The United States Federal Drug Administration (FDA), has approved a new treatment to treat a rare blood-clotting disorder. It comes with a high price tag.
The drug is the most expensive in the world at US$3.5 million per dose.
Although it seems like a very high price, the actual cost is quite reasonable. Recent analysis of the cost-effectiveness and effectiveness of the drugThis suggests that the treatment is relatively affordable for the results it achieves – At least in the USA.
The medicine is also known as HemgenixThe gene therapy treatment for hemophilia A, which is a rare genetic disorder that causes blood clotting to be reduced, is called. The most serious symptoms are Repeated bleeding episodes and spontaneous bleedingIt is difficult to stop.
Hemophilia B is more common in men than in women. While it’s difficult to find an exact number, estimates suggestNearly 8,000 American men are living with the disease.
The US’s main medication for hemophilia B treatment is based on patients receiving the following: Clotting factor: A much-needed and important componentHowever, it is not expensive to treat for the long-term. For those suffering from severe symptoms, a regiment and costly treatment program is necessary. This can be expensive over the course of time. You will begin to fadeIt is a matter of effectiveness.
Researchers estimate that each patient with hemophilia B, moderate or severe, will have to pay an adult lifetime cost. Around US$21-$23 million. Although treatment costs in the UK may be lower than elsewhere in Europe or the US, they can still add up. Tens of Millions of DollarsEach patient during their lifetime.
Hemgenix on the other hand is a once-a-day intravenous product that costs only a fraction. The product is absorbed into the body through a Viral-based vectorIt is designed to deliver DNA to liver cells. Cells then reproduce this genetic information, which is used to spread the instructions for Factor IX, a clotting protein.
Hemgenix has been tested in two studies. Researchers found that Hemgenix was more effective than other replacement therapies in a study involving 54 patients with hemophilia B.
Patients who received gene therapy experienced a drop in the incidence of uncontrolled bleeding by more than 50 percent, compared to their baseline rate.
Side effects of the drug include headaches, flu symptoms, and elevated enzyme levels in the liver. All should be carefully monitored by doctors.
“Gene therapy is available for hemophilia for over two decades. Despite the advances in hemophilia treatment, individuals can still experience adverse effects on their quality of life. SaysPeter Marks is the Director of the FDA’s Center for Biologics Evaluation and Research.
“Today’s approval offers a new treatment option to patients with hemophilia A and is a significant step in the development of innovative therapies that will help those suffering from this severe form of hemophilia.”
Although it is not clear if this gene therapy treatment will cure hemophilia B or not, the initial results seem promising.
FDA has an FDA approved treatment plan for hemophilia B. This is a rare but serious condition. special designationTo encourage medical research. Hemgenix, for example, is an ‘orphan medication’ since it only treats a few patients.
CSL Behring (the manufacturer of Hemgenix) has for the next seven consecutive years exclusive rights to the US markets as part this designation.
Although the incentives offered by the US government to encourage scientific research are a valuable way to stimulate innovation in rare diseases, this policy is not without its risks.
It also means the US market is responsible for maintaining drug monopolies. However, other countries who place restrictions on drug price are able to reap the benefits of international drug research.
The US pays today between Two to six timesPrescription drugs are more expensive than in other countries.
The previous record holderThe most expensive drug was a ‘one-off’ form of gene therapy to treat spinal muscular atrophy. It costs approximately US$2,000,000 per course. This sparked heated debateThe way that drug companies finance their businesses.
There are many drug companies You have taken advantageThe policy could work in this case because of the use of orphan drugs over the past several decades to create drug monopolies.
Although the upfront costs can be overwhelming, Hemgenix could help you save thousands in medical expenses and improve your life in many ways.
The European Medicines Agencyand its counterparts as drug regulators The United Kingdom AustraliaWe are currently examining gene therapy as a treatment option.
It will be fascinating to see what Hemgenix prices drug makers in other parts the world allow.
