Breaking News
April 22, 2018 - New Drug Combo Ups Survival in HER2/neu Uterine Serous Cancer
April 22, 2018 - Researchers chart a new way to look at concussion
April 22, 2018 - Every parent needs to know fundamental red flags for autism
April 22, 2018 - Anatotemp expands anatomic dental implant healing abutments with 4Side anti-rotational connection
April 22, 2018 - Gene Twist Can Make Your Blood Pressure Spike From Salt
April 22, 2018 - Study suggests failed osteoarthritis drug could help treat opioid addiction
April 22, 2018 - More Americans aware of growing problem of opioid addiction
April 22, 2018 - KHN’s ‘What the Health?’ Nothing in health care ever goes away
April 22, 2018 - BGS to promote high-quality sterilization services at Health GB in Manchester
April 22, 2018 - New integrated POC tool detects biomarkers of heart failure rapidly and precisely
April 22, 2018 - Direct electrical current can be delivered to nerves for blocking pain signals
April 22, 2018 - Newly Published Phase 2 Study Found Esketamine Demonstrated Significantly Rapid Improvements in Depressive Symptoms and Suicidality
April 22, 2018 - Healthy red blood cells owe their shape to muscle-like structures
April 22, 2018 - Researchers present case study of management of rheumatic mitral regurgitation in woman contemplating pregnancy
April 22, 2018 - New black Porvair Krystal UV Quartz microplates for Circular Dichroism measurements
April 22, 2018 - Advanced flow chemistry modules enhance control of nanoprecipitation
April 22, 2018 - Look! Down in the petri dish! It’s a superplatelet!
April 22, 2018 - Research reveals why people with tetraplegia more likely to suffer from sleep apnea
April 21, 2018 - New non-invasive nerve stimulation may offer relief for people with hand tremor
April 21, 2018 - Smartphone App May Up Medication Adherence in HTN
April 21, 2018 - Western diet depletes artery-protecting immune cells
April 21, 2018 - Excelitas Technologies launches new powerful LED light source for fluorescence microscopy
April 21, 2018 - Academia and high tech companies join forces to increase production capacity for microfluidic systems
April 21, 2018 - Developing cooking skills as young adult may have long-term health benefits
April 21, 2018 - Study compares survival outcomes of different drugs for type 2 diabetes
April 21, 2018 - More Than 40 Percent of Americans Breathe Dirty Air: Report
April 21, 2018 - Obstructive sleep apnea – Genetics Home Reference
April 21, 2018 - More evidence shows exposure to traffic and outdoor air pollution increases risk of asthma
April 21, 2018 - Novel gold nanoparticle technology could guide cancer treatment in real-time
April 21, 2018 - News coverage of Ebola impacted public’s perception on disease and survivors
April 21, 2018 - S.Africa’s DIY battle against HIV
April 21, 2018 - Children with autism have gastrointestinal and immune system deregulation, research finds
April 21, 2018 - Human brain processes sight and sound in the same way, shows study
April 21, 2018 - Evolutionary history of tumor helps predict severity of prostate cancer
April 21, 2018 - Pepper plant metabolizes antibiotic in personal care products
April 21, 2018 - Tradeshow Talks with Integra
April 21, 2018 - EPFL becomes part of Chan Zuckerberg’s project to develop Human Cell Atlas
April 21, 2018 - Pfizer Announces Positive Topline Results From Phase 3 ATTR-ACT Study Of Tafamidis In Patients With Transthyretin Cardiomyopathy
April 21, 2018 - Breaking through the HIV vaccine ‘logjam’
April 21, 2018 - IntelliCyt introduces new QSol buffer to enable robust, consistent sampling
