The good news is that her child’s disease has a cure, the bad news is that it’s the most expensive medicine on the planet

This is Ayah Lundt, a Danish girl with curly hair and light brown eyes. Ayah was born in January 2020, weighing 3 kg 7 and quickly growing like any other normal child.

By the time she was 6 months old, she was already crawling and scooping up oatmeal with a spoon. Mary Mithika, Ayah’s mother, is very happy to see her daughter achieve each developmental milestone.

But in October of that year, everything suddenly reversed.

That was when Ayah was 9 months old. Mithika noticed that when her baby was lying down, she had to work very hard to raise her head. Before, Ayah liked to clap, but now she doesn’t do it often anymore.

At first, Mithika thought her son was just being less hyperactive because he was sick. But the moment Lundt dropped the spoon into the bowl of oats, her mother’s instincts told her that something very wrong was going on.

It seems, Ayah is losing all her grip.

Immediately, Mithika took her child to the hospital. Doctors ran a series of complex diagnoses just to rule out the worst-case scenario they could think of. But again, Murphy’s law is correct: “If something bad can happen, it will happen”.

Doctors said neurological exams, CT scans, magnetic resonance imaging and biopsies all pointed to a single truth: Ayah had spinal muscular atrophy (SMA), an inherited disease. Rarely affects only 1 in 10,000 newborns.

Her symptoms were the result of a series of nerve cells in her brain and spinal cord having ruptured. Nerve damage prevented Ayah’s brain from sending signals that control movement to her muscles. As a result, she can no longer raise her head, sit up on her own, or grasp objects.

Statistics show that SMA is the #1 cause of infant death among all genetic conditions. The disease progressed extremely quickly, a few months after receiving the diagnosis, Ayah had to be on a ventilator twice a day because the nerves connecting to the diaphragm had been damaged.

Every time she saw Ayah cry when she had to take a ventilator, Mithika didn’t know what to do but gently sing the song “You are my sunshine” to comfort her:

“You are the sunshine of my life, the only sunshine in the world. Even when the sky is gray outside, having you here still makes me smile. Baby, how do you know, I love you Baby, don’t go anywhere, please don’t make my sunshine disappear.”

If left untreated, SMA will attack the swallowing muscles in the neck, Ayah will sooner or later have to eat through a nasogastric tube. The prognosis after that was really bleak. Many children with SMA do not live past their second birthday.

In an effort to try to slow the death of motor neurons in Ayah’s body, Danish doctors resorted to a therapy. They injected her spinal cord with a dose of the drug that costs 150,000 USD (equivalent to 3.5 billion VND) / dose.

The good news is that her child s disease has a cure the bad news is that it s the most expensive medicine on the planet | Uncategorized

But the drug is only temporary, she will have to get a booster shot every 3 months for the rest of her life. Doctors say that if she wants to cure the disease, Ayah will need to use gene therapy.

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The good news, it’s 2020. Well, there’s an SMA gene therapy approved in the US. But the bad news, this gene therapy costs up to 2.1 million USD per dose (equivalent to 50 billion VND). In Denmark, this drug is not yet licensed and so it is of course not on the list of insurances under which Ayah is supported.

“Every day I think about that price, the number weighs on me. Then every day I watch my son’s condition get worse,” Mithika said with tears in her eyes. “As a mother, what would you do if you knew there was something out there that could save your child’s life?”

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Ayah’s drug used to treat spinal muscular atrophy (SMA) is under the brand name Onasemnogene abeparvovec. It’s a gene therapy that is being commercialized exclusively by the Swiss multinational Novartis pharmaceutical company.

Under the common name Zolgensma, each intravenous dose of the drug contains billions of viruses encased in envelopes called capsids. The viruses are given the scientific code AAV9 and belong to the family Adenoviridae. They have all been genetically modified so that they cannot cause disease in humans.

In gene therapies, viruses are often used as carriers of the genetic material package. This package is the modification and replacement of the faulty gene for the patient. Specifically in the case of Zolgensma, AAV9 will “ship” a gene called SMN1 into the cell in the patient’s nerve cell.

