aware of the medicine’s aspirin component.  Following this same therapeutic strategy, many of my patients drink apple cider vinegar – an acid – to combat heartburn which is caused by acid.  And many of them swear it delivers relief!

Healing is complex and extends beyond the boundaries of science.  We have all experienced this phenomenon and have witnessed it in others.  We simply cannot understand every pathway that leads to relief.  Those of us who are in the healing business need a daily tincture of open-mindedness and humility if we are to heal others.  This shouldn’t be too much for doctors to swallow.

Sunday, October 16, 2022

New Study Questions Screening Colonoscopy - Doctors Push Back

Colonoscopy became ‘breaking news’ about a week ago.  The preeminent medical journal, The New England Journal of Medicine (NEJM) published a randomized trial assessing the effectiveness of screening colonoscopy in reducing the risk of contracting colon cancer and dying from it.  The results were lackluster.

While there is accumulated evidence that colonoscopy can reduce colon cancer risk, the bulk of this data has not been the results of randomized controlled trials, the gold standard in medical research.  In the NEJM study, there was a group who was offered a screening colonoscopy and a separate control group who was not.

Gastroenterologists, along with the medical community at large, have been preaching the lifesaving benefits of screening colonoscopy for decades.  The simple strategy is to remove ‘pre-cancerous’ polyps that are lurking silently in the colon and to remove them before they have an opportunity to transform into cancer.  What makes colonoscopy such an attractive screening tool is that is can destroy a precursor to colon cancer – a benign polyp – which can remain dormant for years.  Other cancers, such as lung and pancreatic cancers, do not have ‘pre-cancerous’ lesions like colon polyps that can be easily screened for and removed.  This is one reason why these cancers often announce themselves after they have already spread. 

In the NEJM study, the group who was offered screening colonoscopy had an 18% reduction in developing colon cancer, a statistic much lower than prevailing opinion. Let me illustrate how modest an 18% reduction truly is. If your lifetime risk of developing colon cancer is 4%, then an 18% reduction lowers your risk to 3.28%.  In other words, you won’t enjoy much benefit with this risk reduction even though an 18% decrease may sound substantial.  In the NEJM study, it was calculated that for every 455 patients in the colonoscopy arm of the trial, 1 case of colon cancer would be prevented.  I’ll let readers decide if this statistic represents a game changer. 



Colon cancer under the microscope.
Is colonoscopy the weapon we thought it was?

As expected, professional gastroenterology societies argued that the study was flawed for several reasons and argued that screening colonoscopy does protect patients from colon cancer.  I felt that their criticisms of the study were legitimate.  However, I wondered if they would have voiced these same concerns if the study concluded that colonoscopy was highly effective.  

As one who has read many of these studies over the years, I don’t think we truly know to what extent colonoscopy reduces risk and mortality.  Shouldn’t we know this when designing colon cancer prevention strategies for the public?  Colon cancer screening is difficult to study as it may take a decade or longer to demonstrate a benefit when the rate of colon cancer development is very low and the disease is relatively uncommon.  It’s much easier for investigators to show that an intervention is effective when a disease is more common.

I have done more than 50,000 colonoscopies thus far in my career.  Have I saved lives?  I certainly hope so, but perhaps less so than I had thought.  The vast majority of my colonoscopies have been normal or have discovered small polyps, which I removed.   Gastroenterologists have no method to determine which polyp may be at risk of malignant transformation.  That is why scour the colon and remove any and all polyps encountered.  But, most of them will remain benign and innocent.  We are using a wide net.

How much risk reduction would justify screening every adult starting at age 45, which is the new recommended age to begin screening.?  I doubt 18% would be the holy grail.  Even doubling this to 36% risk reduction seems modest to me.  I’ll bet if you asked most doctors, including gastroenterologists, how effective colonoscopy is, you would likely hear much more optimistic responses than is justified by existing data, including this latest study.  Same issue with mammography.  The actual benefit that women enjoy from this test is much less than most of them, and perhaps their doctors, believe.

