Products for the Treatment of Factor VIII Deficiency

Transcription

1 Products for the Treatment of Factor VIII Deficiency Conference Call August 24, pm-8pm Presenters: Marcus E. Carr, MD, PhD & Gita V. Massey, MD I. Overview of Factor VIII products currently on the market Advate (Baxter) Helixate FS (ZLB Behring) Kogenate FS (Bayer) ReFacto (Wyeth) Recombinate (Baxter) II. Thoughts to consider when choosing a factor product Cost Infusion III. Question and answer time This program is brought to you to with support from Hemophilia Health Services, a specialty homecare pharmacy that serves the bleeding disorders community.

2 Factor VIII Products Comparison Chart Advate Helixate FS Kogenate FS ReFacto Recombinate (Third generation) (Second generation) (Second generation) (Second generation) (First generation) Distributor Baxter Bioscience ZLB Behring Bayer Corporation *86% good/excellent *80-90% excellent/good *80-90% excellent/ good Efficacy response rate (100% in response rate (100% for response rate (100% for surgeries) surgeries) surgeries) Wyeth *92% excellent/good response rate Baxter Bioscience *92-95% excellent/good response rate Studies *No PUP studies *PUP and PTP *PUP and PTP *PUP and PTP *PUP and PTP Half-Life / hours /- 1.6 hours /- 1.6 hours /-5.3 hours /- 4.9 hours Inhibitors No PUP studies; no inhibitors in patients under 6 years 16% in PUP studies 16% in PUP studies 30% in PUP studies 32% in PUP studies Safety MAIC** Exposure to Human Albumin Vial Ranges None; uses a sugar (trehalose) as stabilizer 250, 500, 1000, 1500 IU MAIC** and solvent detergent MAIC** and solvent detergent Uses albumin in Uses albumin in fermentation; no albumin in fermentation; no albumin final form; sugar (sucrose) in final form; sugar as stabilizer (sucrose) as stabilizer MAIC** Uses albumin in fermentation; no albumin in final form; sugar (sucrose) as stabilizer 250, 500, 1000 IU 250, 500, 1000 IU 250, 500, , 2000 IU MAIC** Uses human albumin and bovine protein as stabilizers 250, 500, 1000 IU Reconstituted Volume 5 ml 2.5 ml 2.5 ml 4 ml 10 ml Storage Refrigeration or room temp for 6 months Peel off label Refrigeration Refrigeration Refrigeration or Refrigeration or room temp for 6 months room temp Peel off label Peel off label Peel off label Other Features Butterfly needle included Letter coded packaging Butterfly needle included Color coded packaging Butterfly needle included Color coded packaging Butterfly needle included Color coded packaging Color coded packaging * PUP- Previously Untreated Patients; PTP- Previously Treated Patients ** MAIC- Monoclonal antibody immunoaffinity chromography

3 CONSIDERATIONS WHEN CHOOSING A FACTOR PRODUCT: COST I. Cost of factor to the family is determined by several factors: 1. Co-pays dictated by insurance a. Some carriers require a percent of cost co-pay to the family for every order of factor concentrate; b. More expensive products would therefore lead to higher costs for families; c. Some home infusion companies will consider financial waivers for co-pays or will work with a third party to waive or reduce co-pays. 2. Insurance formularies a. Some insurance companies cover only certain medications on their routine medication list. Those medications not on the list may be more expensive or not covered. b. Some insurance companies require special authorization for more expensive medications and may limit the amount of medication available at a time. 3. Insurance caps a. Some insurance carriers have a ceiling or maximum amount that they will pay for a patient for their lifetime. Lifetime caps can be expended more quickly for more expensive medications. II. Cost of factor to the insurance company is determined by several factors: 1. Average wholesale price of the medication a. Affected by manufacturer s pricing 2. Contractual agreements between home infusion companies and insurance companies a. Based on size of company, partnerships with manufacturer etc

