Skip to content
  • About
  • Contact
  • Contribute
  • Book
  • Careers
  • Podcast
  • Recommended
  • Speaking
  • All
  • Physician
  • Practice
  • Policy
  • Finance
  • Conditions
  • .edu
  • Patient
  • Meds
  • Tech
  • Social
  • Video
    • All
    • Physician
    • Practice
    • Policy
    • Finance
    • Conditions
    • .edu
    • Patient
    • Meds
    • Tech
    • Social
    • Video
    • About
    • Contact
    • Contribute
    • Book
    • Careers
    • Podcast
    • Recommended
    • Speaking

You can save your baby for $2 million. Can we afford to do this?

Edward Hoffer, MD
Meds
May 31, 2019
Share
Tweet
Share

You can save your baby for $2 million. Can you afford to do this? If you cannot, should we all pay?

Spinal muscular atrophy (SMA) is a rare genetic disease affecting the part of the nervous system that controls voluntary muscle movement. All types of SMA result from a single known cause, a deficiency of a protein called SMN, for “survival of motor neuron.” When SMA symptoms are present at birth or by the age of 6 months, the disease is called SMA Type 1 (also called Werdnig-Hoffmann disease). Babies with SMA Type 1 typically have generalized muscle weakness, a weak cry, and breathing distress. They often have difficulty swallowing and sucking, and don’t reach the developmental milestone of being able to sit up unassisted. These babies have two copies of the SMN2 gene, one on each chromosome 5. Over half of all new SMA cases are SMA Type 1. It is a horrible disease; untreated, half of all affected infants will not live to one year of age, or if alive will require a ventilator (breathing machine). The disease affects about one in every 10,000 births, so there are about 300 to 400 babies born each year in the United States with this up-until-now fatal disease.

On May 24, the FDA approved a gene-based therapy for SMA-1 called Zolgensma, developed by a company that was bought by Novartis. The therapy involves injecting affected babies with an adenovirus containing a copy of the correct gene, that is taken up by the babies’ spine and theoretically cures the disease permanently. The trial which led to FDA approval was done on 22 affected babies; it showed that 21 of 22 were alive and reaching expected developmental milestones at 9.5 months of age.

Untreated, 50 percent of these babies would not be expected to be alive at one year. For the parents of these babies, a miracle. The only fly in the ointment was that Novartis announced the treatment would be priced at $2.125 million! The only previously-approved gene therapy for an inherited disease was approved in 2017 for a rare, previously untreatable, eye disorder leading to blindness. This was priced at $425,000 for each eye. These two treatments promise (though long-term results are many years away) to cure a disease. We are now also starting to see new drugs to treat uncommon and hard-to-treat diseases that are not cures but which carry a sky-high price tag. One is Orkambi, used to treat cystic fibrosis, a hereditary disease that is treated much more easily than it used to be, though children with it are still prone to repeated lung infections. This drug clearly improves the course of patients with cystic fibrosis, but modestly, and is priced at $250,000 a year and must be taken indefinitely.

The focus of lawmakers has been, not surprisingly, on common drugs that are seeing steady price rises well above inflation, and which are taken by millions of people. Insulin is the classic example. Its price has resulted in diabetics skipping their doses and getting sicker or even dying, and this for a drug that has been in use since 1922. While pharmaceutical companies have tweaked the molecule, there have been no breakthroughs. I believe that there is enough outrage that Washington will convince or force the pharmaceutical industry to at least moderate their price increases.

What we have not yet come to grips with is how to deal with the astronomical prices of the new “breakthrough” drugs such as Zolgensma. It is hard to see a health insurer refusing to pay for a truly life-saving drug. Among the many problems to be faced are how an insurance company deals with the fact that they may be paying for a drug for a patient who will not be even in their plan next year: Is it fair to them? What happens if the miracle loses its luster a few years down the road. The results for Zolgensma are after less than a year of use. What happens if the effect wears off? Does Novartis have to refund some or all of the money? If we treat 350 babies a year, we are talking $700 million per year — and the U.S. already spends too much on health care. Does that money come at the price of bridges falling down? Since many of these new gene-based therapies grew out of public (NIH) funded research, it is reasonable for our elected representatives to negotiate a “fair” price that takes this into account and does not simply allow big pharma to enrich its executives and shareholders.

You remember that old Chinese curse: “May you live in interesting times.” Well, hold onto your hats. The next few years will be very interesting.

Edward Hoffer is an internal medicine physician and author of Prescription for Bankruptcy: A doctor’s perspective on America’s failing health care system and how we can fix it. He blogs at What’s wrong with health care in America?

Image credit: Shutterstock.com

Prev

A physician's sudden, disastrous day. And its tragic consequences.

May 30, 2019 Kevin 1
…
Next

Ageism in medicine is real. How can it be stopped?

May 31, 2019 Kevin 1
…

Tagged as: Genetics, Medications

Post navigation

< Previous Post
A physician's sudden, disastrous day. And its tragic consequences.
Next Post >
Ageism in medicine is real. How can it be stopped?

ADVERTISEMENT

ADVERTISEMENT

ADVERTISEMENT

More by Edward Hoffer, MD

  • Unveiling alcohol’s health paradox: heart benefits and detrimental effects

    Edward Hoffer, MD
  • Gun crisis in America: Youth fatalities on the rise

    Edward Hoffer, MD
  • One person’s wasteful medical spending is another person’s income

    Edward Hoffer, MD

Related Posts

  • Tips to help you afford medications

    Roy Benaroch, MD
  • Why the baby formula shortage happened

    Divya Srinivasan and Tejas Sekhar
  • The fight to save Howard University College of Medicine

    Vicky Li and Naveen Balakrishnan
  • Saving our mothers requires taking more than baby steps 

    Janice Phillips, PhD, RN and Gina Lowell, MD, MPH
  • I’m sorry that we couldn’t save you

    Evan Schauer
  • Nurse practitioners will save primary care

    Leah Hellerstein, LCSW

More in Meds

  • Why retail pharmacies are the future of diverse clinical trials

    Shelli Pavone
  • Why does rifaximin cost 95 percent more in the U.S. than in Asia?

