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

Published: Wednesday, January 03, 2018 @ 8:33 AM
Updated: Wednesday, January 03, 2018 @ 8:32 AM


            FILE - 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)
FILE - 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.

Can one man survive for a day with only Apple Pay?

Published: Monday, January 23, 2017 @ 1:10 PM

When there's no Apple Pay option or a wallet, coins scrounged from the inside of a car are one way to pay for gas.
Omar L. Gallaga / AMERICAN-STATESMAN
When there's no Apple Pay option or a wallet, coins scrounged from the inside of a car are one way to pay for gas.(Omar L. Gallaga / AMERICAN-STATESMAN)

In the large list of calamities that could happen to you on a given day (let’s not dwell on the possibilities), forgetting your wallet at home is a pretty minor one.

It happened to me recently and would have been absolutely no big deal, except…

1. I live about 45 minutes outside of Austin and didn’t realize the wallet was missing until I was long gone. I couldn’t just run home on a break and retrieve it.

2. I didn’t have any cash of any kind around. There was no $20 bill hiding in my car’s glove compartment or an envelope of petty cash in my desk. I was without dollars, completely.

3. I didn’t bring a lunch to work with me.

4. My car was already running very low on gas.

But I am resourceful and I am a technologically capable man about town in the year 2017. So this should be no problem, right? I have an iPhone and I have Apple Pay, with one of my credit cards housed in the digital guts of my device, like a tiny and very accommodating financier. I imagine him a little guy who wears a monocole and says things like, “Care for a spot of Starbuck’s, sir? I think the Flat White sounds agreeable, don’t you?”

I decided to see how long I could last for the day using only Apple Pay and not asking co-workers for a loan or, say, selling my blood. (Do people really buy blood? Is that a thing?)

My first stop was Company Kitchen, the semi-automated snack area at my workplace where you grab stuff and pay for these items at an ATM-like kiosk. Company Kitchen has a thumbprint reader and it knows my thumb well from dozens of purchases of Topo Chico drinks and maybe lots of bags of chips. Don’t judge.

I rolled up to Company Kitchen to see if lunch might be in order. My Company Kitchen balance was 52 cents, not enough to buy anything, really. I was getting hungry and my thumb would not save me. I considered ordering food from a delivery service such as Favor or UberEATS, which I have installed in my phone with a credit card enabled. But I wasn’t going to be in one place long enough to wait 30 minutes to an hour; I had an appointment to keep.

The next stop, hunger growing, was a local mall for an Apple Store Genius Bar visit. My computer mouse got mangled in an unfortunate drop and my iPhone battery has been inconsistent lately. I asked the Apple employee who was helping me where someone could use Apple Pay at the mall since Apple doesn’t sell anything edible in its store. “Well, you’ve got Starbucks and Chick-fil-A and…” He took a thoughtful pause, “…that’s about it.”

looked on Apple’s website to make sure I might not be missing another nearby Apple Pay-friendly eating option. Nope. He was right.

Armed with an excuse to eat fast food without my kids around, I ordered a chicken salad sandwich, a light lemonade and some waffle fries. I paid with my phone by tapping it on the pay terminal and mashing my thumb on the home button. Easy. Fast. But, unfortunately, not a widely available option given all the food choices around the mall and in the food court.

Chick-fil-A is one restaurant that accepts Apple Pay. You can buy a chicken salad sandwich, waffle fries and a lemonade with your phone if you forget your walled the way writer Omar L. Gallaga did.(Omar L. Gallaga / AMERICAN-STATESMAN)

I had an event in the evening and wasn’t able to start commuting home until close to 10 p.m. that night. I was starting to get hungry again, but made myself wait to eat until I got home. All I had to do was get there. Which was a problem as my car, a Prius, was already edging toward empty.

Let me tell you something about owning a Prius; it makes you feel like you have conquered energy. Even when the tank is on empty, the gas gauge blinking and beeping at you in a miniature panic, you know you’ve got at least 20 or 30 miles before the situation gets dire. You can go quite a ways on no gas.

My empty indicator went off before I’d even left Austin. Could I really make it 50 miles on fumes? I wasn’t sure I was willing to find out.

About 20 miles into my trip, I began to get panicky, my devil-may-care Prius attitude replaced by sweaty fear. I pulled over to see what my gas station options might be with Apple Pay. Exxon and Chevron accept Apple Pay, but it seemed like every station I passed was Valero or Shell. I finally pulled over at a Shell station, hoping against hope that some agreement had been brokered with Apple and new pay terminals installed since the last time I checked. Nope. The cashier was more than adamant: no Apple Pay. No gas.

