Tackling High Drug Costs -Lessons from Australia and Brazil

In two related commentaries referenced below, the authors detail how Australia and Brazil managed to provide a blockbuster hepatitis C virus (HCV) medication without following the going-broke example of Blockbuster video stores.

  • Australia: S Moon et al. NEJM 2019; 380: 607-9
  • Brazil: EM da Fonseca et al. NEJM 2019; 605-6.

Australia provided a lump-sum payment of approximately 770 million dollars (in U.S.) over 5 years in exchange for an unlimited volume of direct-acting antivirals (DAAs). As a result of this approach, Australia managed to treat many more patients at a much lower cost.  “The government would have to spend …U.S. $4.92 billion more to treat the same number or it could treat 93,000 fewer patients with a fixed budget” of approximately U.S. $766 million.

With the Australian approach, the authors note that it is analogous to a patent buyout and works if the ongoing drug manufacturing cost is low and the manufacturer is able to meet growing volume demand.

Brazil’s approaches for DAAs relied on either threatening loss of patent protections and/or enabling local generic production of sofosbuvir.  This resulted in ~90% price discount. Patent protection in Brazil is granted only if a medication is approved by both INPI (Instituto Nacional da Propriedade Industrial) and ANVISA (Brazilian Health Regulatory Agency).

My take: Given the rising costs of medicines, examining how other countries surmount these financial barriers is important.  In my view, the often arbitrary and exorbitant pricing by pharmaceutical companies will erode the support of protective policies in the U.S. which thus far has helped produce many advances.

Skull Rock, Joshua Tree National Park

 

Telemedicine -Still Not Popular

Several recent reports indicate that telemedicine is being adopted at a slow pace  –thanks to Ben Gold for these references.

AP: Telemedecine’s Challenge: Getting Patients to Click the App

An excerpt:

Widespread smartphone use, looser regulations and employer enthusiasm are helping to expand access to telemedicine, where patients interact with doctors and nurses from afar, often through a secure video connection…

Doctors have used telemedicine for years to monitor patients or reach those in remote locations. Now more employers are encouraging people covered under their health plans to seek care virtually for several reasons…

Telemedicine can reduce time spent away from the job, and it also can cost half the price of a doctor’s visit, which might top $100 for someone with a high-deductible plan…

Research firm IHS Markit estimates that telemedicine visits in the U.S. will soar from 23 million in 2017 to 105 million by 2022. But even then, they will probably amount to only about one out of every 10 doctor visits.

My take: Telemedicine can overcome geographical barriers. However, I worry about the person-to-person connection as this is hard even with face-to-face visits.

An unrelated article using telemedicine: IBD News Today: Remote Monitoring Offers Little Benefit to IBD Patient, Study Finds In this randomized study with 348 adult patients wtih IBD, telemedicine (in addition to clinic visits) did not improve patient confidence or management more than the control populaiton. 

Joshua Tree National Park

 

Big Pharma Neglecting ‘Required’ Pediatric Studies

A recent retrospective study (TJ Hwang et al. JAMA Pediatr 2019; 173: 68-74) examined the completion rate of FDA-required pediatric studies. Thanks to Ben Gold for this reference.

Background: In 2003, the Pediatric Research Equity Act (PREA) was signed into law and authorized the FDA to require clinical stuides to assess the safety and efficacy of new drugs and drugs with new indications in pediatric subpopulations.  However, the FDA cannot withdraw approval for a drug if a manufacturer fails to comply with PREA.  In addition, the authors note that “to our knowledge, to date, no financial penalties or enforcement proceedings have been brought against manufacturers fo noncompliance…and only 31 noncompliance letters have been issued.”