4 Questions to ask your home infusion company when selecting a factor product: 1. What is the cost per unit charged to your insurance company of the factor concentrate that you are currently using? 2. What is the cost per unit of the factor concentrate that you are considering? Recall that different home infusion companies may charge different prices for the same product. However, some companies will charge less for a unit of factor, but bill for every ancillary supply (needles, syringes, port supplies, Sharps container) and for nursing services not covered by insurance. 3. Can your home care company obtain the factor concentrate that you desire in the assays that you need? Remember that using assays (number of units in a vial) that are much larger than what you need can dramatically increase the cost of the infusion of factor concentrate. For example, if your prescribed dose is 750 units, but you can only get 900 unit vials, you are being charged for the extra 150 units of factor that you may not need. Questions to ask your insurance company when selecting a factor product: 1. Do you have a lifetime cap on your insurance policy? If so, how much have you used toward the cap? Many insurance policies have a lifetime cap (limit) for payments, meaning there is a maximum amount the policy will pay for an individual. Once the policy has reached this limit, the patient caps out, meaning their insurance company will no longer pay for coverage. 2. Which home infusion companies are covered on your policy? Insurers contract with home infusion companies to provide factor. Some insurance policies may allow patients to choose between several companies, while others may have only one designated home infusion company.

5 CONSIDERATIONS WHEN CHOOSING A FACTOR PRODUCT: INFUSION 1. Volume of the Factor VIII to be Infused: Different factor VIII products offer the same dose in different volumes to infuse. It may be easier to infuse a smaller volume. If the factor VIII product you are infusing is one that is a smaller volume, such as 2.5cc, it is very important to make sure the entire product is infused, leaving none of the medicine in the butterfly tubing. To insure this, you must flush the tubing with a small amount of saline into the vein, after infusing the factor. 2. Butterfly Infusion Sets: For convenience, all of the factor VIII products come packaged with a butterfly infusion set, except for Recombinate. For this product, infusion sets can be sent from the home care company separately. 3. Type of Diluent Mixed with Factor VIII Product: Refacto brand of factor VIII uses saline as its diluent to mix with the powdered factor. The other brands we mentioned in the table use sterile water. It is important to use the correct diluent when mixing factor to be infused.. 4. Peel Off Label: All of the factor VIII products mentioned, except for Refacto, have a peel off label on the bottle with the lot number and expiration date. This makes it convenient to put infusion information in a log. All of the Factor VIII products mentioned should be infused within three hours of reconstituting (mixing the diluent with the factor powder).

6 PARTICIPANT Q&A: Question: Is there an increased risk of inhibitor formation as you switch from product to product? Should this limit one s participation in the free trial offerings from companies/distributors? Answer: There is very little scientific data that supports the theory that switching products leads to development of inhibitors. There have been several anecdotal reports of this occurring, but so far there is no evidence to show this is related to switching products. It was noted that patients are frequently switched in emergency situations (hospital may have different product) as well as during the factor shortage a few years ago. Factor sample programs require a prescprition from a physician. Concerns about changing products should be reviewed on an individual basis between physician and patient Question: Is there increased risk of impotence with Advate? (Participant was told by two friends that they developed difficulty with erections while on the Advate trial, and this problem went away when they returned to their former products). Answer: This is not listed as a side effect on the prescribing insert for Advate. As a follow up, HTC staff presented this question to Baxter representatives who also were not aware of this side effect. Patients should call Baxter medical affairs department to inquire further or to report side effects. Question: What is the closest agent to Recombinate? Is it Refacto? Answer: Refacto is the least similar to Recombinate. Kogenate FS and Helixate FS are probably the most similar to Recombinate. Question: Is Recombinate going away? Answer: Baxter has not made any official announcements about discontinuing Recombinate. It is expected that distribution of Recombinate will eventually be reduced or eliminated, as Baxter has put time and resources into developing a 3 rd generation product, Advate. When/if this happens, Baxter is expected to provide notice to patients well in advance so they can consider other options.

7 PRODUCT INFORMATION FROM MANUFACTURERS Please visit the manufacturer websites for more information about factor products and services: Baxter (Advate & Recombinate) Bayer (Kogenate FS) Wyeth (ReFacto) ZLB Behring (Helixate FS)

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