I wondered if pulling over, shutting down my engine, and starting it back up was going to waste more gas than if I had just kept going. I had visions of tow trucks and embarassing calls to my wife and needing to be bailed out. It didn’t feel great.

Tokens from video arcades and pizza parlors are no help when one forgets to carry a wallet and is trying to rely on Apple Pay for a day.(Omar L. Gallaga / AMERICAN-STATESMAN)

I ransacked my car, looking for any loose change under the floor mats, in the plastic storage compartment between the seats, in the glove compartment. A collection of pennies, a few quarters and some grimy nickels and dimes began to add up. I found a token from Pizza Piper Pizza and stared at it in my hand in disgust. In all, I collected $1.50 in usable coinage. I took it to the cashier. I got enough gas to maybe get home and the cashier got to grudgingly count out a handful of coins.

When there's no Apple Pay option or a wallet, coins scrounged from the inside of a car are one way to pay for gas.(Omar L. Gallaga / AMERICAN-STATESMAN)

The next 30 minutes were filled with anxiety. But I thought I could make it.

I finally got off on my exit, where there’s an Exxon station. I decided to give it a try, filling up my tank so I wouldn’t be in such a panic taking my kids to school the next morning. A sign placed on all the pumps read, “CREDIT/DEBIT DOWN. PLEASE PREPAY CASH INSIDE.”

Exxon is one gas-station chain that accepts Apple Pay, unless the credit card machines are down, as happened to writer Omar L. Gallaga.

I went home, empty tank, with plans to never forget my wallet again.

Here’s the thing about mobile payments: they are clearly the future. They’re easy, convenient, and more intuitive to use than carrying around pieces of plastic and paper. 

But mobile payments are far from ubiquitous. After I mentioned my predicament online, friends suggested great local coffee shops, restaurants and delivery services that accept Apple Pay. But even though it’s getting more common, Apple Pay never seems to be at the right pay terminal. It’s never a problem you don’t have to think about, or the purely in-the-moment experience Apple probably hopes its users are having.

Apple Pay, and alternatives such as Android Pay and Samsung Pay, aren’t always where you want them when you need them. It only takes a day of scrambling for a meal and a tank of gas to show that we’ve got a long way to go.

Senators try to speed up deployment of self-driving cars

Published: Monday, February 13, 2017 @ 5:12 PM

In this Thursday, Aug. 18, 2016, photo, Uber employees test a self-driving Ford Fusion hybrid car, in Pittsburgh.
Jared Wickerham/AP
In this Thursday, Aug. 18, 2016, photo, Uber employees test a self-driving Ford Fusion hybrid car, in Pittsburgh.(Jared Wickerham/AP)

WASHINGTON — In the first major congressional attempt to address the advent of self-driving cars, two senators said Monday they're launching a bipartisan effort to help to speed up the deployment of the vehicles on the nation's roads.

Republican John Thune of South Dakota, chairman of the Senate Commerce, Science and Transportation Committee, and Democrat Gary Peters of Michigan, said they're exploring legislation that "clears hurdles and advances innovation in self-driving vehicle technology."

The senators' counterparts in the House are also gearing up to address the new technology, with a hearing scheduled for Tuesday.

Automakers cite federal requirements that all vehicles must have steering wheels and brake pedals as examples of regulations that presume there will be a human driver and might inhibit the introduction of self-driving cars. Congressional action may be needed to make changes.

"Without changes to those regulations, it may be years before the promise of today's technology can be realized and thousands of preventable deaths that could have been avoided will happen," Michael Ableson, General Motors' vice president of global strategy, plans to tell the House Energy and Commerce Committee, according to prepared testimony.

Proponents of self-driving cars say they hold the potential to dramatically reduce traffic deaths by eliminating human error, which the National Highway Traffic Safety Administration says is a factor in 94 percent of all fatal crashes. More than 35,000 people were killed on the nation's roads in 2015, up over 7 percent from the previous year. Traffic deaths surged an additional 8 percent in the first nine months of last year.

Automakers also say that states are moving ahead with their own regulations, creating the potential for a confusing "patchwork" of laws.

"Our effort will also include a discussion on the existing patchwork of laws and regulations and the traditional roles of federal and state regulators," Thune and Peters said in a joint statement.

