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From Novartis' $2.
1 million Zolgensma, to Bluebird Bio's $2.
8 million Zolgensma and $3 million Skysona, to CSL
Behring
With $3.
5 million in Hemgenix, we marvel at the ever-refreshing record for "the world's most expensive drug", but we can't help but wonder, how can such an expensive drug be sold?
On August 17, 2022, the US FDA announced that it approved Bluebird
Zynteglo, a gene therapy developed by bio to treat β-thalassemia, is priced at $2.
8 million, surpassing Novartis' previous Spinal Spinal Muscular Atrophy
Muscular atrophy, SMA), a disposable gene therapy for pediatric patients, Zolgensma priced at $2.
1 million, making it the world's most expensive drug
.
However, less than a month later, Bluebird Biologics launched its second drug for the treatment of cerebral adrenoleukodystrophy (Cerebral), a rare neurological disease in boys aged 4 to 17
ADRENOLEUKODYSTROPHY, CALD), priced at $3 million, surpassed Zynteglo as the most expensive drug
in history.
On November 22, the FDA again announced that Hemgenix, a disposable gene therapy for hemophilia B by an Australian pharmaceutical company, was approved for $3.
5 million, another price record
.
In less than half a year, the "world's most expensive drug" has changed hands
three times.
three times.
It is worth noting that it may also be reasonable to say that these sky-high drugs are gene therapy products
.
This therapy means that patients can obtain foreign normal genes, correct or compensate for diseases caused by defects and mutated genes in their own bodies, and achieve a complete cure
from the root at one time 。 In the case of Zynteglo's β-thalassemia, if not cured with gene therapy, the patient will need lifelong blood transfusion and iron removal therapy, and Bluebird estimates that the cost of treatment and related care in the patient's lifetime will exceed 6 million US dollars; For SMA patients targeted by Zolgensma, the domestic price of the life-saving drug nocinaxen sodium injection was as high as 700,000 yuan / injection, and the price in the United States was 125,000 US dollars, requiring 6 injections in the first year and 3 injections
per year thereafter.
Therefore, in the long run, gene therapy, although expensive, still has certain economic benefits
for patients.
of completely curing the root at one time.
Second, these gene therapies target rare or even ultra-rare diseases, which means that the therapies themselves are destined to have a very limited audience and fewer patients
who end up being treated 。 In the case of Zynteglo, Bluebird estimates that there are about 1,500 transfusion-dependent β-thalassemia patients in the United States, and there may be 800 to 850 of them who are healthy enough to receive Zynteglo, says Tom, chief commercial and operating officer at Bluebird Biologics
Klima estimates that about one-third of people are willing to try gene therapy and may consider Zynteglo, another third are likely to wait and see, and the rest lack demand and interest
in gene therapy.
Figure 1 The estimated number of Zynteglo patients by Bluebird Biology (Source: Bluebird Bio official website)
Gene therapy is not only a long and complex R&D investment, its manufacturing and application to patients, the cost involved is also very high
.
When a patient begins Zynteglo treatment, they first need to be Qualified
Treatment
Centers, QTCs), which are then shipped to CMO's factory for genetic modification and then back to QTC for redelivery to patients, a production process that takes 70 to 90 days
.
This has led to pharmaceutical companies having to set extremely high prices to spread to a small number of patients to ensure that the company's previous investment has paid off, and it will not be lost
.
Figure 2 Zynteglo production and delivery process (Source: Bluebird Bio official website)
But the insistence on high prices also cost the bluebird creature.
In fact, Zynteglo and Skysona were approved in the EU in May 2019 and July 2021 before their listing in the United States, respectively, but both failed to commercialize in Europe: Zynteglo announced its withdrawal from Germany in April 2021, followed by a broader withdrawal from the European market in early 2022; Skysona, on the other hand, was launched less than three months after announcing its withdrawal from the European market for business strategy reasons
Medicines Agency (EMA) and Medicines and healthcare products regulatory
Agency, MHRA) regulatory approval for Skysona is fundamentally related to
the difficulty of negotiating prices.
According to STAT
According to the report released by News, the German government of Zynteglo gave the initial price of $790,000 per patient between November 2020 and September 2022, and if it is subsequently confirmed that the patient does benefit from treatment and no longer relies on blood transfusion, then the German government will compensately increase the price to $950,000 per person.
