Is gene therapy the future of hemophilia treatment?
Gene therapy provides a potential phenotypic cure for hemophilia, yet the cost of this novel treatment is high, tempering enthusiasm and raising questions regarding cost vs benefit.
What are the treatment options for hemophilia A?
FVIII infusions continue to be the primary treatment for hemophilia, but in November of 2017 a biological product for treating hemophilia A called Hemlibra (generic name, emicizumab) was approved by the FDA. Hemlibra acts by binding with other factors involved in the coagulation process, allowing bypass of factor VIII in that process.
Are hemophilia products covered under the pharmacy benefit?
If hemophilia products are covered under the pharmacy benefit, the medical carrier may not identify the member as being a candidate for their case management program. Payers have a lot of things to consider when addressing the high cost of hemophilia treatments. At a high level, these fall into the following categories:
How big is the market for hemophilia drugs?
The current market just for hemophilia FVIII replacement products and drugs has been estimated as close to $11 billion, and it is expected that the market will continue to expand. A major disrupter is potentially in the wings for hemophilia treatment.
What is the most effective treatment for hemophilia?
The best way to treat hemophilia is to replace the missing blood clotting factor so that the blood can clot properly. This is typically done by injecting treatment products, called clotting factor concentrates, into a person's vein.
How much does treatment for hemophilia cost?
The cost of drug therapy for a person with hemophilia can be several hundred thousand dollars per year and annual treatment costs of $1 million or more are not unheard of for patients with the most severe forms of the disease.
What is the standard treatment for hemophilia?
The main treatment for hemophilia is called replacement therapy. Concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are slowly dripped or injected into a vein. These infusions help replace the clotting factor that's missing or low.
What is on demand treatment for hemophilia?
On-demand therapy. On-demand therapy involves the use of clotting factor to treat bleeds after bleeding has started. In general, people with mild or moderate hemophilia, who tend to bleed less often, use this type of treatment.
Is hemophilia treatment covered by insurance?
The majority of people with hemophilia are covered under a commercial plan or a public plan like Medicare or Medicaid.
How much does factor IX cost?
See above for information about the drug. The per patient cost for this drug was $366,496 in 2014. This Coagulation Factor IX drug is used to control and prevent bleeding in patients with hemophilia B. The cost per patient in 2014 was $302,364.
What is the cost of factor VIII?
In that year, the price of factor VIII averaged 2.53 U.S. dollars in the United States. Factor VIII is a drug used, for example, to treat patients with hemophilia A....Average prices of factor VIII in selected countries in 2017 (in U.S. dollars)*CharacteristicAverage price in U.S. dollarsUnited States2.536 more rows
Is gene therapy available for hemophilia?
Abstract. Gene therapy offers the potential for a cure for patients with hemophilia by establishing continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a functional gene to replace the hemophilic patient's own defective gene.
Why can hemophilia be cured?
Hemophilia cannot be cured because it is a genetic disorder. This means that the problem comes from an error in a person's DNA. Since every single cell in a person's body has their own copy of DNA, every single cell has the same disorder.
How do you stop haemophilia from bleeding?
First, control the bleeding: Apply pressure with sterile gauze, a bandage, or a clean cloth. Place another bandage over the first if blood soaks through the bandage, and continue to apply pressure. Raise the injured body part to slow bleeding.
How do you inject a factor 8?
Factor VIII is generally administered as a slow IV push (bolus injection). Continuous infusion of Factor VIII is indicated for patients requiring admission for severe bleeds or surgical procedures. Factor VIII replacement for such patients should be managed in consultation with Clinical Haematology.
What medication is prescribed for hemophilia B?
Factor IX, recombinant (BeneFIX, Rixubis, Alprolix, Ixinity, Rebinyn) Recombinant factor IX (rFIX) is indicated for control and treatment of spontaneous or surgery-related bleeding or prevention of bleeding in patients proven to be deficient in FIX.
What is the best treatment for hemophilia?
Often the best choice for care is at a comprehensive hemophilia treatment center (HTC). An HTC provides patients with the care and education to address all issues related to the disorder. The team consists of physicians (hematologists or blood specialists), nurses, social workers, physical therapists, and other healthcare providers who are specialized in the care of people with bleeding disorders.
How to treat hemophilia?
Treatment of Hemophilia. The best way to treat hemophilia is to replace the missing blood clotting factor so that the blood can clot properly. This is typically done by injecting treatment products, called clotting factor concentrates, into a person’s vein. Clinicians typically prescribe treatment products for episodic care or prophylactic care.