Safety advocates have urged the government to set standards that specifically address the safety of self-driving cars. The Obama administration last year issued a voluntary set of safety goals for makers of self-driving cars to meet with the understanding that enforceable regulations could follow.

The Trump administration hasn't yet indicated what approach it will take to the technology.

Some people may first experience riding in a self-driving car by hailing an on-demand, ride-sharing service like Uber or Lyft. Uber began offering passengers rides in autonomous cars with a human as a backup driver last year in Pittsburgh. Joseph Okpaku, Lyft's vice president of government relations, says in prepared testimony for the House hearing that the company's goal is "to operate a pilot in a major city this year that will permit consumers to enjoy, for the first time, a Lyft in an autonomous vehicle."

Tech crawl: Passenger-carrying drones and new features from Google Maps

Published: Monday, February 13, 2017 @ 11:02 AM

The EHang 184 autonomous aerial vehicle is desplayed at the EHang booth at CES International in Las Vegas. 
John Locher/Associated Press
The EHang 184 autonomous aerial vehicle is desplayed at the EHang booth at CES International in Las Vegas. (John Locher/Associated Press)

Happy Monday, Austin. Hope your weekend was a good one.
As you settle into the work week, here’s a look at some of the technology stories building a buzz around the Interwebs this morning:

Passenger carrying drones? Yes, please!

In Dubai, government leaders have an idea for battling traffic jams: a passenger-carrying drone.  Yes, seriously.

Dubai officials say they hope to have the passenger-carrying drone -- which is a Chinese-made device known as the EHang 184 -- buzzing through the skyline of the city-state as soon as July.

The craft can carry a passenger weighing up to 100 kilograms (220 pounds) and a small suitcase. After buckling into its race-car-style seat, the craft's sole passenger selects a destination on a touch-screen pad in front of the seat and the drone flies there automatically.

It’s hard to know if the egg-shaped, four-legged craft will really take off as a transportation alternative in this car-clogged city.

"This is not only a model," said Mattar al-Tayer, head of Dubai's Roads & Transportation Agency. "We have actually experimented with this vehicle flying in Dubai's skies.”

Here’s hoping it works. And that we can get a fleet of them for Austin.

New features for Google Maps

Google Maps is rolling out a new feature that lets you create Spotify-like "playlists" of favorite local spots on Google Maps that you can then share with friends. The idea is, obviously, pretty similar to Foursquare, but with Google’s size and reach,  the idea is a huge network of lists can be created that gives users "a speed-dial-like network of places if you're trying to figure out where to dine or visit on short notice," Engadget writes.  TechCrunch and The Verge also weigh in on the new Google feature and its possible uses, with The Verge piece wondering if Google Maps is trying to step into the social network space.

New tech might mean better Wi-Fi

We’d all like better, more consistent Wi-Fi networks, right? Well, Qualcomm on Monday rolled out some new technology that some tech experts say could make your home Wi-Fi a lot better. Mashable’s Raymond Wong has a piece on how the new Qualcomm tech works, and what it might mean for you.

Spotify helps listeners cook turkey with Thanksgiving-themed playlists

Published: Tuesday, November 22, 2016 @ 3:09 PM



Spotify
(Spotify)

Spotify wants to help you cook your Thanksgiving turkey just right.

Time for Turkey, the streaming service's holiday-specific program,  gives users three steps for a perfectly-done turkey: provide the turkey's weight, choose what genre of music you'd like to listen to, and enjoy.

Genres include Americana, "Family Time," "Feeling Thankful," "Club Kitchen," "Freshly Baked" and "Golden Oldies." Each themed playlist name works off of the genre. For example,  the "Freshly Baked" playlist brings out the newest hits for you to enjoy some never-tasted-before singles. "Family Time" includes songs that every generation can enjoy, such as "I'm A Believer" by the Monkees or "Riptide" by Vance Joy.

Unfortunately, none of the playlists include the classic Thanksgiving songs of Bob's Burgers, including one of the best Thanksgiving song collaboration of all time.

While the songs themselves may not be Thanksgiving-themed, the variety of turkey accompaniment that Spotify has provided contributes so much to the dinner process. Even if only for timing purposes, Time for Turkey will add to your Thanksgiving feast. In an age where we see Christmas advertisements earlier and earlier each year, this feels like a good way for a company to give Thanksgiving a nod of acknowledgment.