The SMN1 gene is the faulty gene in SMA patients. It made their neurons unable to produce SMN (motor neuron survival protein). As the cell calls it, SMN is an essential protein for cell survival. Without it, motor neurons of patients with spinal muscular atrophy will gradually die.

Now, after Zolgensma is infused into a patient’s vein, its adenoviruses seek out motor neurons and “infect” them. The virus inserts the SMN1 gene it carries into the patient’s cells, replacing the faulty mutated version of SMN1. These neurons will then be saved and maintain mobility for SMA patients.

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Clinical trials have shown that children with spinal muscular atrophy treated with Zolgensma have increased muscle strength. They then recovered significantly, were able to sit up, no longer need mechanical ventilation or feeding through a nasogastric tube.

A proportion of approximately 16.7% of SMA patients subsequently recovered to a level approaching normal development. These children were able to stand and walk without assistance – something that before Zolgensma no drug had ever been able to do.

Overall, once approved by the FDA, the effectiveness of gene therapies like Zolgensma is warranted. Their treatment is also simple, with a single intravenous dose within 60 minutes and the life of an SMA child forever changed. The only thing that matters is the price.

Approved by the US Food and Drug Administration (FDA) in 2019, Zolgensma costs up to $ 2.1 million per dose. The number surpassed the previous record of Luxturna, another gene therapy used to treat congenital blindness.

Luxturna was priced at $850,000 per dose by the company that makes it, Spark Therapeutics, and held the record for being the most expensive drug ever approved by the FDA — until Zolgensma came along for more than twice the price.

Even so, the story doesn’t take too long to repeat itself. Zolgensma only holds the record as the most expensive therapy on the planet until 2022, the price of this drug has been knocked – not once but three times.

Last August, the FDA approved Zynteglo, a gene therapy for the treatment of congenital hemolytic anemia (beta-thalassemia), for $2.8 million per dose. Less than a month later, a therapy called Skysona continued to be approved by the FDA for 3 million USD / dose (equivalent to 71 billion VND) to treat neurodegenerative disease (CALD).

In November 2022, the world record for the most expensive drug was set by Hemgenix, a gene therapy used to treat hemophilia B. CSL Behring, the Australian pharmaceutical company that sells the drug exclusively, has announced Valuing each intravenous dose of Hemgenix up to 3.5 million USD (equivalent to 86 billion VND).

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What all these drugs have in common is that they are all advanced gene therapies. Only commercialized in the last 10 years or so, gene editing is still at the dawn of medicine. This means that research and production costs are extremely expensive.

Gene engineering requires sophisticated infrastructure and machines, highly trained staff, expensive raw materials, and complex biomaterials. To make a single dose of gene drug, it takes companies 9-10 months to grow the virus, assemble the gene vectors, encapsulate them in a capsid, purify the final dose before packaging and distribution. distribute.

The prices of gene therapies are therefore in the range of several hundred to several million dollars, with the average figure being around $1.5 million.equivalent to 36 billion VND.

The second thing the most expensive drugs on the planet have in common is that they all require a single dose. Single-dose gene therapies aim to treat the root cause of the disease, by replacing drugs that patients have to take for life (with a total cost that is many times more expensive).

Take the example of Hemgenix, the most expensive drug on the planet at the moment with a price of 3.5 million USD used to treat hemophilia B. The patient has Hemophilia B due to a defect in the F9 gene that causes hemophilia B. they can’t make a protein needed for blood clotting called clotting factor IX.

On the one hand, this causes the patient’s open wounds to bleed longer, resulting in more blood loss. On the other hand, it creates a risk of bleeding under the skin (appearing as bruises), bleeding in the joints (leading to osteoarthritis) and bleeding in the brain (causing frequent headaches, triggering the convulsions and decreased consciousness).

Prior to the introduction of Hemgenix, patients with Hemophilia B would have to be prophylactically administered factor IX 2-4 times/month, isolated from donor sera, or using recombinant protein.

A study in the Journal of Medical Economics estimated that this treatment cost could add up to 23 million USD, equivalent to 552 billion VND if people with hemophilia B want to maintain maximum life expectancy. This “maximum” is the average age of a healthy person minus 10 years.

Rarely do hemophilia B patients live longer than that. And conversely, without treatment, most children with this disease do not live to adulthood. Many patients will die before the age of 11.