April 21, 2018 - Scientists publish comprehensive lineage tree of whole adult animal in Science journal
April 21, 2018 - Innovative method based on FluidFM technology could revolutionize biological research
April 21, 2018 - Americans world’s biggest TV addicts, watching four hours a day
April 21, 2018 - Investigational drug may help increase protein levels in babies with spinal muscular atrophy
April 21, 2018 - Study shows distinctions between age groups in predicting and responding to stress at home
April 21, 2018 - Aziyo Biologics, BIOTRONIK enter into US co-distribution agreement
April 21, 2018 - Opiate Use Linked to Early Mortality in IBD Patients
April 21, 2018 - Online ads help pregnant smokers quit
April 21, 2018 - Opioid pain medications may not be safe for hemodialysis patients
April 21, 2018 - Rare variants in non-coding DNA inherited from parents heighten autism risk
April 21, 2018 - A needleless glucose monitor for diabetes patients
April 21, 2018 - BD introduces new informatics and automation solutions for clinical laboratories
April 21, 2018 - Turn Chores Into a Fitness Routine
April 21, 2018 - DNA methylation plays key role in stem cell differentiation
April 21, 2018 - Scientists find link between soil metals and cancer mortality
April 21, 2018 - Experts discuss implications of low calcium intake in global population
April 21, 2018 - GNA Biosolutions to display Pharos V8 Laser PCR instrument at Analytica trade fair
April 21, 2018 - People with vitamin D deficiency may be at greater risk of diabetes
April 21, 2018 - Study findings could open new possibilities for treating cancer with adenovirus
April 21, 2018 - People who use medical marijuana have higher rates of prescription drug use, study finds
April 21, 2018 - Study debunks ‘myth’ that strenuous exercise dampens immunity
April 21, 2018 - FDA approves marijuana based medication for epilepsy treatment
April 21, 2018 - Researchers find novel genes for longevity in mammals
April 21, 2018 - GNA Biosolutions and project partners launch new research project to develop TB diagnostic platform for POC applications
April 21, 2018 - $2 million funding boosts progress of UAB biomedical startup
April 21, 2018 - Scientists identify gene responsible for evolution of recombination rates
April 21, 2018 - UConn researchers develop new composite for healing broken load-bearing bones
April 21, 2018 - Study examines how higher-order gene combinations help maintain normal cell physiology
April 21, 2018 - Study challenges use of whole-brain radiation for small-cell lung cancer patients with brain metastases
April 21, 2018 - Researchers discover blood biomarkers that may help detect, confirm mild traumatic brain injury
April 21, 2018 - People who become physically active after heart attack more likely to live longer, shows research
April 21, 2018 - CPRIT awards $2 million grant to push forward breast cancer research in West Texas
April 21, 2018 - Unhealthy diet damages the development of immature fat cells, study shows
April 21, 2018 - Consumption of protein supplements with meals may provide better weight control
April 21, 2018 - 4 Types of Foods to Help Boost Your Memory
April 21, 2018 - How did gonorrhea become a drug-resistant superbug?
April 21, 2018 - DePuy Synthes announces clinical results related to use of CORAIL Hip System Femoral Stems
April 21, 2018 - New initiative launched to support goals of Human Cell Atlas
April 20, 2018 - Teen patient gets a new lease on life
April 20, 2018 - Cancer Australia launches new framework to improve outcomes for lung cancer patients
Price tag on gene therapy for rare form of blindness: $850K

