Legislative Agenda for Drug Pricing

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There have been some recent terrific advances in pharmacology –a few that come to mind:

The one common feature is that these are all very expensive; there are many other expensive medications with less benefit.  Given the rise in costs of these medications, there is a need to do a better job in getting good value in our drug costs.  A potential path forward is outlined in a recent commentaries (SB Dusetzina, J Oberlander. NEJM 2019; 381: 2081-4; PB Bach. NEJM 2019; 381: 2084-6).

In the first commentary, the authors review the Elijah E Cummings Lower Drug Costs Now Act of 2019 (HR 3).

  • In essence, this act establishes a drug-price negotiation process and limits price increases on existing products.  “Companies whose products are selected for ‘negotiation’ will in reality face price regulation and a severe penalty for noncompliance.”
  • The act would examine U.S. prices compared to prices paid in other countries.  “There would also be a legislatively set maximum price that could not exceed 120% of the average net price paid for the same drug in designated countries.”
  • The bill also would cap Medicare Part D out-of-pocket spending at $2000 per year.

In the second commentary, Dr. Bach notes that drugs that have too little evidence to support full approval and those that are ‘too late in their life cycle’ both should have their pricing negotiated by the government.  This would side step some of the arguments about undermining the incentive for new drug development.

“Too little”

  • The FDA grants approval of some drugs on the market conditionally on the basis of data indicating that they improve a surrogate marker of patient benefit. “Despite the conditional nature of the approval, …the pharmaceutical firms currently charge the same high prices that fully approved drugs capture.”
  • Required studies frequently show that these conditionally-approved medications are ineffective.  Of the 198 indications granted accelerated approval since 1992, only 115 have garnered full approval.  Also, conditional approval may result in less incentive to complete the needed trials in a timely fashion.

“Too late”

  • In this category, the author notes that some medications have found many ways to extend their monopolies, which are intended as a time-limited reward for the effort of developing a new medication.  These include overlapping patents, refusing to provide samples to competitors, and paying other companies to delay bringing generic or biosimilar products to market
  • Most of the potential for savings are in this category rather than the ‘too little’ category
  • Negotiating prices of the top 10 too little and 10 too late medications with reference to 120% of UK pricing would have provided about nearly 27 billion in savings in 2019

My take: While current partisanship makes reaching agreement difficult, targeting soaring pharmaceutical costs is one area in which I predict common ground can be found.  While many are going to benefit from the therapeutic advances listed above, there are other medications which are overpriced and should be negotiated like in other high-income countries.

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Bathroom mural for bicycle enthusiasts (at a stop on the Petit Train du Nord Linear Park)

The Best Information We Have To Date on the Emerging Coronavirus

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The NEJM has made the information it has on the emerging coronavirus open access.  Here are the links:

An excerpt from the editorial:

For the third time in as many decades, a zoonotic coronavirus has crossed species to infect human populations. This virus, provisionally called 2019-nCoV, was first identified in Wuhan, China, in persons exposed to a seafood or wet market. The rapid response of the Chinese public health, clinical, and scientific communities facilitated recognition of the clinical disease and initial understanding of the epidemiology of the infection. First reports indicated that human-to-human transmission was limited or nonexistent, but we now know that such transmission occurs, although to what extent remains unknown. Like outbreaks caused by two other pathogenic human respiratory coronaviruses (severe acute respiratory syndrome coronavirus [SARS-CoV] and Middle East respiratory syndrome coronavirus [MERS-CoV]), 2019-nCoV causes respiratory disease that is often severe.1 As of January 24, 2020, there were more than 800 reported cases, with a mortality rate of 3%…

Both SARS-CoV and MERS-CoV infect intrapulmonary epithelial cells more than cells of the upper airways.4,6 Consequently, transmission occurs primarily from patients with recognized illness and not from patients with mild, nonspecific signs. It appears that 2019-nCoV uses the same cellular receptor as SARS-CoV (human angiotensin-converting enzyme 2 [hACE2]),3 so transmission is expected only after signs of lower respiratory tract disease develop…

It is likely that 2019-nCoV will behave more like SARS-CoV and further adapt to the human host, with enhanced binding to hACE2.

 

Neurologic Toll of Celiac Disease

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A recent prospective cohort study (M Hadjivassiliou et al. Clin Gastroenterol Hepatol 2019; 17: 2678-86) shows an alarmingly-high level of neurologic deficits in 100 consecutive adults (mean age 43 years) with a new diagnosis of celiac disease.