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Hemgenix, now emerges as a new and groundbreaking solution for patients with Hemophilia B. It is a single-dose therapy, meaning that patients need only a single infusion of Hemgenix to manage their condition, compared to a single dose of Hemgenix to manage their condition. having to infuse factor IX several times a month for the rest of his life.

As a Zolgensma-like gene-targeted therapy, each infusion of Hemgenix contains billions of adenoviruses, tuned to carry copies of the F9 gene (a gene that produces clotting factor IX).

Once the virus reaches the liver, it transfers the gene into the cells, allowing the liver of a person with hemophilia B to start producing clotting factor IX naturally. Basically, this is a method of fixing the root of the problem.

So at $3.5 million a single dose versus $23 million for a lifetime course, Hemgenix still seems like a bargain.

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Back to spinal muscular atrophy (SMA) that little Ayah is suffering from. To prolong her life and slow nerve cell death, doctors in Denmark are using a drug delivered directly to her spinal cord.

The drug, called Spinraza, was approved by the FDA in 2016. And since then, it’s been the only treatment for SMA patients. After being injected into the patient’s spinal cord, Spinraza will work to stimulate the SMN2 gene responsible for producing SMN protein (instead of the faulty SMN1 gene), thereby saving the motor neurons of the patient. patient.

However, the disadvantage of Spinraza is that it has a half-life ranging from 63-87 days in plasma and 235-177 days in cerebrospinal fluid. This means that the patient will have to receive medication every 3 months. Treatment costs are estimated at up to $750,000 for the first year and $350,000 for each subsequent year.

That means that Zolgensma’s $2.1 million price tag is now equivalent to only 5 years of treatment with Spinraza. Meanwhile, the first children treated with Zolgensma in clinical trials were predicted to survive 5.2 years with normal developmental milestones. The effect of this gene therapy is expected to last many times, even a lifetime.

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Novartis used the comparison above to provide a price for Zolgensma. “Other treatments and care for SMA patients can cost up to $6 million for the first 10 years,” said Dave Lennon, president of Novartis Gene Therapies.

“But here, we’re talking about the patient’s lifetime benefit condensed into a single therapeutic dose. People aren’t used to thinking that way. We’re used to a chronic drug system, where we spread treatments for years if not decades.”

From his perspective, Lennon considers Zolgensma’s price to be “reasonable” and “highly cost-effective”.

On the one hand, it provides a well-deserved benefit to the patient over previous treatments. On the other hand, this price would allow Novartis to recoup the money it invested in Zolgensma research – which Lennon said would then be used to reinvest in new, targeted gene therapies. future genetic diseases.

According to a study in the Journal of Health Economics, it takes pharmaceutical companies an average of 12 years to develop a drug from preclinical testing to approval and commercialization.

This R&D process costs an average of $2.6 billion, including opportunity costs and high failure rates, up to 88-90%. This means that only 1 out of every 10 drugs developed is sold and makes a profit for pharmaceutical companies.

Therefore, before deciding to invest in the development of a new drug that they themselves are not sure will succeed, any pharmaceutical company will have to look at their potential customer file. For drugs, it is the number of patients who are suffering from the same disease.

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Profitability is calculated based on the financial readiness of the patient, the price of the drug, the ability of the insurance company to pay multiplied by the number of patients in the population. Ideally, pharmaceutical companies want to develop a drug that is moderately priced, but for a large customer base who can afford it and take it regularly.

For example, Adalimumad, a drug for rheumatoid arthritis, sells for about 15 million VND/box. Adalimumad is always at the top of the list of the most profitable drugs for pharmaceutical companies, because at all times about 1% of the population has rheumatoid arthritis and can afford it.

In contrast, pharmaceutical companies will do their best to avoid developing drugs for a disease with a small customer base. On the one hand, they don’t want to risk billions of dollars in R&D costs when they don’t see the potential for profitability.

On the other hand, even if the drug is successfully developed, the small customer base will cause pharmaceutical companies to push up drug prices, to hundreds of thousands to millions of dollars without insurance companies and insurance companies. can the patient pay?