    Jai Kumar, MD, Brian Nohomovich, DO, PhD and Leonid Shamban, DO
  • A world without antidepressants: What could possibly go wrong?

    Tomi Mitchell, MD
  • The truth about GLP-1 medications for weight loss: What every patient should know

    Nisha Kuruvadi, DO
  • The hidden bias in how we treat chronic pain

    Richard A. Lawhern, PhD
  • Biologics are not small molecules: the case for pre-allergy testing in an era of immune-based therapies

    Robert Trent
  • Most Popular

  • Past Week

    • What the world must learn from the life and death of Hind Rajab

      Saba Qaiser, RN | Conditions
    • Why Medicaid cuts should alarm every doctor

      Ilan Shapiro, MD | Policy
    • When the diagnosis is personal: What my mother’s Alzheimer’s taught me about healing

      Pearl Jones, MD | Conditions
    • Key strategies for smooth EHR transitions in health care

      Sandra Johnson | Tech
    • Reassessing the impact of CDC’s opioid guidelines on chronic pain care [PODCAST]

      The Podcast by KevinMD | Podcast
    • Why flashy AI tools won’t fix health care without real infrastructure

      David Carmouche, MD | Tech
  • Past 6 Months

    • Why tracking cognitive load could save doctors and patients

      Hiba Fatima Hamid | Education
    • How dismantling DEI endangers the future of medical care

      Shashank Madhu and Christian Tallo | Education
    • What the world must learn from the life and death of Hind Rajab

      Saba Qaiser, RN | Conditions
    • How scales of justice saved a doctor-patient relationship

      Neil Baum, MD | Physician
    • The silent toll of ICE raids on U.S. patient care

      Carlin Lockwood | Policy
    • Why shared decision-making in medicine often fails

      M. Bennet Broner, PhD | Conditions
  • Recent Posts

    • The hidden incentives driving frivolous malpractice lawsuits

      Howard Smith, MD | Physician
    • Why what doctors say matters more than you think [PODCAST]

      The Podcast by KevinMD | Podcast
    • How Mark Twain would dismantle today’s flawed medical AI

      Neil Baum, MD and Mark Ibsen, MD | Tech
    • Mastering medical presentations: Elevating your impact

      Harvey Castro, MD, MBA | Physician
    • Marketing as a clinician isn’t about selling. It’s about trust.

      Kara Pepper, MD | Physician
    • Graduating from medical school without family: a story of strength and survival

      Anonymous | Education

Subscribe to KevinMD and never miss a story!

Get free updates delivered free to your inbox.


Find jobs at
Careers by KevinMD.com

Search thousands of physician, PA, NP, and CRNA jobs now.

Learn more

View 3 Comments >

Founded in 2004 by Kevin Pho, MD, KevinMD.com is the web’s leading platform where physicians, advanced practitioners, nurses, medical students, and patients share their insight and tell their stories.

Social

  • Like on Facebook
  • Follow on Twitter
  • Connect on Linkedin
  • Subscribe on Youtube
  • Instagram

ADVERTISEMENT

ADVERTISEMENT

ADVERTISEMENT

ADVERTISEMENT

  • Most Popular

  • Past Week

    • What the world must learn from the life and death of Hind Rajab

      Saba Qaiser, RN | Conditions
    • Why Medicaid cuts should alarm every doctor

      Ilan Shapiro, MD | Policy
    • When the diagnosis is personal: What my mother’s Alzheimer’s taught me about healing

      Pearl Jones, MD | Conditions
    • Key strategies for smooth EHR transitions in health care

      Sandra Johnson | Tech
    • Reassessing the impact of CDC’s opioid guidelines on chronic pain care [PODCAST]

      The Podcast by KevinMD | Podcast
    • Why flashy AI tools won’t fix health care without real infrastructure

      David Carmouche, MD | Tech
  • Past 6 Months

    • Why tracking cognitive load could save doctors and patients

      Hiba Fatima Hamid | Education
    • How dismantling DEI endangers the future of medical care

      Shashank Madhu and Christian Tallo | Education
    • What the world must learn from the life and death of Hind Rajab

      Saba Qaiser, RN | Conditions
    • How scales of justice saved a doctor-patient relationship

      Neil Baum, MD | Physician
    • The silent toll of ICE raids on U.S. patient care

      Carlin Lockwood | Policy
    • Why shared decision-making in medicine often fails

      M. Bennet Broner, PhD | Conditions
  • Recent Posts

    • The hidden incentives driving frivolous malpractice lawsuits

      Howard Smith, MD | Physician
    • Why what doctors say matters more than you think [PODCAST]

      The Podcast by KevinMD | Podcast
    • How Mark Twain would dismantle today’s flawed medical AI

      Neil Baum, MD and Mark Ibsen, MD | Tech
    • Mastering medical presentations: Elevating your impact

      Harvey Castro, MD, MBA | Physician
    • Marketing as a clinician isn’t about selling. It’s about trust.

      Kara Pepper, MD | Physician
    • Graduating from medical school without family: a story of strength and survival

      Anonymous | Education

MedPage Today Professional

An Everyday Health Property Medpage Today
  • Terms of Use | Disclaimer
  • Privacy Policy
  • DMCA Policy
All Content © KevinMD, LLC
Site by Outthink Group

You can save your baby for $2 million. Can we afford to do this?
3 comments

Comments are moderated before they are published. Please read the comment policy.

Loading Comments...