How does hemlibra work?
Hemlibra ® works by replacing the function of factor VIII (8) , rather than replacing the missing clotting factor VIII directly. It can be used to either prevent or reduce the frequency of bleeding episodes in people with hemophilia A. This treatment product can be given by injection under the skin. Patients who use Hemlibra ® for prophylaxis and use clotting factor concentrates to treat breakthrough bleeds, can still develop an inhibitor. Traditional laboratory inhibitor testing methods do not work when testing for inhibitors in patients on Hemlibra ®; as such, a specialized testing method called the chromogenic Bethesda assay is needed.
What is HTC care?
An HTC provides patients with the care and education to address all issues related to the disorder. The team consists of physicians (hematologists or blood specialists), nurses, social workers, physical therapists, and other healthcare providers who are specialized in the care of people with bleeding disorders.
Why is cryoprecipitate not used?
However, because there is no method to kill viruses, such as HIV and hepatitis, in cryopreci pitate, it is no longer used as the current standard of treatment in the United States.
Can you give clotting factor at home?
Today, it’s possible for people with hemophilia, and their families, to learn how to give their own clotting factor treatment products at home. Giving factor treatment products at home means that bleeds can be treated quicker, resulting in less serious bleeding and fewer side effects.
How rare is hemophilia?
Hemophilia is relatively rare , with an estimated 20,000 Americans impacted, the overwhelming majority of whom are male. 2 Hemophilia is also subdivided into two main types: hemophilia A and hemophilia B. Fortunately, a broad array of medications are now available for hemophilia patients.
What is the drug that works to bond clotting factors together?
One recently approved drug, Hemlibra ® (emicizumab-kxwh), looks to sidestep the issue of inhibitors altogether. Instead of replicating a specific clotting factor, the drug works to bond existing clotting factors together to help form blood clots. 6.
Is hemophilia an inherited disease?
In terms of human and financial costs, few diseases rival the toll exacted by hemophilia, an inherited bleeding disorder. Briefly, the blood of hemophilia patients does not contain adequate amounts of the specialized proteins needed for blood clotting. As a result, they are subject to medical complications including anemia, internal bleeding, damage to muscles and joints and excessive bleeding after minor injuries. 1
Does hemophilia need clotting factor?
One longstanding issue for hem ophilia patients is the need to frequently readminister clotting factor. Accordingly, many new versions of clotting factor are formulated to offer a longer half-life of efficacy.
How much does gene therapy cost?
A 2020 story in the Wall Street Journal about gene therapy for hemophilia A mentioned a possible per-patient price of $2 million to $3 million. That would make it one of the world's most expensive drugs.
How much does factor replacement cost?
Depending on the formulation, factor replacement ranges from about $300,000 to $776,000 annually. Preventive (prophylactic) therapy requires infusions several times each week.
How much is gene therapy for hemophilia?
Gene Therapy Drug For Hemophilia May Be Priced As High As $3 Million Per Patient : Shots - Health News The first gene therapy for hemophilia could be approved by the FDA within six months, according to the drugmaker, raising hopes among families. But the drug's price could be $3 million per patient.
Who is the director of research at the Hemophilia Federation of America?
Jack Grehan. Others are more cautious. "This is really exciting, but also raises a lot of questions," says Meg Bradbury, director of research at the Hemophilia Federation of America, a patient advocacy group. One of the biggest questions is the possible cost.
How much does Biomarin cost?
Biomarin Pharmaceutical, a California company that makes what could become the first gene therapy for hemophilia, says its drug's price tag might be $3 million per patient. Biomarin Pharmaceutical, a California company that makes what could become the first gene therapy for hemophilia, says its drug's price tag might be $3 million per patient.
Is gene therapy a one time treatment?
The gene therapy would, researchers hope, be a one-time treatment that lasts a lifetime. "The context is this gigantically expensive disease to treat," says Jeff Ajer, BioMarin's executive vice president and chief commercial officer.
Is hemophilia rare?
Hemophilia is a relatively rare condition. Hemophilia A, the most common form of the disease, occurs in 1 of about every 5,000 boys. (Though girls can be born with hemophilia, too, that's much rarer .) People born with the condition are missing a crucial protein that their blood needs to clot.
Is BioMarin a breakthrough?
The Food and Drug Administration has designated the treatment a "breakthrough" therapy and accepted the company's application to give the gene therapy priority status for evaluation, representatives of BioMarin say. That makes it the first gene therapy for hemophilia the agency has agreed to consider.