As a result, patients with rare diseases are often “abandoned”, with no cure. The medical industry uses the term Orphan diseases to describe this bleak valley of the pharmaceutical market.

In the United States, an orphan disease is defined as one that affects less than 200,000 people, equivalent to an incidence of 6 in 10,000. In Europe, that number is 5/10,000.

There are about 7,000 orphan diseases that satisfy these numbers, including spinal muscular atrophy (SMA) that little Ayah is suffering from. But there are only about 300 orphan drugs currently on the market.

A study in the European Journal of Human Genetics estimates that orphan diseases affect 3.5-5.9% of the world’s population, or 300 million people at any given time.

Governments, of course, know what they need to do to fill the void and keep these patients from being left behind. The effort is realized by tax incentives or financial subsidies for pharmaceutical companies with orphan drug development programs.

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For example, the US Orphan Drugs Act (ODA) provides credits for up to 50% of R&D costs, along with grants, protocol support, and FDA waivers for pharmaceutical companies to develop generic drugs. this drug.

In addition, they will have exclusive rights to distribute them in the US for 7 years. Essentially, this gives drug manufacturers the power to price their products at whatever threshold they want.

While not doing it too openly, this is still the main reason why the prices of orphan drugs are so high, and they even tend to increase as the old record prices are constantly pushed. to pour.

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So, preferential policies for orphan drugs only solve half of the problem. On the efficiency side, they were compelling enough to get pharmaceutical companies to launch their for-profit science machines.

Statistics show that, since the US Orphan Drug Act (ODA) went into effect, it has attracted more than 200 companies to participate in the research, contributing to bringing 450 orphan drugs to market. These orphan diseases have gone from having no cure to having a cure.

But on the downside, preferential policies remove barriers to research motivation – it creates a new barrier that follows immediately: Drug prices. The flow of benefits to hundreds of millions of orphaned patients has thus yet to be unleashed.

A study published in the journal Plos ONE surveying access to orphan drugs in 35 countries around the world found that: With treatment costing up to millions of dollars, most patients will not be able to access it. with your own treatment, unless the drug is covered by insurance.

Regrettably, most insurers would approach orphan drugs very cautiously. For example, a survey by the US research and brokerage firm Bernstein found that only 11/30 insurance companies in the US included Zolgensma in their payout portfolio.

Even if they do, the criteria for patients to get financial assistance are very strict. For example, Anthem, one of the largest private insurance companies in the United States, stipulates that only children under 6 months of age can use Zolgensma under insurance.

Other companies such as Horizon Blue Cross will not pay Zolgensma if a patient has been treated with Spinraza. Nine of the nine insurers in Bernstein’s survey will cover Zolgensma but refuse to cover Spinraza at the same time.

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According to the specific criteria above, SMA patients like Ayah Lundt will still be abandoned. Ayah received the diagnosis at 10 months, too late when her parents discovered the symptoms, and she had also been treated with Spinraza before.

Without insurance, the financial burden of up to $2.1 million is now pushed entirely on the patient’s family. In an almost desperate situation, Mithika could only think of one way: Ask for help from the community.

That November, she and her husband assembled a core group of eight family members and a circle of friends. They started fundraising campaigns on Instagram and Facebook.

Some people will take photos and videos, others write articles to share Ayah’s story. Some volunteers will be responsible for translating documents into multiple languages. Mithika also did not forget to write to the government, sending emails to all the potential companies and charities she knows to call for help.

On Easter weekend that year, the volunteer group held a 36-hour Instagram livestream. During those 36 hours, Mithika did not sleep in exchange for a $70,000 donation to Ayah.

Even so, all know the numbers they achieve are still just the surface – compared to the price of a dose of Zolgensma. Even more difficult is that donations in Denmark are still dwindling. Mithika then had to ask a colleague to help isolate her GoFundMe account in the US.

GoFundMe is a crowdfunding site, where hundreds of thousands of families are also looking for financial help for their children, when the children suffer from a serious illness with expensive treatment costs.

That means they always have to compete with the limited generosity of the community. The problem is that medical treatment-related fundraising campaigns on GoFundMe only have an average goal of about $8,000. Even so, only 12% of the campaigns achieved success, 16% did not even receive a single penny of donation.