Key findings:

  • Gait instability in 24%
  • Persistent sensory symptoms in 12%; peripheral neuropathy was identified in 2%
  • Frequent headaches in 42%
  • Abnormal results from Brain MRI in 60%; 25% had brain white matter lesions beyond expectation for age group and 46% had abnormal MR spectroscopy of the cerebellum
  • Anti-TG6 antibodies were detected in 40% of patients and this subgroup had significant atrophy of subcortical brain regions compared to patients who were Anti-TG6 antibody-negative

Some neurologic findings improve on a gluten-free diet (GFD).  In previous studies of patients with CD and headaches, 75-80% improved or subsided after a year of strict adherence to a GFD.

My take: This study indicates that early diagnosis of celiac disease along with strict adherence to a gluten-free diet is likely to prevent permanent neurologic disability.

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“Original Sin” and U.S. Health Care

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Several recent articles regarding reforming our current healthcare system have been published in Annals of Internal Medicine (Jan 2020) and are open access.  Highlighted text in images below by Eric Topol, MD.

Link: Envisioning a Better U.S. Health Care System for All: Health Care Delivery and Payment System Reforms

In this position paper, the American College of Physicians (ACP) proposes strategies to address social determinants of health and reduce barriers to care in order to achieve ACP’s vision for a better U.S. health care system for all. The ACP’s vision, outlined in an accompanying call to action (1), includes 10 vision statements, 4 of which are particularly relevant to the policies discussed in this paper (Figure). The companion position papers address improving payment and delivery systems (2) and coverage and cost of care (3). Together, these papers provide a policy framework to achieve ACP’s vision for a better U.S. health care system.

Link: Envisioning a Better U.S. Health Care System for All: Coverage and Cost of Care

Link: The American College of Physician’s Endorsement of Single-Payor Reform

An excerpt:

Public choice’s second purported advantage may also be illusory. Although surveys indicate that voters value choice, it’s choice of doctor and hospital—not insurer—that they care about.
Although no reform achieves perfection, evidence indicates that a well-structured single-payer reform might resolve our nation’s coverage and affordability problems, preserve the choices patients value, and allow doctors to focus on what matters most: caring for our patients.

Link: “Original Sin” and U.S. Health Care

An excerpt:

This series of articles describes a vision and makes important recommendations to improve coverage and control costs; reform health care delivery and payment to promote person-centered; high-value primary care; and address social and environmental determinants of health…

Seen through the lens of the American College of Physicians’ recommendations, how might addressing an original sin of failure to directly finance universal coverage in the United States facilitate progress on other recommendations?…

Implementation of the American College of Physicians’ recommendations, with an emphasis on promoting transparent, direct financing of universal access, holds great promise for replacing the current system of opaque and distorting subsidies with one that better serves all Americans.

Link: A New Vision for Quality and Equity

 

Link: Health is More Than Health Care

Link: Envisioning a Better U.S. Health Care System for All: A Call to Action by the American College of Physicians

Link:  The U.S. Health Care System Is Ill and Needs a Bold New Prescription

Alcohol -More Deadly Than Opioids

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NPR: U.S. Alcohol-Related Deaths Have Doubled, Study Says

An excerpt:

Death certificates spanning 2017 indicate nearly 73,000 people died in the U.S because of liver disease and other alcohol-related illnesses. That is up from just under 36,000 deaths in 1999…

Overall, researchers found men died at a higher rate than women. But when analyzing annual increases in deaths, the largest increase was among white women…

Only cigarettes are deadlier than alcohol: More than 480,000 people die each year in the U.S. because of smoking-related illnesses.

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Venous Thrombosis in Pediatric Inflammatory Bowel Disease

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A recent “Grand Rounds” review of venous thrombosis (VT) in pediatric inflammatory bowel disease (E Mitchel, T Diamond, L Albenberg. J Pediatr 2020; 216: 213-7) provides some practical advice in an area in need of more clarity.

