Gene therapies offer dramatic promise but shocking costs

Washington Post

For most of her life, Allison Corona lived in a world dimmed by bad genetic luck. A disease called Leber’s congenital amaurosis left her legally blind at age 4. She could not navigate the short distance from her driveway to her front door after dusk.

Three years ago, Corona, now 23, received an experimental medical treatment aimed at fixing the faulty genes in her eyes. Researchers at the Children’s Hospital of Philadelphia injected viruses carrying a good copy of her errant gene into her right eye and, nine days later, her left eye.

The world around her, once dark and austere, soon grew brighter. Her vision is still far from perfect, but for the first time, she sees that paper towels have texture. She marvels at the velvet floral wallpaper that covers her bedroom wall. She takes a college class that gets out at 10:30 p.m. and no longer fears getting stranded in the night.

Right now there seems to be a kind of crossroad in the healthcare industry. On the one hand there is real promise showing in trials being run on patients with genetic disorders. Cures with a one time shot, as opposed life long treatments at the cost of $100,000s a year in some cases. On the other hand, we have a Pharmaceutical Industry that will not want to lose their profits. It is way to complicated for me to understand.

Here is a video with the head honcho at Bluebird Bio explaining a little bit about what gene therapy is.

In the video you can also see the dilemma the corporations face. "Forget the price. What's the value of that treatment?"

There seems to be a bright future in the use of gene therapy.

A group of researchers at the University of Pennsylvania’s Perelman School of Medicine has developed a way to modify the human body’s immune system, using a deactivated HIV virus, to recognize and kill leukemia cells.

Other institutions are also researching this form of treatment, known as immunotherapy, but the results at Penn, led by Dr. Carl June, have been so promising that the drug company Novartis has entered into a commercial partnership with the school, and the Food and Drug Administration has granted “breakthrough” status to the treatment to speed their review.

Last December, June and his colleagues updated the results of their trial: Of the nearly 100 adult and pediatric patients who have undergone the treatment, about 70 percent responded, meaning their tumors shrunk or disappeared. The research team is still trying to determine how long patients will remain in remission, but several adult patients are cancer free three or four years after their treatment. And the first pediatric patient, Emily Whitehead, is still in remission two years after she received the treatment.

PBS
PennMedicine

As of now, there have been no approved treatments in the US. Only clinical trials. Last year, Dutch biotech firm UniQure rolled out the first gene replacement drug at a cost of over $1 million.

The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1 million euro ($1.4 million) price tag, a new record for a medicine to treat a rare disease.

The sky-high cost of Glybera, from Dutch biotech firm UniQure (QURE.O) and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model.

That sounds awfully expensive to me, how do the justify the price tag?

Proponents of the gene-fixing technology insist it stacks up as a cost-effective treatment, despite the high cost, since it could permanently cure many patients.

In the case of Glybera, Chiesi said the annualized cost was no more than that charged for some expensive enzyme replacement therapies used in other rare diseases, taking into account the drug's proven benefits of at least six years.

Reuters

The justification is that these one time cures should cost the equivalent of what will be outdated procedures. They say we can treat it like a home loan that never gets paid off. A down payment and annuity payments for the rest of your life. They are afraid the insurers won't pay a million bucks up front, but payments for life are more reasonable. They say everyone comes up in the long run because it will still end up being cheaper than modern treatments.

Drugmakers contend that a one-time cure, even at a price of more than $1 million, would save money over the long term. But there are concerns that health insurers will balk at covering that kind of upfront cost.

"The place that we are moving towards is more of a pay-for-performance type of strategy," said Edward Lanphier, Sangamo's chief executive officer. Under this model, the price would be amortized over a period of time and contingent on proof that the treatment is effective and safe.

At least they say insurers can stop paying if the treatment stops working. I don't know how well that can be determined but there are much smarter people than me working on it. Insurers do have clout also.

But insurers are much less willing to foot that kind of bill for drugs used in large numbers of patients. Last year, they pressured Gilead Sciences Inc to cut the $1,000 per-pill cost of its new hepatitis C drug, saying the cost of treating more than 3 million Americans infected with the virus was unacceptable. Gilead said recently that it is discounting its prices by nearly 50 percent.

Reuters
A little push by insurance corporations and the drug maker cut the price in half. And they didn't go belly up.
Proposals have been brought forward to create a new health currency into the US health industry.

True cures in health care are rare but likely not for long. The high price tag that accompanies a cure along with its rapid uptake create challenges in the financing of cures by public and private payers. In the US, the disaggregated nature of health insurance system adds to this challenge as patients frequently churn across multiple health plans. This creates a 'free-rider' problem, where no one health plan has the incentive to invest in cure since the returns will be scattered over many health plans. Here, a new health currency is proposed as a generalized version of a social impact bond that has the potential to solve this free-rider problem, as it can be traded not only between public and private payers but also within the private sector. An ensuing debate as to whether and how to develop such a currency can serve the US health care system well.

PubMed.gov

I understand new developments in health and technology do not happen in a vacuum. Scientists and the corporations that develop medicine and cures need to be rewarded for their achievements, but I feel like they are in the wrong industry for ruthless pursuit of profits. People are born with genetic diseases at no fault of their own. They are a victim of their circumstances. If the health industry is really about health these are people that truly need it. I hate to think of people not being able to afford treatments and dying or suffering because shareholders want more money. This is a case of the treatment not being overly expensive to produce. It is about an industry more interested in profits than people's health. It is above my pay grade and I apologize if I got some of this wrong. I may even have gotten it all wrong because I just don't understand it. I don't believe the Government taking over the industry because I believe they are inept. What's the answer to making this fair all around? If these guys were putting up all their own capital it would be one thing. But to see guys like that one in the video above overly concerned about profits, when his company receives government grants pisses me off.

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that the California Institute for Regenerative Medicine (CIRM) has approved an award to the company for $9.3 million for the first round of its new Strategic Partnership Awards initiative. The award is to support a Phase 1/2 study to evaluate the safety and efficacy of LentiGlobin®, the company’s development-stage program for the treatment of beta-thalassemia, which will be initiated in the United States in 2013.

BusinessWire

originally posted by: harvestdog

The justification is that these one time cures should cost the equivalent of what will be outdated procedures.

They say we can treat it like a home loan that never gets paid off. A down payment and annuity payments for the rest of your life.

They are afraid the insurers won't pay a million bucks up front, but payments for life are more reasonable.

They say everyone comes up in the long run because it will still end up being cheaper than modern treatments.

Important to think about - and I would hope that costs would come down (I'm naieve that way especially where Big medical/pharma are concerned) with experience and improved methods but utimately - health care should be 'not-for-profit'. Nobody, anywhere should have to choose between eating and healthcare.

originally posted by: FyreByrd

originally posted by: harvestdog

The justification is that these one time cures should cost the equivalent of what will be outdated procedures.

They say we can treat it like a home loan that never gets paid off. A down payment and annuity payments for the rest of your life.

They are afraid the insurers won't pay a million bucks up front, but payments for life are more reasonable.

They say everyone comes up in the long run because it will still end up being cheaper than modern treatments.

Important to think about - and I would hope that costs would come down (I'm naieve that way especially where Big medical/pharma are concerned) with experience and improved methods but utimately - health care should be 'not-for-profit'. Nobody, anywhere should have to choose between eating and healthcare.

Even "not for profit" is not going to help here. It costs a drug company close to $1 Billion to bring a single drug to market. Even if they made ZERO profit, it still costs $1 Billion. This is because the FDA puts their feet to the fire and makes them prove--exhaustively--that a given treatment will work. No more Coca Cola as a magic tonic that cures all ills.