Gene Therapy Cure For Blindness Gets $850K Price Tag

A research scientist works in Spark Therapeutics’ laboratory.				Spark Therapeutics Inc

The treatment called Luxturna is manufactured by Spark Therapeutics and helps treat Leber congenital aumaurosis, an inherited condition that leads to blindness.

Philadelphia-based Spark Therapeutics said yesterday that it planned to charge $425,000 per eye for Luxturna, the first gene therapy for an inherited disease to reach the USA market. The rare condition only affects two to three people per 100,000. Luxturna will be distributed through just six to eight treatment centers in the USA initially and is expected to be available later in the first quarter. 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.

In an article, Bloomberg's Michelle Cortez noted that the schemes are very different from the approach offered by pharma companies, and could set a precedent for how expensive gene therapies could get to market in the future.

Critics, however, say Luxturna's price is still too high, adding that people without insurance and those who have high deductibles will face the brunt of the cost.

Like most prescription medicines in the US, most of the immediate costs of Luxturna will be borne by insurers - not patients - including private plans and government programs.

We're just going to have to wait and see what impacts the price of Luxturna has on the drug manufacturer, as well as on health insurers.

Spark Therapeutics CEO Jeff Marrazzo co-authored a recent blog post in the journal Health Affairs that said "the current payment system - accustomed to ongoing treatment of chronic diseases - creates significant challenges" for gene therapies and other treatments.

It will retail for $425,000 (Rs 2,69,89,625) per eye, which is less than the $1 million price tag that had been expected before the company received FDA approval in mid-December. The company also stated that its proposal to the Centers for Medicare and Medicaid Services, the government program that covers about 100 million Americans, would allow installment payments for the drug. And the company is also working on covering patients' out of pocket costs as part of the overall price. But the Spark pricing team has worked it out so that under one model Spark can offer payers unspecified rebates at 30 days and 30 months - which is about the average amount of time a patient stays in a commercial plan - if Luxturna falls short of established efficacy goals on vision. 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.

Marrazzo's not saying how much he's offering in rebates, but when I asked him why not a full refund for patients who don't respond adequately, he said that's not possible. "We're at the infancy of what I think could be a lot of innovations in how we pay for these really expensive drugs", Express Scripts' Chief Medical Officer Steve Miller told CNBC. "Our system can not handle unjustified prices like this, and the new payment models announced today are merely a way to disguise a price that is simply too high".

Related News:

  • LG is adding Google Assistant into their 2018 TVs

    LG is adding Google Assistant into their 2018 TVs

    The TV industry surely doesn't need another acronym, but in 2018 it will get at least one more: HFR, short for "high frame rate". But beyond that they will also act as a smart hub for the home meaning you'll be able to control smart home gadgets, too.
    Costa scores on Atleti return

    Costa scores on Atleti return

    But it nearly threatened to ruin his second debut after he injured his knee over-stretching to bag the third goal . Both entered the game after Diego Godin and Fernando Torres had put Atletico 2-0 ahead before the break.
    Paul George: Not 'Championship or Bust' in OKC

    Paul George: Not 'Championship or Bust' in OKC

    I would have loved to go back home and play for my city, so no regrets at all". "So, no regrets at all". George feels this story is what it is and he cares less about the attention it draws.
  • Exiled Mugabe Ally Says Mnangagwa's Government Is 'Illegitimate'

    Exiled Mugabe Ally Says Mnangagwa's Government Is 'Illegitimate'

    Muduvira said that Zhuwawo was supposed to "thank government for taking care of his uncle and not confuse Zimbabweans". Mnangagwa is introducing himself to the regional leaders ahead of general elections in his country later this year.
    White House Aide Miller Booted From CNN Tapper Interview

    White House Aide Miller Booted From CNN Tapper Interview

    Tapper: First of all, I had plenty of questions about immigration but you kept telling the same story over and over. Thank you, Stephen", then turned away from Miller - who was still talking - to tersely announce the next guest.
    Education without skill is troublesome to land jobs: PM Modi

    Education without skill is troublesome to land jobs: PM Modi

    India has taken leaps and bounds in past few years and PM Modi has undoubtedly been the torch bearer of it. The Prime Minister observed that some people say that today's youth does not have "Dhairya" or patience.
  • Many Oregonians Not Happy About Pumping Their Own Gas

    Many Oregonians Not Happy About Pumping Their Own Gas

    An additional three counties will allow self-service gas pumps between 6 p.m. and 6 a.m. "It's staying the way it is", she said. A new law means people in some counties will have to learn how to do something many Oregonians might not even know how to do.
    Pope urges effort to rebuild trust in North Korea, Syria

    Pope urges effort to rebuild trust in North Korea, Syria

    But a house built on the sand of frail and fickle relationships can not stand, " the Pope said January 8. President Donald Trump announced he was recognizing Jerusalem as the capital of Israel.
    Sensex closes flat in cautious trade ahead of earnings season

    Sensex closes flat in cautious trade ahead of earnings season

    ICICI Bank (+1.7%), Vedanta (+1.3%), Hindalco (+1.2%), HPCL (+1.08%) and Adani Ports (+1.02%) were the top gainers on Nifty50. Titan rose 1.30 per cent after the company said its third-quarter jewellery retail growth is in-line with its expectation.
  • 1.26 is Amazon Com Inc's (NASDAQ:AMZN) Institutional Investor Sentiment