Risk factors for VT:

  • inflammation
  • malnutrition
  • dehydration
  • malabsorption
  • need for surgery
  • medications (eg. steroids)
  • immobilization
  • infection
  • placement of central line
  • hormonal contraceptive use
  • cigarette use
  • hereditary thrombophilia/first-degree relative with VT

Key points:

  • Pediatric patients with IBD are at increased risk for VT with an estimated incidence between 0.09% and 1.9%.  Patients hospitalized with an IBD flare have a “6-fold increased risk for pulmonary embolism and deep-vein thrombosis as compared” to hospitalized patients without IBD.  In another study, the risk was lower with a relative risk for VT of 2.37 for Crohn’s and 1.99 for ulcerative colitis (UC).
  • ESPGHAN guidelines recommend prophylactic anticoagulation in patients with acute severe colitis and at least 1 risk factor (in prepubertal children — at least 2 risk factors).  Mobilization and hydration are also recommended.
  • At the authors’ institution, “patients <12 years do not meet routine criteria” for thromboprophylaxis unless at high risk.
  • Patients >12 years who are at medium or high risk are given mechanical prophylaxis with a pneumatic compression device (if no contraindications).
  • In those at high risk and >12 years, pharmacologic prophylaxis is considered in concert with hematology service. “High risk is considered altered mobility and at least 1 risk factor.”

My take: In adolescents hospitalized with IBD, this article suggests that almost all should receive mechanical prophylaxis for VT and a subset at increased risk may benefit from pharmaccologic prophylaxis.

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From a visit to Montreal

Improving Care Process in Celiac Disease

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Previous studies have documented numerous deficiencies in the care of children with celiac disease, particularly with regard to followup.  A recent study (B Sparks et al. J Pediatr 2020; 216: 32-6) demonstrates that using a prospective patient registry can improve many aspects of care and allows scrutiny of other aspects for further improvement.

In this single center study with 25 pediatric gastroenterologists, the authors reviewed the experience in establishing their “Celiac Care Index.”

Key findings:

  • There was improved adherence: 77%–>89%
  • Improved rates of followup serology: 50–>90%
  • Improved completion of agreed-upon bloodwork: testing for ALT increased from 74% to 96%, Vitamin D from 36% to 83%, and checking hepatitis B immune status from 30% to 80%

When looking at their ‘smartset’ labs obtained in most of their 145 patients, the authors note that several may not be needed:

  • Iron: the authors state that serum iron is not needed in those who have had a ferritin and a CBC.
  • Thyroid testing: no patients had an abnormal free T4 and very few had an abnormal TSH (8 of 120 =7%).  In the subset with abnormal TSH, 5 were normal on repeat testing, 2 had previously recognized thyroiditis, and 1 had TSH elevation related to obesity.

Lab Findings:

  • Hepatitis B: 80 of 115 (70%) showed a lack of immunity to hepatitis B
  • Vitamin D (25-OH): 19 of 114 (17%) had values less than 20 ng/mL
  • ALT: 23 of 131 (18%) had values of ≥40 U/L

My take:

  1. This study shows that careful tracking of patients results in better adherence with established goals and allows for useful modifications.
  2. More long-term followup is needed –some abnormalities, like Vitamin D, may improve with treatment of the underlying disease even in the absence of vitamin D supplementation.
  3. Also, a majority of children lacked an adequate immune response to hepatitis B; testing is important to determine who needs repeat immunization.

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Signage at a restaurant’s bathroom near Mount Tremblant

Disclaimer: This blog, gutsandgrowth, assumes no responsibility for any use or operation of any method, product, instruction, concept or idea contained in the material herein or for any injury or damage to persons or property (whether products liability, negligence or otherwise) resulting from such use or operation. These blog posts are for educational purposes only. Specific dosing of medications (along with potential adverse effects) should be confirmed by prescribing physician.  Because of rapid advances in the medical sciences, the gutsandgrowth blog cautions that independent verification should be made of diagnosis and drug dosages. The reader is solely responsible for the conduct of any suggested test or procedure.  This content is not a substitute for medical advice, diagnosis or treatment provided by a qualified healthcare provider. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a condition

AAP Bariatric Surgery Recommendations

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A recent policy statement (SC Armstrong et al. Pediatrics 2019; 144 (6): e20193223) outlines current evidence regarding adolescent bariatric surgery and makes recommendations for practitioners & policymakers.  There is also an accompanying technical report which provides more detail and supporting evidence.  Thanks to Ben Gold for this reference.

Full PDF Link: Pediatric Metabolic and Bariatric Surgery: Evidence, Barriers, and Best Practices

This policy statement uses “adolescent” to refer to a person from age 13 years to age 18 years.