Key findings:

  • Between 2007-2014, there were 438 new drugs and/or new indications.  114 were subject to PREA. 84 were new drugs and 30 were new indications.
  • 222 studies required pediatric postmarketing clinical studies (in these 114 drugs). Only 75 (33.8%) were completed; rates were lower for efficacy studies (38 of 132 –28.8%) compared to pharmacokinetic studies (19 of 34 –55.9%).
  • As a result of the PREA-mandated studies, there was an increase in some pediatric information of drug labels in 41.2% after a median follow-up of 6.8 years, compared with 15.8% at time of approval of these 114 drugs.

The authors note that PREA is responsible for “nearly 80% of pediatric drug studies completed for FDA.” Congress also passed the Best Pharmaceuticals for Children Act which provides a financial incentive to companies if they perform certain pediatric studies.

My take: Pharmaceutical companies, for a multitude of reasons, are not completing requied pediatric studies.

The Health Consequences of Climate Change

A recent review article, “The Imperative for Climate Action to Protect Health,” (A Haines, K Ebi. NEJM 2019; 380: 263-73, and commentary 209-11) explains why many brilliant minds are so concerned about our climate.

What is happening to our climate:

  • “Climate change is already adversely affecting human health…if no additional actions are taken, then over the coming decades, substantial increases in morbidity and mortality are expected.”
  • “August 2018 was the 406th straight month during which global mean temperatures were above the long-term mean.”
  • “Carbon dioxide (the primary greenhouse gas) have risen from approximately 280 ppm in preindustrial times to approximately 410 ppm today. Carbon dioxide remains in the atmosphere for centuries, with about 20% persisting for more than 1000 years.”
  • “The global mean temperature is currently increasing at a rate of 0.2 degrees C per decade owing to past and continuing emissions.”

Health Consequenes:

  • Major climate events including heat waves, floods, rising sea levels, droughts and storms with their immediate and long-term effects of health: heat-related illnesses, fatalities, injuries, and mental health effects
  • Worsened air quality: asthma and COPD exaccerbations, worsened cardiovascular outcomes
  • Water-borne illnesses due to effects on water quality: cholera, campylobacter infection, algae blooms, cryptosporidium, leptospirosis are some examples
  • Disruption of food supply and safety –heat can interfere with soil moisture and crop yield: malnutrition
  • Proliferation of vector-related illnesses: zika virus, dengue, lyme disease, malaria to name a few
  • Social: flooding and heat are likely to lead to social upheaval, mass migration, and violent conflicts

Even if all of the goals of the Paris Agreement were honored by all of the signatories, “it would not be sufficient to limit warming to 2 degree C above preindustrial levels…[it] would be expected to result in a temperature increase of approximately 3.2 degree C by the year 2100, relative to the preindustrial period.”

The authors cite estimates that mitigating adverse climate effects could prevent more than 175,000 premature deaths and 22,000 fewer deaths annually by 2030.

The associated commentary link the recent destructive California wildfires to climate change.  They note that “tackling this challenge may feel overwhelming.” Working on this can include both individual lifestyle actions and institutional efforts.

Individual actions:

  • Walking/cycling more
  • Eating less meat
  • Reducing food waste
  • Conserving energy

Institutional actions:

  • Health care system accounts for 1/10th of greenhouse gas emissions and health systems need to work on cutting their emissions
  • Health care institutions can invest/divest in industries who are helping and harming efforts to limit fossil fuel consumption

Resources:

From review article by Haines and Ebi:  “Health professionals have leading roles to play in addressing climate change. They can support health systems in developing effective adaptation to reduce the health risks of climate change, promote healthy behaviors and policies with low environmental impact.”

My take: Interestingly, the issue of delay in addressing problems is discussed in an unrelated commentary (NEJM 2019; 380: 118-9) related to denial.  For the author, she was attempting to deny the possibility that her father had ALS.  At the end, she quotes Elisabeth Kubler-Ross: “Denial helps us to pace our feelings of grief. There is a grace in denial.  It is nature’s way of letting in only as much as we can handle.”  Clearly, much of the world remains in denial of the necessitiy to address climate change.