    Clarivest Asset Management Limited Liability holds 99,429 shares or 2.13% of its portfolio. 60,145 are owned by Westpac. Therefore 94% are positive. (NASDAQ:AMZN) has "Outperform" rating given on Friday, October 28 by RBC Capital Markets.
    Dana White: UFC Will Move On With Or Without Conor McGregor

    Dana White: UFC Will Move On With Or Without Conor McGregor

    It is highly unlikely to materialise but that did not stop the Irishman from trying to goad "Money" Mayweather into a fight. To watch the clip containing some of the biggest fights we've shown you, just hit play at the top of the article .
    A-list actresses bring activists to Golden Globes red carpet

    A-list actresses bring activists to Golden Globes red carpet

    Streep said Carey made a play to get up, but the actress thought of another solution. Kelly said of Oprah: "I love Oprah".


Most liked

Deepika Padukone wants Ranveer Singh to quit doing this thing
On the other hand, her beau Ranveer Singh has a "quirky" dressing sense which Deepika isn't exactly in love with. Previous year in December, Sabyasachi Mukherjee's sartorial skills were put to display at Virushka wedding.

Morgan Stanley (NYSE:MS) Getting Somewhat Favorable Media Coverage, Accern Reports
After $0.93 actual EPS reported by Morgan Stanley for the previous quarter, Wall Street now forecasts -16.13% negative EPS growth. Morgan Stanley is understood to be announcing bonuses to managing directors today, making it one of the first USA banks to do so.

Minnesota's Reggie Lynch 'categorically denies' sexual misconduct allegations
The university's investigator recommended Lynch be suspended from campus until August 1, 2020, in that matter. The EOAA investigations began in October 2017, around 18 months after the alleged incidents occurred.

Sells 3446 Shares of Devon Energy Corporation (NYSE:DVN)
Rafferty Asset Ltd Liability Company owns 9,332 shares. (NASDAQ:ADI) for 28,017 shares. 317,336 were reported by Meyer Handelman. It worsened, as 55 investors sold DVN shares while 230 reduced holdings. 35 funds opened positions while 113 raised stakes.

NHL: Wild defeat Blackhawks on Suter's goal
Nick Schmaltz , playing in his 100th National Hockey League game, was credited with a secondary assist. Crawford's undisclosed upper-body injury likely would have prevented him from playing, anyway.

Lookout for Price Target? Constellation Brands, Inc. (STZ), WPX Energy, Inc. (WPX)
This company shares are 10.44% off its target price of $239.47 and the current market capitalization stands at $43.54B. The insider Newlands William A sold $616,644. (NASDAQ:EBAY) on Thursday, September 7. $5.45M worth of Stamps.com Inc.

RSS opposes government's FDI in single-brand retail, Air India move
Swadeshi Jagran Manch has said that easing the norms for FDI in SBRT would go against the best interest of the country. The Union Cabinet chaired by the Prime Minister had given its approval to a number of amendments in the FDI Policy.

Trump Refers To Some Countries As 'Sh*thole' Nations
Several African leaders came out on Friday to decry Trump's reported comments, including the African Union (AU). Trump ended DACA in September, leaving around 800,000 recipients - also called DREAMers - in a state of limbo.

USA auto sales drop in 2017
The GM plant in Oshawa is being repurposed to start producing GMC Sierra and Chevrolet Silverdo trucks starting this month. Both auto manufacturers' December sales beat analyst's forecasts and ended a disappointing year on a brighter note.

Subaru VIZIV Performance STI Concept to be revealed at Tokyo Auto Salon
These include an aggressive splitter and sills pinstriped in pink and a stacked rear wing sitting atop the rear deck-lid. It's likely that the concept will offer a glimpse of what a new WRX STi production vehicle could look like.

Worth Watching Stock Occidental Petroleum Corporation (OXY)
To measure price-variation, we found OXY's volatility during a week at 1.27% and during a month it has been found around 1.09%. According to their observations and findings, the stock could provide a high EPS of $0.75/share and a low EPS of $0.21/share.

Rep. Jeff Hoover resigns as Kentucky House Speaker
Still, Hoover made note of the blessings from the entire ordeal. "I will be back here tomorrow fighting for what I believe in". Hoover also made note of people he thought had wronged him in the months since the scandal.

Taoiseach concerned that abortion proposals 'go too far'
Health minister Simon Harris has suggested that the date could be May 25 if there are no delays in the Dail. The referendum is expected to take place early this summer.

Martha McSally launches Senate campaign in heated GOP race
And she concludes with a declaration that "after taking on terrorists in combat, I'm not afraid of the liberals in the Senate". Kyrsten Sinema (D-Ariz.) for the race, a capable fundraiser whom party officials see as a formidable candidate for the seat.

'Star Wars: Rivals' Coming Soon To Mobile Devices, Adds PvP Combat Mode
Though, that shouldn't be too surprising for Star Wars fans to find out, as Disney is always looking to make some money. Lucasfilm and Disney have announced a new PvP mobile game for iOS and Android and it features " Star Wars " characters.