Background: “Although nearly 4.5 million US adolescents have severe obesity, current estimates suggest that only a small faction undergo metabolic and bariatric surgery…Many providers prefer a “watchful waiting” approach, or long-term lifestyle management.50 However, current evidence suggests that pediatric patients with severe obesity are unlikely to achieve a clinically significant and sustained weight reduction in lifestyle-based weight management programs53 and that watchful waiting may lead to higher BMI and more comorbid conditions…In addition, comparative data examining
postoperative outcomes along the severely obese BMI spectrum (low, middle, and high) suggest that adolescents within a lower BMI range (BMI <55) at the time of bariatric
surgery have a higher probability of achieving nonobese status when compared with individuals with a higher starting BMI (BMI ≥55).”

From Table 2 -Indications for Bariatric Surgery:

  1. Class 2 obesity, BMI ≥35, or 120% of the 95th percentile for age and sex, whichever is lower  along with clinically significant disease, including obstructive sleep apnea (AHI .5), T2DM, IIH, NASH, Blount disease, SCFE, GERD, and hypertension
  2. Class 3 obesity, BMI ≥40, or 140% of the 95th percentile for age and sex, whichever is lower. Clinically significant disease is not required but commonly present

Recommendations for practitioners:

  • Seek high-quality multidisciplinary centers that are experienced in assessing risks and benefits of various treatments for youth with severe obesity, including bariatric surgery, and provide referrals to where such programs are available.
  • Identify pediatric patients with severe obesity who meet criteria for surgery and provide
    timely referrals to comprehensive, multidisciplinary, pediatric-focused metabolic and bariatric surgery programs.
  • Monitor patients postoperatively for micronutrient deficiencies and consider providing iron, folate, and vitamin B12 supplementation as needed.
  • Monitor patients postoperatively for risk-taking behavior and mental health problems.

SYSTEM-LEVEL RECOMMENDATIONS:

  • Advocate for increased access for pediatric patients of all racial, ethnic, and socioeconomic backgrounds to multidisciplinary programs
  • Consider best practice guidelines, including avoidance of unsubstantiated lower age limits, in the context of potential health care benefits and individualized patient-centered care.
  • For insurers: Provide payment for care (pre-operative, operative & post-operative). Reduce barriers to pediatric metabolic and bariatric surgery (including inadequate payment, limited access, unsubstantiated exclusion criteria, and bureaucratic
    delays in approval requiring unnecessary and often numerous appeals) for patients who meet careful selection criteria.

My take: These recommendations are in general agreement with previous guidelines.  I think having the stamp of approval from the AAP is likely to help in getting coverage and may shift attitudes.

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IBD Depression Screening

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LM Mackner et al. JPGN 2020; 70: 42-47. Bonney Reed, our psychologist at GI Care for Kids is one of the authors as well.

Key points:

  • Recommendation #1: Screen adolescents with IBD ages 12 and older for depression annually.
  • Recommendation #2: Screening Measures
    Age 12 years: Moods and Feelings Questionnaire, Short Form (MFQ-SF) ; age 13: Patient Health Questionnaire-9 (PHQ-9)
  • Recommendation #3: Evaluate youth who endorse SI (eg, PHQ-9 item # 9) further
    per clinic protocol or via a suicide screener, such as the Columbia Suicide Severity Rating Scale (C-SSRS)
  • Recommendation #4: Educational Resources. Provide patients, families, and other clinicians with educational resources as needed. An additional aim of our tool kit is to give GI providers resources to assist patients, families, and other clinicians
  • Resources for modules 1-4, Supplemental Digital Content http://links.lww.com/MPG/B721

My take (borrowed from authors): “Implementing depression screening in a busy clinic may seem like a daunting task and is likely to require changes in workflow and procedures. Nonetheless, optimal IBD care treats all aspects of health, and identifying depression symptoms, that often go undetected and can affect IBD outcomes, benefits patients, families, and providers.”  In our office, we have implemented screening and there is now a smartform available in EPIC.  We are fortunate to work closely with psychologists who can help when there is an abnormal screen.

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Disclaimer: These blog posts are for educational purposes only. Specific dosing of medications/diets (along with potential adverse effects) should be confirmed by prescribing physician/nutritionist.  This content is not a substitute for medical advice, diagnosis or treatment provided by a qualified healthcare provider. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a condition.