Price tag on gene therapy for rare form of blindness: $850K

image_pdfDownload PDFimage_print
Price tag on gene therapy for rare form of blindness: $850K
In this Oct. 4, 2017, file photo, Dr. Albert Maguire, right, checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelphia. Misa was 4-years-old when he received his gene therapy treatment. The first-of-its kind genetic treatment for blindness will cost $850,000, less than the $1 million price tag that had been expected, but it’s still among the most expensive genetic therapies in the world. Spark Therapeutics said it decided on the lower price tag for Luxturna, after hearing from health insurers about their ability to cover the injectable treatment. (AP Photo/Bill West, File)

A first-of-its kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising questions about the affordability of a coming wave of similar gene-targeting therapies.

The injectable treatment from Spark Therapeutics can improve the eyesight of patients with a rare genetic mutation that affects just a few thousand people in the U.S. Previously there has been no treatment for the condition, which eventually causes complete blindness by adulthood.

Pricing questions have swirled around the treatment due to a number of unusual factors—it is intended to be a one-time treatment, it treats a very small number of patients and represents a medical breakthrough.

Previously, Spark suggested its therapy, Luxturna, could be worth more than $1 million. But the company said Wednesday it decided on the lower price after hearing concerns from health insurers about the affordability of the treatment.

Consternation over skyrocketing drug prices, especially in the U.S., has led to intense scrutiny from patients, politicians, insurers and hospitals.

“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said CEO Jeffrey Marrazzo, in an interview with The Associated Press.

Luxturna is still significantly more expensive than nearly every other medicine on the global market, including two other gene therapies approved earlier last year in the U.S.

Pharmaceutical industry critics said the slightly lower cost is a distraction from the ongoing problem of unsustainable drug prices.

“The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable,” said Dr. Peter Bach, director of a policy center at Memorial Sloan Kettering Cancer Center in New York.

Approved last month, Luxturna, is the nation’s first gene therapy for an inherited disease. It requires a 45-minute operation in which a tiny needle delivers a replacement gene to the retina, tissue at the back of the eye that converts light into electric signals that produce vision. The therapy will cost $425,000 per injection. The price does not include the cost of the operation, which Spark estimates will cost between $4,000 and $5,000.

The treatment is part of an emerging field of medicine that could produce dozens of new gene-targeting medications in the next few years.

Like Luxturna, these therapies are generally intended to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years. Even compared to other one-time gene therapies Luxturna is still an outlier. Two customized gene therapies for blood cancer approved last year are priced at $373,000 and $475,000.

Many older drugs for ultra-rare diseases also cost hundreds of thousands of dollars per year, and can quickly exceed a million dollars. For instance, a drug from Biogen called Spinraza, which treats a rare neuromuscular disorder, costs $750,000 for the first year’s supply and $375,000 for subsequent years. The drug is intended to be taken for life.

Drug prices are not regulated in the U.S., as they are in many other countries, so drugmakers can price their goods like any other manufacturer. Drugmakers have historically offered little explanation for the prices they charge, other than to cite the high cost of developing a drug and the fact that so many drugs fail during trials and must be abandoned. However, some companies have begun to offer more detailed reasoning as the backlash against drug prices has grown more heated.

Spark Therapeutics, based in Philadelphia, has said that the cost for a lifetime of blindness—including lost earnings and caregiver wages—can easily exceed $1 million.

Not everyone agrees with that argument.

Even at $850,000 a preliminary analysis by one group found that the drug would need to be priced significantly lower to be a good value.

The estimate by the non-profit Institute for Clinical and Economic Review assumes the drug will maintain patients’ vision for 10 years. However, Spark expects the drug’s effect to be long-lasting, if not lifelong, though it has only tracked patients for about four years.

The group’s director, Dr. Steven Pearson, said paying for gene therapies that have not yet shown lasting benefits will be an ongoing issue.

“If the payment is going to be done all at once that will create real affordability concerns if we don’t have tremendous confidence about how long the effects of the treatment will last,” he said.

At least one gene therapy sold overseas already crossed the $1-million price threshold, a treatment for a rare protein disorder launched in Europe. Manufacturer uniQure stopped selling the therapy last year due to a lack of demand. It was never approved in the U.S.

Like most prescription medicines in the U.S., most of the immediate costs of Luxturna will be borne by insurers—not patients—including private plans and government programs. For patients, Spark said it will cover all out-of-pocket expenses needed to obtain the medication, including transportation to hospitals trained to administer the injections.

Given Luxturna’s federal approval and strong study results, experts say U.S. insurers will likely cover the drug.

Spark will try to deflect some pricing concerns by offering unconventional payment plans to insurers. Under one arrangement with the non-profit insurer Harvard Pilgrim, Spark will refund some costs if patients don’t experience the expected improvements in vision. The company did not disclose how much money would be returned to the insurer, which covers more than a million people in New England.

Spark said it is also discussing a proposal in which insurers would pay for the drug in installments over several years. That idea would apply to government programs like Medicare and Medicaid, which provide health coverage to the poor and elderly.


Explore further:
Gene therapy for rare form of blindness wins US approval

Tagged with:

About author

Related Articles