Spark Therapeutics offers medicine for treatment of rare inherited form of blindness

The Most Expensive U.S. Medicine Now Has an Official Sticker Price

Spark Therapeutics, the company producing Luxturna, has just revealed the new therapy will cost $850,000.

Anticipating criticism, Spark promised to reimburse patients if the treatment proves not to be effective, and said it expects U.S. health authorities to authorize payment in stages.

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. It isn't hard to understand why these drugs are hitting the market with sky-high prices, but it is also undeniably problematic when a life-saving medication arrives with a price-tag that is out of reach to many.

"New technologies such as gene and cell therapies hold out the potential to transform medicine", FDA Commissioner Dr. Scott Gottlieb-said in a statement announcing the August approval of Kymriah, considered to be the first gene therapy to be approved for the United States market. Spark Therapies says $850,000 with a money-back guarantee for its Luxturna gene therapy.

8 best things Arizona Wildcats said after beating Oregon State
Sophomore Mikayla Pivec has handled much of the point guard duties, and she hadn't played the position since the seventh grade. The great equalizer, as always, is on the defensive end-with the Beavers ranking first in the Pac 12 in points allowed (68.6).

The drug is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene.

These therapies are generally meant to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years.

Consternation over skyrocketing drug prices, especially in the U.S., has led to intense scrutiny from patients, Congress, insurers and hospitals. As a result of current government drug price reporting rules, the company can not offer an installment plan without being forced to sell the drug to Medicaid at an unsustainably low price.

"For a one-time therapy, like Luxturna, a non-traditional payment and distribution model is necessary to ensure needs of all parties - patients, payers and providers - are addressed", said Jennifer Luddy, a spokeswoman for Express Scripts. The treatment for one pair of eyes was expected by Wall Street to be around $1 million, Bloomberg Technology reported on Wednesday, with the final announced price coming in at $425,000 per eye - just short of the prediction. The gene therapy is widely expected to be the first in a wave of cutting-edge treatments that are targeted at fixing the causes of a wide range of genetic diseases.

US Studies Possible Viral Attacks on its Diplomats in Cuba
He added that he will not reverse those actions until the Cuban government can guarantee the safety of Americans. President Trump and Secretary of State Rex Tillerson have said that Cuba is responsible for the sonic attacks.

This restores the patient's ability to make the missing enzyme - dramatically improving their eyesight and restoring a large degree of independence, although not offering 20/20 vision.

The group said it would share the risk of the treatment failing by paying some health insurers a rebate linked to whether the treatment worked in the first 30 to 90 days, and also to whether it was still effective after 30 months.

Steve Miller, chief medical officer at Express Scripts, said: "For one-time therapies like Luxturna, we believe non-traditional payment and distribution models are needed". "But ... what is a fair price that will maximize affordability and accessibility and provide a reasonable return for the drug?" The pharmaceutical company said that is now negotiating with other insurers for similar payment schemes linked to treatment outcomes.

However, even though the cost is lower than the $1 million price tag that analysts were expecting, there remain concerns that the drug is overpriced.

5 deputies shot, one fatally, in Colorado shooting, 2 civilians wounded
He also said that the officers talked to the gunman and his roommate before Riehl barricaded himself in the bathroom. BuzzFeed News has contacted the Douglas County Sheriff's Office for more information.

Related News:

  • Actually, Oregon's ban on self-service gas is good

    Actually, Oregon's ban on self-service gas is good

    The Bend Bulletin reported many gas stations said they'll continue to pump gas for customers despite the new law. I had to do it once in California while visiting my brother and nearly died doing it.
    Iran Blocks Instagram and Telegram after Protests

    Iran Blocks Instagram and Telegram after Protests

    How broadly Tehran is interfering with internet access remains uncertain. It's better, but it's not like other days, where there wasn't anything.
    Jardim gives Liverpool & Arsenal Lemar hope

    Jardim gives Liverpool & Arsenal Lemar hope

    I wouldn't rule it out, but I don't see him beating down their door saying "it's now or never" because I don't think it is. Seagulls boss Chris Hughton is said to be keen to tie up a deal for the 25-year-old, who plays for French side Angers.
  • Turkey summons USA  charges d'affaires

    Turkey summons USA charges d'affaires

    Both the USA and Turkish interventions in Syria, however, have been dwarfed by that of Assad supporters Russian Federation and Iran.
    Firefighting efforts resume on burning tanker off China

    Firefighting efforts resume on burning tanker off China

    Rabiei as the head of the committee to follow-up the case of the tanker accident off Chinese coast. So far, only one body from the 32-person crew has been recovered.
    Number of people attending stop smoking program rises amid high cigarette prices

    Number of people attending stop smoking program rises amid high cigarette prices

    Non-cigarette tobacco use is associated with subsequent cigarette smoking among USA adolescents, according to a study published online January 2 in JAMA Pediatrics .
  • Surging trucks emerge as bright spot in slowing USA  auto market

    Surging trucks emerge as bright spot in slowing USA auto market

    Cadillac and Lexus both saw their sales drop 8 percent previous year ; Lincoln sales plummeted 17 percent in December. While overall results were unimpressive, sales of trucks and sport utility vehicles remained robust at year end.
    Finally, Atletico Madrid unveils Diego Costa

    Finally, Atletico Madrid unveils Diego Costa

    Atletico Madrid coach Diego Simeone finally has his favorite striker back at the club. "It was very important for me", said Costa. The second leg of the tie against third-tier club holds next Tuesday, with Atletico hosting Getafe in the league this Saturday.
    13 people awaiting admission to Letterkenny University Hospital

