Category Archives: General Health

Reform Needed of Orphan Drug Act

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A recent commentary (A Sarpatwari et al NEJM 2019; 381: 106-8) details the need for reforming the Orphan Drug Act (passed in 1983).  To promote drugs for rare diseases, this act offered incentives including exclusive marketing rights for 7 years, a 50% tax credit for costs with clinical testing (reduced to 25% in 2017) and grants for clinical trials.

The problems that need to be addressed related to this act:

  • Soaring drug prices
  • “Slicing indications”

Key points:

  • “In 2017, the 100 best-selling rare-disease drugs had an estimated mean annual cost of more than $147,000 per patient, about $116,000 higher than that of the 100 best-selling drugs for other diseases.”  One of the most recent drugs for spinal muscular atrophy is priced at $2.1 million per patient.
  • 22% of these rare disease drugs have a non-rare disease indication (including Humira (adalimumab)).  This has led to concerns that manufacturers are slicing indications to secure the statutory benefits.
  • The authors argue that several of these favorable provisions need to be scaled back for blockbuster medications.

My take (borrowed from the authors): “The status quo increasingly threatens public health, as rising drug prices present growing access challenges for patients and indication slicing hampers collection of critical preapproval information on safety and efficacy when used in ways that will reflect their most common use in the market.”

Related blog posts:

Park Guell, Barcelona

Park Guell, Barcelona

“Mistrust in Science –A Threat to the Patient-Physician Relationship”

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A recent commentary, “Mistrust in Science –A Threat to the Patient-Physician Relationship” (RJ Baron, AJ Berinsky. NEJM 2019; 381: 182-5), addresses the deterioration of trust in the patient-physician relationship and potential ways to improve this.

Shortly before reading this, I read a newspaper article titled “Newtown Parents Fight Back” in Sunday’s AJC (related article online: Newton Parents Score a Win…). In this article, some of the parents of the 20 children who were killed in Newton, Connecticut have brought successful lawsuits against ‘hoaxers’ who claimed that the “rampage had been staged, with crisis actors.” Some of the parents have received death threats subsequently.

In this ‘misinformation’ age, it would be naive to expect that medicine and science would be spared. The alluded commentary makes the following points:

  • “Clinicians enter patient encounters with the reasonable presumption that they will be trusted. After all, they have powerful knowledge and good intentions…But, medicine is changing.”
  • “The legitimacy of the medical community rests on the the credibility of medical science…Physicians rely on that foundation in every interaction they have with their patients.” And on “the intimate and personal nature of each individual doctor-patient relationship”
  • “Medical practice is becoming increasingly corporate…In 2016, for the first time, less than half of practicing physicians owned their own practice…Less attention has been paid to how corporatization changes patients’ experience and …trust.”
  • “Gallup polling has revealed that confidence in almost all institutions in the United States…has deteriorated greatly…confidence in the medical system fell from 80% in 1975 to 37% in 2015.”
  • “Alternative sources of ‘authority’ have emerged to fill gap” including social media platforms, friends and relatives.

“Given the decline in trust in the institution of medicine, simply asserting medical authority or citing evidence is unlikely to win adherents…Skepticism…is a widespread phenomenon…appealing to a neutral or independent ‘referee’ of the truth…on a given subject–does not actually change minds…attempts by experts to correct misinformation may further entrench erroneous beliefs.”

Pathways to Trust

  • “Feeling recognized is a precondition for trust.”  Having to repeat stories over and over again can be ‘trust-destroying’ as the individual feels as a stranger in the health care system
  • “Explicitly acknowledging the role…of other members of the health care team may be another way…Speak positively about the staff [and colleagues]…This practice …increased patients’ trust and satisfaction.”

My take: The authors note that in this age, science s devalued and relationships are more influential.  Thus, creating trust goes back to Peabody’s 1927 admonition: “The secret of the care of the patient is in caring for the patient.”

In the newspaper article, a book publisher involved in the promoting the Sandy Hook conspiracy had to meet one of the parents as part of a lawsuit.  “At the end of the day, Gahary shook Pozner’s hand and apologized. He offered condolences for Noah’s death.”  As in medicine, getting to know each other is the surest way to garner trust.

