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For most of his life, Rob Schroeder
Schroeder) had to be injected with clotting factors regularly to stop the uncontrolled bleeding
caused by the genetic disorder hemophilia B.
But his life changed
4 years ago when he volunteered to be one of the first patients on the experimental gene therapy Hemgenix.
"I haven't had any bleeding since I was treated
.
Nothing
.
Schroeder told the Financial Times, "I effectively achieved my wildest dream goal
with this therapy.
" ”
In November, the FDA approved Hemgenix's marketing application
.
The gene therapy, developed by CSL Behring and UniQure, is administered
as a single dose by intravenous fluids.
This is the fifth gene therapy for a rare disease since 2017 that U.
S.
regulators have given the green light, highlighting the potential
of this new class of drugs to treat and in some cases cure rare diseases, cancers and other diseases.
1 The most expensive in history, from gene therapy to gene therapy
1 The most expensive in history, from gene therapy to gene therapyGene therapy works
by adding new genes or repairing genes that are mutated in the body.
That is, it can greatly improve health by repairing the root cause of the disease, usually with a single treatment
.
But they are complex drugs, can have dangerous side effects, and sell very high
.
CSL's Hemgenix costs $3.
5 million per dose, making it the most expensive drug
in the world.
Skysona and Zynteglo are gene therapies targeting a rare childhood brain disorder and an inherited blood disorder, developed by Bluebird
Bio is the second and third most expensive drug
on the market.
They cost $3 million and $2.
8 million
per dose, respectively.
Health care experts warn that the cost of gene therapy could limit the willingness of public and private insurers to pay for those costs and keep many patients out of access to the treatments
.
With more than 1,000 gene and cell therapies in clinical trials, there are fears that health systems will collapse
under short-term cost pressures to meet treatment needs.
Last year, Bluebird
Bio withdrew Zynteglo, a one-time gene therapy
for blood disorders β thalassemia, from the European market after failing to persuade the government to pay its $1.
8 million price.
The company told analysts that payers in Europe are not pushing their gene therapies
in a way that values innovation.
Bluebird
Bio described some elements of the European payment system as "severely dysfunctional" and "unsustainable" investments
.
Bluebird
Bio was forced to cut costs after exiting the European market, cutting 30 percent of its workforce
.
Now, it is targeting sales in the United States, where regulators approved Zynteglo
in August.
Miguel Ford, president of the International Association of Cell and Gene Therapy, a group of clinicians, researchers and industry partners who support the development of these drugs
Forte said the withdrawal of a proven therapy was disappointing and any duplication would raise "serious concerns.
"
"I think the discussion about affordability is critical because we need to make sure that these innovative drugs are available to patients, and we need to continue to understand these drugs and develop new products
.
" Ford added
.
In contrast, Novartis' spinal muscular dystrophy gene therapy Zolgensma was more fortunate
.
When it was first approved in 2019, Zolgensma became the most expensive drug in the world at the time, though its early sales were good
.
In 2021, Zolgensma's sales increased 36% year-over-year as Novartis expanded access to the drug in Europe and emerging markets
.
But in the final quarter, sales fell 15 percent, which the company attributed to slowing
sales while adding new markets.
Novartis said it did not believe the decline in sales was related
to reports of deaths from 2 children who took the drug.
2 The "tug-of-war" between short-term costs and long-term benefits
2 The "tug-of-war" between short-term costs and long-term benefitsGene therapy products can be expensive, but they offer tremendous benefits
to patients and health systems.
Ford said the therapy's one-time properties improve the quality of life for patients with rare diseases and can lead to long-term savings
by reducing or eliminating the need for medical care.
"If we can't address costs and reimbursements, we won't be able to provide the right opportunities
to patients.
" Ford said
.
The biopharmaceutical industry says high prices are necessary
to cover the cost of developing gene therapies.
Typically, the development of a gene therapy requires $5 billion, about five times
that of conventional drugs.
The market for these drugs is also often small, and the complex manufacturing and regulatory processes often result in long lead times
before treatments are on the market.