I still recommend screening colonoscopies to my patients, but we will have to see how all of this shakes out.  And the issue will be moot as new technologies such as stool or blood testing will soon render the colonoscope obsolete. 

Sunday, October 9, 2022

Why Do I Have Abdominal Pain?

I am a gastroenterologist who has been practicing for decades.  One would think that with my diagnostic cunning and length of service that I would be able to identify the cause of your stomach distress from across the room.  Alas, abdominal distress is often more cunning than the medical sleuths who aim to unmask its identity.  A reality of gastroenterology is that abdominal pain – an issue I confront every day  – is often unexplained and unexplainable, a frustrating reality for patients.

Here’s another frustrating aspect of the experience that patients must often deal with.

A patient with months or years of abdominal distress is seen in an emergency room.  In some instances, there have been more than one ER visit for the same issue.  Despite repeated laboratory data, a CAT scan or two, other imaging studies of the abdomen, a thorough review of the patient’s history and physical examinations which may be repeated over the course of hours in an ER, no diagnosis is made.   The patient is advised by the ER doc to consult with a gastroenterologist for an office consultation to unravel the conundrum.

The patient then sees a specialist like myself – the ‘expert’- with the expectation that I will untie the Gordian knot and bring clarity to chaos with a wave of my hand.  Indeed, it’s very natural for a patient to feel that seeing the digestive specialist will be the key to unlocking the mystery of his abdominal distress.  And there are many times when I see patients referred to me from area emergency rooms that I sense a new diagnostic angle to pursue or recommend medication modifications. But not always.



Is the Human Brain a Diagnostic Tool?

Even though I have special training and experience in digestive ailments, I face some handicaps that don’t exist in the ER.  I'll explain.  Which scenario would seem more likely to explain a patient’s abdominal pain?  An ER visit with state-of-the-art technology with a team of medical professionals who can assess a patient over several hours or a half hour visit with a gastroenterologist who is equipped with knowledge and experience?   In other words, if an ER or two can’t figure it out, then we gastro specialists might be mystified also.

When I explain all of this to the patients I see sent from ER’s, it makes perfect sense to them.  I still try to do my best for them.  And, I may have an advantage over the ER.  Since I have no available technology in my office to distract me, I have time to really think over the case.  There is only the patient and me sitting in a quiet room as I meditate on the case.  Imagine that, a physician’s brain being a diagnostic tool!

 

 

 

Sunday, October 2, 2022

Why Isn't My Drug Covered by my Insurance Company?

Over recent weeks, several times I have prescribed medications for patient that they could not afford.

Insurance plans do not cover every benefit.  With respect to drug coverage, each insurance plans has a formulary - a listing of drugs that are covered.    As patients have learned well, covered medicines are categorized into different tiers, which determines to what extent the medication will be covered  The lower the tier number, the more money that the patient will have to surrender.  Some drugs are simply not on the formulary and can have eye-popping costs which might approach a patient’s monthly mortgage payment.


Distraught woman hoping for a win so she can afford her colitis medicine.


The two medicines that I had prescribed which were then stiff-armed for coverage were for colitis.  I had the patients research the costs and they and I were shocked by their findings.  At first, I thought they may have misplaced the decimal point, but the more expensive of the two was priced at $2,000 for a prescription.

Sparing my readers the medical details, both of these medicines are considered mainstream colitis medicines.  They have been approved for this use by the Food and Drug Administration (FDA) years ago.  And importantly, neither has an equivalent alternative.  For example, if I prescribe a heartburn medicine and discover it is ‘off formulary’, there will (hopefully) be equivalent alternatives available for the patient.  Not so with my recent colitis patients.

How would it feel to be sick, have medical insurance and not be able to afford the medicine that would make you well?

For luckier colitis patients whose medical plans cover these drugs, the costs are extremely low.  What this means to me is that the system is unfair and broken.

Leaving aside contracts and formularies and the overall labyrinthine insurance companies, shouldn’t a patient who has insurance and who’s played by all the rules be covered for an FDA approved medicine that his doctor recommends?