The drug business has evolved spectacularly over the past few years, with gene therapies, cell therapies, and biologics advancing. Such treatments can be life-saving or even life-saving, but now they are among the most expensive drugs on the planet. That is because of several factors, from the new manufacturing technologies to individualised treatments and the relatively limited patient groups these therapies treat. This article covers the most expensive drugs currently on the market, explains what each one costs, how it works, and how it can affect the world of medicine.
What are the most expensive drugs?
The most expensive drugs available now tend to fall into the gene therapies and biologics class, which are the latest innovations in medicine. These are usually therapies for rare and refractory genetic diseases, promising cures where other treatments fail. But their costs are incomprehensible - hundreds of millions of dollars per treatment. Here's a list of the candidates for the world's most expensive drugs, and what a staggering amount these treatments cost.
How did these drugs come to be so expensive?
It's not a coincidence that these drugs are highly expensive to buy. The most obvious driver here is the extremely specialised manufacturing that gene and cell therapies entail. These medicines generally involve 'one-off' medication, each dose customised to a particular patient's genome. This involves cutting-edge technologies and time, which increases the price per treatment.
What's more, small patient populations for many of these treatments complicate the cost issue. As rare conditions have few people who actually have them, the expense of designing, manufacturing, and marketing the drug will have to be met from a smaller market. It is more acute in treatments for diseases such as metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD), where there are literally thousands, even hundreds of patients.
The FDA's approval process also makes these therapies so expensive. The clinical studies, trials, and safety testing that go into FDA approval cost a ton of money. Then there are the post-marketing obligations, which require further testing to establish whether these treatments work over time, making them expensive.
What are the main technologies used for these expensive drugs?
Gene therapy is at the heart of many of today's most expensive drugs, a technology that has reshaped treatment for a wide variety of genetic diseases. Gene therapies – such as Lenmeldy and Zolgensma – target damaged or replaced genes that cause disease. They usually use viruses, including adeno-associated virus (AAV), to introduce the targeted genes into the cells of the patient.
Zolgensma by Novartis, for example, uses an AAV vector to insert a functional version of the SMN1 gene into patients with spinal muscular atrophy (SMA) and thus reverse the disease. Similarly, Elevidys uses a recombinant AAV vector to transfer a micro-dystrophin transgene to patients with Duchenne muscular dystrophy (DMD), which produces a functional protein that is lacking in DMD patients.
Embryonic treatments, such as Lyfgenia and Skysona, depend on cutting-edge stem cell technology. Such therapies involve transcribing personalised stem cells to synthesize the proteins or cells that sickle-cell patients, such as those with sickle cell disease or cerebral adrenoleukodystrophy (CALD), cannot. Lyfgenia, for instance, employs lentiviral vectors to implant functional versions of the beta-globin gene into a patient's hematopoietic stem cells to treat sickle cell disease in the long term.
The burgeoning CRISPR gene editing technology in Casgevy brings an additional swagger. Casgevy uses CRISPR-Cas9 to edit the genes of sickle cell patients and beta-thalassemia patients to enhance fetal hemoglobin production and avoid blood transfusions.
What are the economic and ethical implications?
These treatments are promising for improving the lives of patients with rare and untreatable illnesses, but their cost raises major economic and ethical issues. Their costs are also a burden on health systems across the world, especially in countries where insurance doesn't fully cover the bill.
In the US, for instance, drugs such as Lenmeldy and Beqvez are fiercely scrutinised on their costs. Even as the pharmaceutical companies contend that these drugs are a cure that will lower healthcare costs over time, opponents point to the short-term financial cost they create for patients and insurance companies. Some pharmaceutical firms, including Bluebird Bio, have also suggested outcome-based pricing arrangements whereby a proportion of treatment costs is returned if the therapy fails to yield the expected benefit.
Moreover, there is a moral cost to the ridiculous cost of such drugs. Although these therapies can save lives, cost can make it difficult to access, especially in lower-income countries. This begs the question of fairness and the availability of innovative medical technologies.
How do these expensive drugs compare to traditional treatments?
Most of the time, the cost of gene therapies is weighed against the long-term costs of older therapies. For example, haemophilia B patients need factor IX infusions for life, which can run into millions of dollars over a lifetime. Gene therapies such as Beqvez, by contrast, are once-and-done cures and may replace maintenance therapy.
So too do sickle-cell patients, who were historically given a constant supply of blood and supportive treatments, which racked up enormous health care bills. Lyfgenia promises a cure by editing the patient's own cells, so there aren't regular treatments.
However, despite the long-term savings, the high initial cost of these therapies may be a barrier for many patients, especially when considering the current healthcare reimbursement landscape.
Gene and cell therapies certainly have changed the nature of medicine, providing hope to patients with rare genetic diseases. But this revolution is expensive. The world's most expensive drugs are not just unaffordable but also reflect the accelerating trade-off between innovation and access. As the field develops, it will be a matter of reconciling these treatments' miraculous efficacy with their practical costs in order to bring them to those who need them most.
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