    13 people awaiting admission to Letterkenny University Hospital

    The previous highest level of overcrowding was recorded on 3 January a year ago when 612 patients were waiting for a bed. In Dr Susan O'Reilly's plan, these two hospitals are supposed to take patients from Portlaoise if it is downgraded.
  • CES 2018: ASUS Announces the ROG Spotlight

    CES 2018: ASUS Announces the ROG Spotlight

    The keyboard has upgraded switches with 20-million-keystroke durability to cope with the grueling demands of intense gaming. It can pivot a full 360° to allow the object projected to always be upright no matter how the projector is mounted.
    Republican US Representative Issa of California to retire: statement

    Republican US Representative Issa of California to retire: statement

    Democrats need 24 seats nationwide to take Congress and restore the Speaker's gavel to House Minority Leader Nancy Pelosi (D-CA). Ed Royce, the Republican chairman of the House Foreign Affairs Committee, who said Tuesday that he would not seek a 14th term.
    Triple H Talks About Ronda Rousey's Future; 2018 Hall Of Fame Class

    Triple H Talks About Ronda Rousey's Future; 2018 Hall Of Fame Class

    The timing increases speculation that she could be a participant in the first-ever women's Royal Rumble match in Philadelphia. Will Ronda Rousey appear at WWE Royal Rumble? "I think she respects what we do enough to know that it's hard", he said.

Most liked

Doctors warning parents about new "Tide pod" challenge fad
Karen Jenkins, medical director of the Piedmont Medical Center emergency department. Ideas, and even dares about eating the pods followed on Reddit and Twitter .

Would You Pee on Ikea's Pregnancy Test Ad for a Huge Discount?
The paper used in this latest venture actually contains the same technology as that used in a normal pregnancy test kit . And that it fits nicely into IKEA's "Where Life Happens" brand.

500 flights, 400 trains hit due to Delhi fog
Six flights were cancelled and over 60 delayed due to fog at the Indira Gandhi International Airport here this morning. In addition to the fog, the air quality of Delhi has not really improved much, and is still at the "very poor" mark.

UT basketball player Andrew Jones diagnosed with Leukemia
AUSTIN - Texas sophomore shooting guard Andrew Jones has been diagnosed with leukemia , the school announced Wednesday. He previously flirted with leaving for the NBA Draft previous year before deciding to return for his sophomore season.

Jaguars Stymie Bills In Playoff Battle
Jacksonville's only touchdown - and the only touchdown scored in the game - was driven by the scrambling ability of Bortles. The Jags throttled the Steelers at Heinz Field 30-9 in October, pick-sixing Ben Roethlisberger twice to blow that game open.

Vident Investment Advisory LLC Invests $414000 in Marathon Petroleum Corp (NYSE:MPC)
Jet Capital Investors LP increased Altaba Inc stake by 6,473 shares to 5.01 million valued at $331.76 million in 2017Q3. Wells Fargo Com Mn holds 1.07M shares or 0.02% of its portfolio. 69,403 were accumulated by Bowling Ltd Liability Com.

Get a quick look at Hit in this Dragon Ball FighterZ video
Fans who have pre-ordered a digital version of the game will be able to take part in the open beta on January 13. Masako Nozawa, the voice actress behind Goku , and theme song singer Takayoshi Tanimoto were also at the event.

10 tweets that showcase Storm Eleanor at its peak throughout Ireland
A spokesman for the Met Office said: "Public transport may be disrupted or cancelled and some bridges are likely to be closed". Trees, branches and other flying debris were reported as having brought down power lines and poles.

Thomas S. Monson, president of Mormon church, has died
After a tiring search, he decided that joining the Church of Jesus Christ of Latter Day Saints was the best decision for him. Monson was also an ardent football fan, NBC affiliate KSL reported, particularly of the University of Michigan Wolverines.

13000 tourists trapped in Swiss Alps
A British skier remains missing in the French Alps ski resort of Tignes in Savoie, which is under red alert for avalanches. Snowfall was over 1 meter (39 inches) over 24 hours in some remote areas of the region, according to the WSL Institute.

UEFA Team of the Year revealed
There is no place for PSG stars Neymar or Kylian Mbappe, despite their stunning individual form in 2017. UEFA picked its all-star team after 8.8 million votes.

Brookfield-led group to buy Westinghouse Electric for US$4.6 billion
Bringing Westinghouse out of bankruptcy could help close a proposed deal for six of the company's new AP1000 reactors in India. Westinghouse Electric Co. declared bankruptcy protection early past year , leaving a number of nuclear projects in limbo.

Kamloops home values in 2018 property assessment numbers are up again
Condos and townhouses saw the largest spike in value, with 2018 numbers up between five and 35 per cent - or more-in urban areas. Those homeowners whose homes have increased in value are not necessarily facing a hike in property taxes.

Oprah Winfrey's Powerful Speech at Golden Globes Stirs Talks Over Presidency Run
Winfrey appeared on CBS's "This Morning" in October to talk about a story she worked on as a correspondent for "60 Minutes". When that new day finally dawns, it will be because of the magnificent women and many of them are in this room tonight'.

Messi, Suarez & Mascherano touchdown in Barcelona on private jet
Jefferson Lerma miscued a shot wide and Ivi's effort deflected off target as a lively Levante sought an immediate response. The Camp Nou outfit is now on top of the table with an unbeaten run of seventeen games.