So with the goal of raising more than 2 million USD, Ayah’s story is a breakthrough that more or less moved the community. Four months of appearing on GoFundMe brought in an additional $60,000 for her. Even so, everyone knows that number is still too small and only a miracle can make a difference.

Fortunately, that miracle finally came one afternoon in March 2021, after CNN published a story about little Ayah and the illness she was suffering from. The virality of the article caused Ayah’s GoFundMe fund to skyrocket to more than 1 million USD overnight – literally.

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“We couldn’t sleep,” Mithika recalls. “We scrolled through the GoFundMe donation directory, updating every minute until morning.”

The story of little Ayah, who is fighting the evil SMA disease, and the huge cost of treatment of one of the most expensive orphan drugs on the planet has moved the international community.

A series of other fundraising campaigns for the little girl have continued to be carried out in Denmark, Kenya (Mithika’s hometown) and the United States – where Ayah’s family plans to go for treatment because Zolgensma has not been licensed. in Denmark).

It took another four months for influencers like musician King Kaka, actress Teresa Palmer or Liverpool football player Divock Origi to join the campaign, and Ayah and her family finally raised enough money that they needed, including $2.1 million for the drug Zolgensma and $300,000 for travel and accommodation expenses in the United States during her treatment.

In early September 2021, Ayah’s family said they traveled more than 8,500 km from Copehagen to San Antonio, Texas to prepare for the procedure with Zolgensma. The girl was examined, tested and monitored at the City Children’s Hospital, more than 10 days before officially receiving a dose of Zolgensma.

That was when Ayah was 20 months and 5 days old, 4 months before the deadline when she could receive the drug (Zolgensma has not been licensed for children over 2. If this time is exceeded, Ayah will have to come back. treatment with Spinraza).

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The story of little Ayah has a happy ending. Although all donations have been closed, Mithika’s family still maintains Ayah’s Instagram and Facebook accounts to update her on her health.

After more than 1 year of treatment, Zolgensma gave a very positive prognosis. 3-year-old Ayah is now able to stand without support. She can crawl, play with toys, roll over in bed, raise her head, turn her face and become a “mouth-watering machine”, a sign that the nerve cells that control the larynx have been saved.

All of this wouldn’t have come if Ayah hadn’t received a $2.1 million dose of the drug. “We will forever be grateful to each and every one of you who helped Ayah find her genes. God bless you all,” the fundraiser wrote.

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It’s not hard to see, crowdfunding is a common strategy used by families with orphaned children, when they can’t afford to access expensive drugs.

The evidence is shown in a study in the journal Lancet. The authors estimated that from the time Zolgensma was licensed in May 2019 until May 2021, there were at least 171 GoFundMe fundraising campaigns for SMA patients who needed treatment with this drug. The total target amount that these campaigns set is more than 255 million USD.

Among them, only 13 successful fundraising campaigns and 33 campaigns surpassed 50% of the amount to be raised. An article in the New York Times also said: Since Zolgensma was approved, only about 10 pediatric patients have raised enough $ 2.1 million from community funds to cover medical costs.

Newspapers can tell a lot of success stories, giving hope to children. But they say little about what’s left under the ice. For example, a 7-month-old girl in India died last year, raising just over $50,000 out of $2.1 million for Zolgensma.

A boy in the UK also missed the opportunity to receive the drug despite raising up to $ 900,000. The money was then transferred to the family of another SMA child, who is also crowdfunding on GoFundMe.

The problem for pediatric patients with SMA is that these children and their families don’t have much time. Zolgensma must be used as soon as possible and is currently only prescribed for children under 2 years of age. If families are unable to mobilizeIf you have enough money before your child’s second birthday, kids will really miss out on a precious treatment window.

Novartis said to solve this problem, they have allowed Zolgensma to be paid in installments for 5 years. That means the first payment will only stop at $425,000 (equivalent to VND 9.97 billion).

However, that is still a high price and the families of the children will have to work with their insurance company or payment assistance program to be able to pay Zolgensma installments under that route.

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In another attempt to get to the bottom of the problem, Novartis is committed to giving away 100 free doses of Zolgensma each year to pediatric SMA patients around the world. This is part of the “Global Zolgensma Accessibility Disassembly Program”, as they call it.