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“Rude Surgeons May Have Worse Outcomes”

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NPR: When Surgeons Are Abrasive To Co-Workers, Patients’ Health May Suffer

An excerpt:
A study published Wednesday in JAMA Surgery challenges these ideas. The study, which looked at interactions between surgeons and their teams, found that patients of surgeons who behaved unprofessionally around their colleagues tended to have more complications after surgery. Surgeons who model unprofessional behavior can undermine the performance of their teams, the authors write, potentially threatening patients’ safety.

For the study, researchers gathered data on nearly 13,700 patients and 202 surgeons from the National Surgical Quality Improvement Program, a system designed to track and improve surgical care…

The researchers found that when surgeons had one or more reports of unprofessional behavior over the previous 36 months, their patients were 12% to 14% more likely to experience complications in a 30-day period following surgery. These complications included infections, pneumonia, stroke and kidney failure.

Full text article: Association of Coworker Reports About Unprofessional Behavior by Surgeons With Surgical Complications in Their Patients WO Cooper et al. JAMA Surg. Published online June 19, 2019. doi:10.1001/jamasurg.2019.1738

My take: It is interesting to speculate about why rude behavior may affect long-term outcomes.  My suspicion would be that team members would be more hesitant to offer advice or to call quickly if concerns arose.  Alternatively, it could be that if someone is not considerate enough to work well with their colleagues/other health professionals that they could be less attentive in their care.

Related blog post: How Rudeness Affects Performance in Medicine (and probably elsewhere)

Gun Carrying Adolescents –Why We Are Going to See More School Shootings

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A recent cross-sectional study (RMC Kagawa et al. J Pediatr 2019; 209: 198-203) shows a high rate of gun carrying among adolescents in the U.S.

Key findings:

  • Based on a sample of 10,112 adolescent who completed surveys, 2.4% of adolescents reported carrying a gun in the prior 30 days.
  • Carrying a gun was more common among adolescents with a conduct disorder (adjusted prevalence ratio 1.88), drug use disorders (APR 1.91) and patients with specific phobias (APR 1.54)
  • The authors estimate that 1.1% of adolescents with a disorder associated with self- or other-directed violence also carry guns.  This extrapolates to 272,000 adolescents with both risk factors.
  • Nearly two-thirds of adolescents who report gun carrying had a mental health disorder

My take:

  1. Guns are everywhere.  Gun carrying among adolescents, while only a small percentage of all adolescents, represent a grave risk; especially, since the majority who report carrying guns (in this study) have mental health issues.
  2. Safe storage needs to be a requirement of gun ownership.  Gun access and misuse by adolescents is a ‘clear and present danger’ (apologies to Tom Clancy).

Related blog posts:

El Retiro Park, Madrid

Good Nutrition News in Our Schools –Why Not a Press Release?

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Washington Post: Why is the USDA downplaying good news

An excerpt:

The best news was that the Healthy Eating Index (HEI-2010), a multicomponent measure of diet quality, shot up dramatically for both school-provided breakfasts and lunches.

For the 2009-2010 school year, the score for breakfast was an abysmal 49.6 out of 100 (even lower than the overall American average of 59), rising to 71.3 by the 2014-2015 school year. In that same time frame, the lunch score went from 57.9 to 81.5. The score for whole grains in school meals went from 25 to 95 percent of the maximum score, and the score for greens and beans rose from 21 to 72 percent.

In addition, there was greater participation in school meal programs at schools with the highest healthy food standards. And the study found food waste, a troubling national problem in the lunchroom, remained relatively unchanged.

The 52-page summary of study findings is chockablock with other good news, so why isn’t Agriculture Secretary Sonny Perdue crowing about it?…

In December, Perdue announced the USDA was weakening school nutrition standards for whole grain, nonfat milk and sodium, all of which had been tightened during the Obama administration. He cited food waste and nonparticipation as key rationales for the shift

 

How Does the U.S Compare to African Nations in HIV Treatment?

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A recent commentary (WM El-Sadr et al. NEJM 2019; 380; 1985-7) shows how poorly we are doing in our efforts to diagnose and treat HIV in this country and what we need to do to make progress in eliminating HIV.