But Bluebird Biotech is pricing Zyntegro at 1.
575 million euros ($1.
8 million), which could be paid
in installments over five years.
Andrew, the current president and CEO of Bluebird Biologics and the head of serious genetic diseases at the time
Obenshain commented that the German government has gone to great lengths in the negotiations, "not only to get a minimum price, but also to set a precedent for the pricing of all gene therapies in the future
.
" "But Bluebird Biotech has the same idea, especially when the company has plans
to open up the larger market for sickle cell disease.
" Accepting their price and setting this precedent is very detrimental to gene therapy innovation
.
Obenshain said
.
for pricing all gene therapies in the future.
Accepting their price and setting this precedent is very detrimental to gene therapy innovation
.
Figure 3 Andrew Obenshain (Source: Bluebird Bio official website)
So, after turning their focus to the US market, will Zynteglo and Skysona's fortunes take a turn?
Unlike the universal health insurance system in China and some European countries, which is managed by the government, medical insurance in the United States mainly buys medical insurance from commercial insurance
companies for employees through enterprises 。 On this basis, commercial insurance companies have also developed a drug benefit management (Pharmacy) in order to reduce expenditures
Benefit
Management, PBM) system, coordinated by third-party organizations between patients, hospitals, pharmacies, pharmaceutical manufacturers and insurance companies, reviews prescriptions issued by doctors, supervises the medical service process, and also uses its own user scale effect to form a high bargaining power for pharmaceutical manufacturers and pharmacies, helping pharmaceutical manufacturers and pharmacies open sales channels, and also greatly reducing the premium expenditure
of insurance companies.
Therefore, the life and death of Bluebird Bio in the United States will be decided
by commercial insurance companies and PBM companies.
Things seem to be going pretty well
.
When Zynteglo was first approved in August, Bluebird said it was in in-depth talks with insurers and the National Drug Benefits Manager about Zynteglo's coverage, and "has received positive feedback
from payers on pricing ideas.
" Kelima said
.
In its Q3 2022 financial results report released Nov.
7, Bluebird said that 27 patients have begun to undergo benefit verification to verify that they are covered by insurance, and about one-third of them have obtained "prior authorization.
"
authorization), which means that the insurance company agrees to cover
the cost of the medical procedure after considering factors such as the patient's age, medical necessity, and the availability of alternative drugs.
Bluebird estimates that although not all patients will receive treatment, this is a positive sign
that there are people with greater needs.
But given that patients need to complete the consent process, benefit verification and prior authorization, it is not expected that the first step in the treatment of the first patient, cell collection
, will begin until the end of this year.
The third-quarter earnings report also noted that Bluebird also received inbound requests from more than 30 unplanned adult and pediatric facilities in the process of building its QTC network, indicating that there is still an unmet treatment need
.
In addition, Bluebird Biotech has partnered with Drug Benefit Manager (Pharmacy) representing more than 40 national and regional programs
benefit managers) signed outcome-based agreements, and 12 payers have issued Medicare policies
aligned with Zynteglo's clinical evidence base.
In addition, in terms of payment methods, Bluebird Biotech has also introduced a new "invalid refund" model: if the patient's condition is not better enough to get rid of the continuous blood transfusion, then 80% of the refund will be made, and Zynteglo will be refunded
The results of the phase III trial show that 90% of patients no longer require blood
transfusions.
However, Bluebird has also made it clear that Skysona is different from Zynteglo, and the rarity and complexity of CALD make this treatment effect-based payment model difficult to implement
.
In the future, it is worth watching
how Zynteglo and Skysona's sales in the United States will evolve, and whether Bluebird Bio's sickle cell disease gene therapy can progress smoothly on this basis.
This is not only related to whether Bluebird can survive the crisis of lack of funds, but also represents the fate
that other sky-high gene therapies may face in the commercialization process.
Resources:
Resources:[1]
[1] [2]https://investor.bluebirdbio.
com/news-releases/news-release-details/bluebird-bio-reports-third-quarter-2022-financial-results-and [2]https://investor.
bluebirdbio.
com/news-releases/news-release-details/bluebird-bio-reports-third-quarter-2022-financial-results-and
[3]
[3] [4]https://db.dxy.
cn/yyzx/1775763.
html [4]https://db.
dxy.
cn/yyzx/1775763.
html
[5]
[5] [6] [6]
