👶 A Real Story: When a Cure Is Too Expensive to Use
In 2024, a child born with ADA-SCID — a rare genetic immune disorder also called “bubble baby syndrome” — had a shot at life thanks to a one-time gene therapy. It wasn’t hypothetical. It worked. It was safe. It was ready.
And then?
The biotech company abandoned it.
Why? Not enough market. Not profitable. Not worth it.
So who delivered the cure?
👉 Great Ormond Street Hospital — a hospital, not a billion-dollar pharma firm — stepped up to license and deliver the therapy themselves.
When life-saving medicine depends on business models instead of biology, something’s broken.
This isn’t an outlier. It’s a symptom of a much larger disease: gene therapies priced like Ferraris and rationed like champagne.
🔥 Hot Take: We’re Building Cures No One Can Afford
Gene editing. CRISPR. Precision medicine.
The headlines scream “breakthroughs.”
But if no one can pay for it, it’s not a breakthrough.
It’s a PR stunt.
| Gene Therapy | Indication | List Price |
|---|---|---|
| Zynteglo | Beta Thalassemia | $2.8 million |
| Hemgenix | Hemophilia B | $3.5 million |
| Lenmeldy | Metachromatic Leukodystrophy (MLD) | $4.25 million |
These are one-time treatments. Yes, they could replace a lifetime of drugs and hospital visits.
But right now, they’re replacing access with elitism.
🧠 3 Expert Opinions You Need to Know
1. Dr. Peter Marks – Director, FDA Center for Biologics Evaluation and Research
“We’re entering a new era — but if we don’t solve pricing, we risk cutting off the very patients we aim to serve.”
👉 Takeaway: The science is moving faster than the system can keep up.
2. Dr. William Padula – Health Economist, University of Southern California
“We’ve normalized drug launches at Ferrari prices. But what happens when the engine fails and no one can afford repairs?”
👉 Takeaway: High price tags ≠ long-term sustainability.
3. Dr. Catriona Crombie – Head of Rare Disease at LifeArc (UK)
“We’ve shown that it’s possible to develop and deliver gene therapy without billion-dollar IPOs — if you’re willing to rethink the model.”
👉 Takeaway: A better system isn’t a fantasy. It just needs guts.
🎯 Tactical Advice: What Clinicians Can Do Right Now
✅ 1. Get Ahead of Payers
Start conversations before therapy. Pre-authorize. Document outcomes. Get alignment early.
✅ 2. Use the Human Angle
Don’t just show data. Show the child who wants to go to school. Tell the story. Create urgency.
✅ 3. Lean on Self-Funded Employers
They have more flexibility and long-term ROI thinking. Craft your pitch around savings over time.
✅ 4. Create a Toolkit
Crowdsource appeal letters, payer contacts, success cases. Share them with other providers.
✅ 5. Challenge "Best Practices"
If what’s “best” isn’t working, question it. Break the rules (ethically). Lead the change.
❓ FAQ – Fast Answers for Busy Professionals
💸 Why are gene therapies so expensive?
Small patient populations, high R&D costs, and lack of pricing oversight create a “charge-what-you-can” market.
❌ Can insurers deny coverage?
Yes — often labeling it “investigational” or lacking long-term data. Your appeals must be airtight.
⚖️ Who regulates pricing?
Nobody, really. The FDA approves safety and efficacy — not price. That’s left to market forces.
🧪 Is there a cheaper alternative?
Rarely — but public hospitals and nonprofit groups like LifeArc are pioneering lower-cost access models.
📣 What can I do as a physician?
Tell stories. Advocate early. Build payer relationships. Join coalitions. Educate yourself — and others.
📚 References – Read the Latest
-
Reuters – “New Drug Prices in U.S. Have More Than Doubled Since 2018”
📊 Prices for newly launched pharmaceuticals have more than doubled in recent years, with gene therapies driving much of the increase.
🔗 Read more here -
Forbes – “Insurers Prepare for the Gene Therapy Boom”
💰 Health plans are bracing for the financial impact of high-cost gene therapies.
🔗 Check out the article here -
The Guardian – “Hospital Rescues Gene Therapy After Company Walks Away”
🌍 Efforts to rescue a rare disease gene therapy after industry abandonment have been detailed in this article.
🔗 Read more here
🚀 Call to Action: Get Involved. This Is the Moment.
This isn’t just a medical problem.
It’s a human one.
Gene therapy should be a right — not a raffle ticket.
👉 Get involved.
👉 Join the movement.
👉 Raise your voice.
👉 Be part of the future.
👉 Claim your spot in the conversation.
👉 Start here.
👉 Share your insights.
👉 Fuel your growth.
👉 Be the change.
Drop your email. Join the coalition. Start the conversation.
Let’s rewrite the future of medicine — together.
📣 Hashtags for Reach and Impact
If this matters to you — amplify it.
#GeneTherapyForAll #FutureOfMedicine #ReimbursementReform #PatientAccess #HealthEquity #DoctorsOfLinkedIn #MedicalInnovation #HealthJustice #RareDiseaseAwareness #DigitalHealth #PharmaEthics #AccessMatters #CRISPR #GeneEditing #HealthPolicy #PublicHealthNow #NextGenHealthcare #TherapyNotLuxury #BeTheChange
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