Ozva AdminFor most of his life, Rob Schroeder had to take regular injections of blood clotting factor to stop uncontrolled bleeding caused by the genetic disorder hemophilia B.
But his life was transformed four years ago after he volunteered to become one of the first patients to try a gene therapy called Hemgenix.
“I have not had a bleed since I had the treatment. Any. I have indeed achieved my wildest dream goals with this therapy,” Schroeder, a pharmaceutical representative from Monclova, Ohio, told the Financial Times.
Last month, the US Food and Drug Administration approved Hemgenix, which was developed by CSL Behring and UniQure and is delivered as a single dose via intravenous infusion. It is the fifth gene therapy targeting rare diseases to receive the green light from US regulators since 2017, underscoring the potential of this new class of drugs to treat and, in some cases, cure rare diseases, cancers and other conditions.
Gene therapies, which work by adding a new gene or repairing a mutated one within the body, can drastically improve health disorders by correcting their underlying cause, usually by delivering a single treatment. But they are complex drugs that can have dangerous side effects and are sold at extremely high prices. high prices.
CSL’s Hemgenix costs $3.5 million per dose, making it the most expensive drug in the world. Skysona and Zynteglo, which are gene therapies targeting a rare childhood brain disease and an inherited blood disorder, developed by Cambridge, Massachusetts-based biotech Bluebird Bio, are the second and third most expensive drugs on the market. They are priced at $3 million and $2.8 million per dose, respectively.
Health experts warn that the costs of gene therapies could limit the willingness of public and private insurers to cover them and make them unaffordable for many patients. With more than a thousand gene and cell therapies in clinical trials, there is concern that health systems could collapse under short-term cost pressure to meet demand for treatments.
Last year, Bluebird Bio withdrew Zynteglo, a unique gene therapy for the blood disorder beta thalassemia, from the European market after failing to persuade governments to cover its $1.8 million price tag. European payers had not evolved their approach to gene therapy in a way that valued innovation, the company told analysts. He described elements of the European system as “frankly broken” and “unsustainable” for investment.
Bluebird was forced to cut costs by cutting 30 percent of its staff after its retirement in Europe and is now targeting sales in the US, where regulators approved Zynteglo in August.
Miguel Forte, president of the International Society for Cell and Gene Therapy, a group of clinicians, researchers and industry partners who support the development of these drugs, said that the withdrawal of a proven therapy was disappointing and that any repetition would be a ” serious concern.”
“I think a discussion about affordability is essential because we need to make sure these innovative medicines are available to patients and we also need to continue to learn about them and develop new products.”
In contrast, Zolgensma, Novartis’ spinal muscular atrophy gene therapy that was the most expensive in the world when it debuted in 2019, it sold well in its early years. Sales increased 36% year-on-year in 2021 as the Swiss drugmaker expanded access to Europe and emerging markets. But in the latest quarter, sales fell 15 percent, which the company blamed on a slowdown as it added new markets. Novartis said it did not believe the decline in sales was related to reports of deaths of two children who had taken the drug.
Gene therapy products can be expensive, but they provide enormous benefits to patients and healthcare systems, Forte said. The unique nature of such curative therapies improves the quality of life for rare disease sufferers and can save money in the long run by reducing or eliminating the need for medical treatment, she added.
“If we are not able to square the circle in terms of cost and reimbursement, we will not be able to provide the right opportunities for patients.”
The biopharmaceutical industry says high prices are necessary to cover the development costs of gene therapies, which at $5 billion is about five times more than traditional drugs. The markets for these drugs are also often small, and complex manufacturing and regulatory processes often lead to long lead times before treatments become available.
Investors and companies poured a record $22.7 billion into cell, gene and tissue engineered therapies last year, up from $19.9 billion the year before, according to the Alliance for Regenerative Medicine, a lobby group that represents large and small companies in the sector.
For now, sales of cell and gene therapies are relatively modest, at about $4 billion in 2021, but are projected to rise to $45 billion by 2026 as dozens more therapies are approved, according to Evaluate Pharma, a market intelligence company. But achieving this sales growth depends on the willingness of governments, insurers and other health care payers in rich countries to cover the cost of treatments.
“Our healthcare system cannot afford the price that the pharmaceutical corporations are demanding for these new drugs, no matter how revolutionary they are,” said Merith Basey, executive director of Patients for Affordable Drugs Now, an advocacy group.
“Hemgenix represents life-changing innovation for patients who can afford it, but that new innovation is worthless to those who can’t. . . At these prices, they will ultimately break the back of our ability to pay in both public and private insurance.”
Bob Lojewski, CSL’s senior vice president and general manager for North America, said Hemgenix’s $3.5 million price tag was justified because it was arguably “the most effective treatment in the world” and would generate big savings in the long run. Some discounts would also be available to health insurers and payers, he added.
“It’s a bit unfortunate that the price steals the headline from the therapy’s safety, efficacy and promise,” he said.
“When you look at the lifetime treatment of a hemophilia B patient, you’ll see costs of more than $20 million for these patients in terms of their medical care.”
A review by the Institute for Clinical and Economic Review, an independent drug watchdog, concluded that a price tag of up to $2.96 million for Hemgenix was justified due to its efficacy. He said that successfully treated patients appeared “cured” for at least a period of time, although the institute cautioned that there was still considerable uncertainty that treatment success would prove durable in the long term.
AHIP, a group representing more than 1,300 companies that provide health insurance to 200 million Americans, said its members were not opposed to paying for gene therapy drugs as long as they were shown to be effective and cost-effective.
Insurers would likely set restrictions to ensure that only those who were best suited for the treatments would have access to them and patient co-pays would be common, he added.
“In the case of the drug Hemgenix, it has a lot to offer. It’s a cure. It’s one and done. It happens to be helping a population, people with hemophilia B, who don’t have many treatment options,” said Sergio Santiviago, AHIP’s vice president of drug policy.
Santiago said insurers are exploring risk-based payment approaches to overcome the challenges of covering expensive therapies that in most cases have not proven durability over longer time frames. For example, members agreed to a coverage agreement for Zynteglo, which sees Bluebird Bio pay 80 percent of reimbursements if patients continue to require blood transfusions within two years of treatment, he said.
But Santiago said the economics of covering gene therapies become more difficult when large numbers of patients may be eligible for multimillion-dollar treatments, particularly if there is no strong evidence to support their effectiveness.
A major test is on the horizon with the expected approval next year in the US of a gene therapy targeting sickle cell disease, which has been developed by Vertex Pharmaceuticals and Crispr Therapeutics. About 100,000 Americans are living with sickle cell disease in the US, compared to only about 30,000 people with hemophilia B.
A study evaluating the impact of gene therapies in US state budgets, which was published last year in the journal Jama Pediatrics, concluded that a successful gene therapy for sickle cell disease would present affordability issues for several Medicaid insurance plans.
“While therapies with high price tags attract a lot of attention, what tends to be more important from a payer perspective is the size of the patient population,” said Patrick DeMartino, a physician specializing in the care of children with of blood and co-author of The Jama study.
“From a patient access and advocacy perspective, if there were delays in access from a limited payer, that would be a public relations nightmare. I think it would hurt people, but it would also be very bad optics for the payer.”
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