The company’s website, which updated the program’s results in January 2023, says that nearly 300 pediatric patients in 36 countries around the world have received Zolgensma for free since it was launched in 2020.

The number is very modest compared to the 14,000 babies born with a new diagnosis of SMA each year. That led Mike Fraser, general manager of Novartis Gene Therapies to admit: “We would love to give this drug to all patients. But we really can’t.”

In fact, Novartis’s free medicine program has also encountered many criticisms, mainly because of their approach. First, the company’s only rollout of this activity in countries that have not yet licensed Zolgensma makes it more of a trial advertising program than charity.

In particular, as soon as that country licenses Zolgensma, the free drug donation program will stop.

In addition, because the number of patients exceeded the number of Zolgensma doses allotted, Novartis chose a random lottery to select the lucky children. Activists criticize this practice for creating an emotional burden on children and their families.

If they are not the chosen one, they will feel down. Some will even have low self-esteem because they think they don’t deserve to be chosen to live. Not to mention the transparency and fairness of the draw that Novartis organizes in each country is also called into question.

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Kevin Doxzen, a biotech and policy analyst at Arizona State University, USA, said that to solve the problem of the high cost of gene therapies, we need a two-way approach. .

“In the short term, it will be important to develop new payment methods, to entice insurance companies to pay for high-cost therapies. This will help spread the risk between the patient, the public, and the public. In the long run, as gene technology improves, the cost of therapies will certainly drop,” Doxzen wrote in a commentary published on the World Economic Forum. .

An innovative payment model proposed by Doxzen based on treatment outcomes. Accordingly, the insurance company can pay part of the cost for the therapy upfront, and pay the rest after the patient has recovered or improved. Conversely, if the patient does not get better, the pharmaceutical company needs to reimburse the insurance company.

Governments can also bargain with pharmaceutical companies to lower the cost of gene therapy, in exchange for including it in national insurance schemes. This is the strategy that Japan has used with Zolgensma and most recently has been applied by Korea.

Accordingly, the ceiling price of Zolgensma is set at about 1.5 million instead of 2.1 million. In South Korea, SMA patients who are eligible for treatment and have insurance can pay as little as 5.98 million won ($4,600) for the therapy.

Calculations show that the inclusion of Zolgensma in the national health insurance program will not break the health budgets of countries, because SMA is a rare disease and the number of patients is small.

However, that would create pressure on social justice, that if Zolgensma is covered by state insurance, drugs for other orphan diseases must also be included in the program. This ensures that not only SMA patients but also other orphaned patients are not left behind.

This is the main bottleneck causing the risk of deficit, even rupture of insurance funds even in high-income countries and the world’s best source of health benefits. Theoretically, the same technology that Novartis is using to deliver the drug Zolgensma, they could create 100 gene therapies to cure 100 different diseases in the next 10 years.

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“There are 1,745 gene therapies under development around the world. Much of this research focuses on a rare genetic disease that affects 400 million people globally. We could soon see cures for it. treat rare diseases such as sickle cell disease, muscular dystrophy and geriatric disease, a rare and progressive genetic disorder that causes children to age rapidly,” Doxzen wrote.

If all of these therapies were valued at $2 million, no country would be able to include them all in health insurance. Therefore, the fact that some countries include each super expensive drug like Zolgensma into national insurance programs is still only a way to solve the problem.

This is where Doxzen proposes a solution called the “Netflix model,” in which the government insurance program will pay a fixed amount upfront to drug companies in return for the right to continue Access an unlimited package of advanced therapies these companies are developing.

In addition, he said, we need to invest in new gene technologies to accelerate the simplification, reducing the cost of these therapies to levels accessible to the majority of patients. However, this process will take time, not only in years but possibly decades.

Until then, every year to come we will still see new orphan drugs come to market. They break price records to become the most expensive therapy on the planet. They give patients hope, but at the same time build a wall of despair, preventing them from accessing the therapy claimed to be for them.

Orphaned patients will continue to be “orphaned,” even if they’re living in an era where there’s no cure for their disease – just not enough money to buy them.

Refer to Nature, CNN, Weforum, Technologyreview, BMC, Lancet, NIH

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