Related blog post:

Badwater Basin, Death Valley -salt-covered dried up basin

How Safe is Marijuana?

A recent link to Malcolm Gladwell’s article in the New Yorker: Is Marijuana as Safe as We Think? One of my sons informed me of this article.

Excerpt from Malcolm Gladwell’s analysis:

A few years ago, the National Academy of Medicine convened a panel of sixteen leading medical experts to analyze the scientific literature on cannabis. The report they prepared, which came out in January of 2017, runs to four hundred and sixty-eight pages. It contains no bombshells or surprises, which perhaps explains why it went largely unnoticed. It simply stated, over and over again, that a drug North Americans have become enthusiastic about remains a mystery.

For example, smoking pot is widely supposed to diminish the nausea associated with chemotherapy. But, the panel pointed out, “there are no good-quality randomized trials investigating this option.” We have evidence for marijuana as a treatment for pain, but “very little is known about the efficacy, dose, routes of administration, or side effects of commonly used and commercially available cannabis products in the United States.” The caveats continue. Is it good for epilepsy? “Insufficient evidence.” Tourette’s syndrome? Limited evidence. A.L.S., Huntington’s, and Parkinson’s? Insufficient evidence. Irritable-bowel syndrome? Insufficient evidence. Dementia and glaucoma? Probably not. Anxiety? Maybe. Depression? Probably not.

Then come Chapters 5 through 13, the heart of the report, which concern marijuana’s potential risks. The haze of uncertainty continues. Does the use of cannabis increase the likelihood of fatal car accidents? Yes. By how much? Unclear. Does it affect motivation and cognition? Hard to say, but probably. Does it affect employment prospects? Probably. Will it impair academic achievement? Limited evidence. This goes on for pages…

Several points discussed in article:

  • Marijuana may increase the risk of psychiatric illnesses. “Many people with serious psychiatric illness smoke lots of pot. The marijuana lobby typically responds to this fact by saying that pot-smoking is a response to mental illness, not the cause of it—that people with psychiatric issues use marijuana to self-medicate. That is only partly true. In some cases, heavy cannabis use does seem to cause mental illness”…
  • Marijuana may increase aggression,  In the state of Washington was the first U.S. jurisdiction to legalize recreational marijuana. “Between 2013 and 2017, the state’s murder and aggravated-assault rates rose forty per cent—twice the national homicide increase and four times the national aggravated-assault increase”
  • Does cannabis serve as a gateway drug?  Like e-cigarettes, cannabis is being formulated into products attractive to youth: gummy bears, bites, and brownies.

My take (borrowed in part from author): “Permitting pot is one thing; promoting its use is another.” We really don’t know that much about marijuana.

CDC Link: Marijuana and Public Health

Related blog posts:

 

NPR: How to Help Kid Overcome Fear of Doctors and Shots

From NPR: How to Quell A Kid’s Fear of Doctors and Shots

An excerpt:

Sasha Albani, a child and adolescent psychotherapist… suggests parents calm themselves and find age-appropriate ways to help children face their medical fears instead of fleeing them.

For very young kids, who have a hard time putting words to thoughts and emotions, imaginary play with mom or dad before the appointment can help, Albani says.

“Use a toy doctor kit to explain what will happen at the appointment and to discuss your child’s specific worries,” she advises…

Children under age 6 may benefit from the book, “Daniel Visits the Doctor” by Becky Friedman.

Kids with needle phobias may be helped by reading, “Lions Aren’t Scared of Shots: A Story for Children About Visiting the Doctor,” by Howard S. Bennett. And the book “Imagine a Rainbow: A Child’s Guide for Soothing Pain,” by Brenda S. Miles, may be useful for older kids between the ages of 8 and 10.

Playing The Coping Skills Board Game can bolster the confidence of preteens… And smartphone apps like “Stop, Breathe & Think Kids” can be a fun way to learn mindful breathing techniques and other relaxation tips that help turn down the alarm of worrisome feelings.