The cost of drug therapy for a person with hemophilia
Hemophilia
An inherited disorder in which the blood does not clot due to insufficient clotting factors.
What is the best way to treat 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.
What are the latest advances in the treatment of hemophilia A?
The most recent advance in the treatment of hemophilia A came with the approval of emicizumab, an engineered bispecific antibody that mimics the activity of activated factor VIII (FVIII).
Is there a solution to the hemophilia gene therapy dilemma?
This dilemma is not unique to hemophilia gene therapy, and experts are still experimenting with coverage models that will solve it. In the meantime, FDA approval of Roctavian, which had been anticipated in October of 2020, has been delayed as the FDA is requiring further research on the long-term benefits of the product.
Is a comprehensive hemophilia treatment center right for You?
Hemophilia is a complex disorder. Good quality medical care from doctors and nurses who know a lot about the disorder can help prevent some serious problems. Often the best choice is a comprehensive hemophilia treatment center (HTC).

Why is hemophilia treatment so expensive?
Expensive factor products are a result of high development and manufacturing costs, according to Caitlin Carroll, director of public affairs for PhRMA, the pharmaceutical industry lobbying group. She also pointed to advances in how these treatments are administered that will make them less costly to payers overall.
What is the average cost of hemophilia per year?
The average annual cost of clotting factor therapies for a person with severe hemophilia is roughly $300,000. Medical expenses for a person with severe hemophilia, the most common form of hemophilia, can be twice that.
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 9 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.
How much does factor 7 cost?
This is a costly medication (retail price is approximately $2000) that is currently only given to a minority of patients [1,2].
How much does Kogenate cost?
The cost for Kogenate FS intravenous powder for injection recombinant is around $11 for a supply of 1 powder for injection, depending on the pharmacy you visit....Intravenous Powder For Injection.QuantityPer unitPrice1$11.45$11.45
Is hemophilia treatment covered by insurance in India?
At present, genetic disorders such as certain cardiac conditions and haemophilia are not covered by policies.
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
How much does factor meals cost?
Comparison with competitorsFactorPrice per serving$11–$15Shippingvaries by locationMenus• Chef's Choice • Keto • Calorie Smart • Vegan & VeggieNotable features• weekly rotating menu • gluten-free facilityApr 13, 2022
Can a hemophiliac donate blood?
Because of the risk of bleeding, many blood collection centers turn away donors with hemophilia. Other centers turn away anyone who has ever received factor concentrate because of the risk of virus contamination. Maybe most importantly, you shouldn't donate blood because you need to protect your veins.
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.
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 many people will have hemophilia in 2020?
Saturday, February 1, 2020. An estimated 20,000 people in the U.S. are living with the inherited bleeding disorder hemophilia, and more than 60% of them have moderate or severe hemophilia requiring lifelong treatment with expensive drugs and clotting factors. 1. The good news is that these treatments work.
What was the life expectancy of a patient with hemophilia in the 1960s?
The good news is that these treatments work. During the 1960s, the average life expectancy for a patient with hemophilia was about 12 years; now, people diagnosed with hemophilia can anticipate a near-normal life expectancy. 2 The bad news is that successful management of hemophilia is associated with a wide variety of direct and indirect costs, ...
How much does FEIBA cost?
According to a 2018 analysis from the Institute for Clinical and Economic Review, treatment of a single bleeding episode with FEIBA can cost $50,000 or more. 6.
Is hemophilia covered by insurance?
The bulk is covered by insurers. “Because the patient does not see the full costs of the treatments, the average person with hemophilia probably does not think about this often,” Dr. Ducore said. “Whether that is a good thing or a bad thing is a philosophical issue.”.
How often should I infuse factor?
According to Dr. Croteau, instead of using factor infusions every other day or thrice weekly for prophylaxis , patients undergoing ITI will infuse factor daily or twice per day, at doses up to 4 times the dose used for routine prophylaxis, amounting to an “astronomical cost.”.
Does factor replacement stop emicizumab?
For patients with hemophilia, the costs of treatment do not stop at factor replacement or emicizumab. Those with severe hemophilia – particularly young children – are at risk for developing an inhibitor, which prevents FVIII or FIX replacement treatment from working.
Do clotting factors help with hemophilia?
At the moment, clotting factors are probably used more than any other therapy for people with hemophilia requiring treatment, according to Jonathan M. Ducore, MD, MPH, of the UC Davis Hemostasis and Thrombosis Center. The major cost of care for these patients is the factor itself, he said.
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 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 long did it take for Grehan to stop injecting himself?
Within a couple of weeks, Grehan could stop injecting himself with the clotting protein that he had previously needed. In fact, the treatment dramatically cut bleeding in all 13 of the patients who got the effective dose of gene therapy determined by the study Grehan was in.
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 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.