Overall, the U.S. overall viral suppression rate, which is the percentage of all people with HIV in whom the virus is suppressed, is only 51%.  In contrast, the rates for Nambia, Uganda, and Zambia are 75%, 55%, and 50% respectively (U.S. measures use slightly different denominators than other countries.)

From NEJM twitter feed: AIDS in America –Back in the Headlines at Long Last

Link to Podcast: Ending the U.S. HIV Epidemic

FDA News: FDA Warning for FMT, IB-Stim Device Approval, Teduglutide Approval

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1.From John Pohl Twitter Feed:  FDA Warns of One Death Linked to Fecal Transplants (6/13/19)

An excerpt:

The consent should include, at a minimum, a statement that the use of FMT to treat C. difficile is investigational and a discussion of its potential risks.

“Two immunocompromised adults who received investigational FMT developed invasive infections caused by extended-spectrum beta-lactamase (ESBL)-producing Escherichia coli (E.coli). One of the individuals died,” the agency said Thursday…

Openbiome, a nonprofit stool bank based in Cambridge, MA, told Focus: “We are saddened to hear of the recent patient death due to an infection from a multi-drug resistant organism (MDRO) transmitted through a fecal transplant. OpenBiome material was not involved. OpenBiome screens its donors and fecal transplant material for MDROs and related risk factors, and this serious event further highlights the importance of rigorous screening and clinical oversight for all fecal transplant procedures.”

 2. FDA approval for IB-stim (a.k.a. Neuro-stim) device.

Link:: FDA permits marketing of first medical device for relief of pain associated with irritable bowel syndrome in patients 11-18 years of age

An excerpt:

IB-Stim treatment resulted in at least a 30% decrease in usual pain at the end of three weeks in 52% of treated patients compared to 30% of patients who received the placebo, and at least a 30% decrease in worst pain in 59% of treated patients compared with 26% of patients who received the placebo.

:Kovacic K1, Hainsworth K2, Sood M1, Chelimsky G1, Unteutsch R1, Nugent M3, Simpson P3, Miranda A4. 2017 Oct;2(10):727-737. doi: 10.1016/S2468-1253(17)30253-4. Epub 2017 Aug 18.

Link to abstract of relevant study:  Neurostimulation for abdominal pain-related functional gastrointestinal disorders in adolescents: a randomised, double-blind, sham-controlled trial.

3. FDA Approves Gattex (Teduglutide) for Pediatric SBS

From CenterWatch: Gattex New FDA Drug Approval

Pediatric SBS: “In a 24-week pediatric study 59 pediatric patients with SBS aged 1 year through 17 years chose whether to receive Gattex or standard of care (SOC)…Based on patient-diary data, patients who received Gattex 0.05 mg/kg/day experienced a 42% mean reduction in PS volume (mL/kg/day) from baseline (-23 mL/kg/day from baseline). At week 24, 38% of patients (10/26) were able to reduce PS infusion by at least 1 day per week. Patients reduced their PS infusion time by 3 hours per day on average compared to baseline.”

Related blog post: Teduglutide for SBS

Jeppesen PB, Pertkiewicz M, Messing B, Iyer K, Seidner DL, O’keefe SJ, Forbes A, Heinze H, Joelsson B Teduglutide reduces need for parenteral support among patients with short bowel syndrome with intestinal failure. Gastroenterology 2012 Dec;143(6):1473-1481

Jeppesen PB, Gilroy R, Pertkiewicz M, Allard JP, Messing B, O’Keefe SJ Randomised placebo-controlled trial of teduglutide in reducing parenteral nutrition and/or intravenous fluid requirements in patients with short bowel syndrome. Gut 2011 Jul;60(7):902-14.

Briefly Noted: Costs of Physician Burnout

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NPR coverage of story: What’s Doctor Burnout Costing America?

An excerpt:

The study authors calculate that for health care organizations, the cost of burnout comes out to $7,600 per physician per year. The study notes that their cost estimate is conservative, only taking into account lost work hours and physician turnover. But other research shows burned out doctors are also more likely to make medical mistakes, have less satisfied patients, and get sued for malpractice, all of which have indirect costs.

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