The Alliance for Regenerative Medicine is a lobbying group
representing companies large and small in the field.
Last year, investors and pharmaceutical companies poured a record $22.
7 billion into cell, gene and tissue engineered therapies, compared with $19.
9 billion in the same period in 2019, according to its data.
Market intelligence firm Evaluate
Currently, sales of gene and cell therapies are relatively small, about $4 billion in 2021, but are expected to rise to $45 billion by 2026 as dozens more therapies are approved
, Pharma said.
But achieving that increase in sales depends on the willingness of governments, insurers and other medical payers in wealthy countries to pay for treatment
.
"Our health system can't pay any price — no matter how revolutionary — for these new drugs
.
" Advocacy group Patients For Affordable Drugs
Merris Bessie, Executive Director of Now
"Hemgenix represents a life-changing innovation for patients who can afford it, but this new innovation is of no value
to those who can't," Basey said.
At these prices, they will end up breaking our ability
to pay in public and private insurance.
”
However, Bob Logisky, CSL's senior vice president and general manager for North America
Lojewski said Hemgenix's $3.
5 million price is reasonable because it could be "the most effective treatment in the world" and will yield huge savings
in the long run.
He added that health insurers and payers will also receive some discounts
.
Letting the price steal the safety, efficacy and prospects of the treatment and make headlines is unfortunate in Logisky's
view.
"If you look at the lifetime treatment of a hemophilia B patient, they cost more than $200,000 in health care
.
"
3 Insurance institutions: What matters is value for money?
3 Insurance institutions: What matters is value for money?The Institute for Clinical and Economic Review is an independent drug watchdog.
Its review concluded that Hemgenix's price of up to $2.
96 million was reasonable because it was effective and successfully treated patients were "cured" at least for a while, though the agency cautioned that there was still a great deal of uncertainty
about whether the therapy's success could prove to be long-lasting.
AHIP, which represents more than 1,300 companies that provide health insurance to 200 million Americans, said its members are not averse to paying for gene therapy drugs as long as they prove effective and worthwhile
.
Insurance companies may set limits to ensure that only those who are best suited for treatment have access to those treatments, and co-payments for patients are common
.
"As far as Hemgenix is concerned, it has a lot of advantages
.
It is a one-time treatment that happens to help a group of people, namely hemophilia B patients, who don't have many treatment options
.
Sergio, vice president of drug policy at AHIP
Santiviago) said
.
Insurers are exploring risk-based payment methods to overcome the challenge
of covering expensive therapies.
In most cases, these therapies have not proven their durability
over longer time frames.
San Diego cited members as an example that members have agreed on Zynteglo's coverage agreement, which states that Bluebird would allow Bluebird if a patient continued to need blood transfusions for two years after treatment
Bio will pay 80% of the refund
.
But Santiviago also worries that when large numbers of patients qualify for multimillion-dollar treatments, especially without strong evidence to support their effectiveness, it becomes more difficult
to cover the economic benefits of gene therapy.
Next year, the U.
S.
is expected to approve a gene therapy for sickle cell disease, which is developed by Vertex Pharmaceuticals and Crispr
Developed
by Therapeutics.
In the United States, about 100,000 Americans have sickle cell disease, compared with about 30,000 people with
hemophilia.
Last year, a study published in the Journal of Pediatrics of the American Medical Association evaluated the impact
of the gene therapy on U.
S.
state budgets.
The study argues that the approval of sickle cell disease gene therapy will create affordability issues
for several Medicaid insurance plans.
Patrick DeMartino (Patrick
DeMartino, a co-author of the research paper published in the American Academy of Pediatrics, is also a physician
specializing in the care of children with blood disorders.
He believes that while expensive therapies are getting a lot of attention, what matters more is the size of
the patient population from the payer.
"From a patient usage and advocacy perspective, it would be a PR nightmare
if there were delays in usage due to payer restrictions.
I think it will hurt people, but it's also a very bad option
for the payer.
”
Resources:
References: References:
1.
Gene therapies may cure disease but can we afford them?； The Financial
Times
Gene therapies may cure disease but can we afford them?； The Financial Times
