Hemophilia Advocates Take Their Case to Georgia Medicaid Meeting

“Different hemophilia drugs work differently in different individuals. I wish everyone’s ‘hemophilia engine’ ran pretty much the same. It would be great if you could just fill us all up with the least expensive version of regular unleaded and change our oil every 5,000 miles to ensure peak clotting performance. It just doesn’t work that way.”

That analogy was part of Chip Hearn’s testimony to Georgia’s Drug Utilization Review Board (DURB) on May 7. Hearn and Jeff Cornett, Hemophilia of Georgia’s (HoG) Vice President of Research and Public Policy, testified before the board about the need for Medicaid patients to have access to all hemophilia drugs.

DURB is part of Georgia’s Medicaid program within the Georgia Department of Community Health.  The members of DURB are pharmacists and physicians who advise the state on how drugs should be made available to patients enrolled in Medicaid. A drug may be “preferred” or “non-preferred,” require “prior approval” from Medicaid before the patient can get it or have a requirement for a doctor to show that cheaper drugs don’t work before receiving it. The Georgia “preferred drug list” for hemophilia changes frequently. HoG is concerned that the changes are based on drug prices and make it difficult for doctors to find the right factor product for the patient.

DURB meets at least four times a year, usually in Atlanta.  At least some portion of each meeting is open to the public, with limited time for comments. If you would like to get involved in Hemophilia of Georgia’s advocacy efforts, including efforts to make Medicaid better serve those with bleeding disorders, let us know.

Below are the full testimonies from Hearn and Cornett.

Chip Hearn’s Testimony

My name is Chip Hearn, and I am a 52-year-old Medicaid recipient from the Atlanta area. From the time of my birth in 1966, I have faced the challenges of living with severe hemophilia A – a deficiency in blood clotting protein number 8 which, over the course of my lifetime, has caused many frequent and destructive painful bleeding episodes into my muscles and joint spaces. These bleeds have resulted in significant hemarthrosis and joint damage to my knees, elbows, left shoulder and ankles and that joint damage greatly restricts my range of motion and ability to walk.

I am here this morning to share just a bit of my personal story with you because I feel very strongly that the treatment decisions I have been allowed to make in concert with my doctors and healthcare providers, the treatment options to which I have had access, have definitely impacted the course of my disease. Being able to utilize the hemophilia therapies that worked best for me has minimized the impact of my bleeding disorder and greatly improved my quality of life.

While the physical manifestations of hemophilia and the challenges they impose on a daily basis are significant and life changing, I consider myself very fortunate. As with approximately two-thirds of those with hemophilia, I have a family history of the disease. That is not to say I ever knew anyone in my family with hemophilia. My grandfather passed in his early 40s from internal bleeding. He had two brothers with hemophilia, and they both died as a result of bleeding complications as well. One was only 20-something years old and died after a relatively minor auto accident. The other was approximately 10 years old and got hit in the head with a baseball. All three lived in a very rural part of Georgia outside Macon and died at a time when there was very little understanding of bleeding disorders and practically nothing in the way of treatment.

I, however, was born at about the same time as a new advance in hemophilia treatment was discovered: factor replacement therapy. Researchers had discovered and isolated the blood clotting protein I was missing. Beginning in the 1970s, pharmaceutical companies were able to prepare this agent as a medication – a medication I could keep at home and administer whenever I first noticed the telltale signs of bleeding into a joint: pain, swelling and restricted range of motion. This advance in treatment promised greater autonomy and a more regular life for those with hemophilia – freedom from constant emergency room visits and painful joint bleeds that kept us in bed for days or even weeks at a time. The comfort, happiness and autonomy that accompanied plasma-derived clotting factor were, unfortunately, short-lived. By the 1980s, we realized that these medicines prepared from untreated pools of human plasma harvested from thousands of donors contained deadly and previously unknown viruses including HIV and hepatitis C. 

Estimates indicate that almost 70 percent of those with hemophilia who shared my severe diagnosis was lost to viral contaminants. I don’t think of being 52 as particularly old, but in the hemophilia community, it is apparently old enough to earn me “elder statesman” status. So obviously for me, just to be here today having survived severe hemophilia and the complications of its treatment is one reason I feel so fortunate.

But in reflecting on my life with hemophilia and summarizing it for my testimony this morning, it is the other ways in which I have been truly blessed and fortunate that I would ask you to take note. It’s challenging for me to cover 52 years of hemophilia experience in approximately 180 seconds. But if you are to remember anything from my testimony this morning, please consider and reflect upon the following three points:

Firstly, different hemophilia drugs work differently in different individuals. I wish everyone’s “hemophilia engine” ran pretty much the same. It would be great if you could just fill us all up with the least expensive version of regular unleaded and change our oil every 5,000 miles to ensure peak clotting performance. It just doesn’t work that way. The pharmacokinetics and the pharmacodynamics of our hemophilia medicines vary from individual to individual. Personalized treatment plans that take into account the experiences and eccentricities of each individual patient’s clotting cascade work best.

Second, the fact that individualized treatment plans developed in close cooperation between the hemophilia patient and his or her treatment physician isn’t just theoretical to me, it is practical. It is something that I have seen with my own eyes and experienced in my own life. I have lived with and lived through not only severe hemophilia, but all of its most significant complications: port infections, advanced joint disease, viral contamination of the early hemophilia meds and inhibitors (which are a sort of “allergy” to particular hemophilia drugs whereby the body creates an antibody to the foreign clotting proteins). I feel that the reasons I have lived this long and done this well are the grace of God, the love and support of my friends and family, and the expertise and professionalism of Georgia’s hemophilia treatment community, namely Hemophilia of Georgia and my long-time physicians at Emory in both the hemophilia treatment center (HTC) and the infectious disease clinic. Given the opportunity to use the full range of FDA-approved medications for both hemophilia and HIV, my doctors and I have worked through the many challenges together to successfully reach an extended period of comfort and stability in my life. Together we have cured my hepatitis C, held my HIV to undetectable levels for almost 25 years and, using twice-weekly injections of an extended half-life hemophilia factor, practically eliminated my joint bleeds. I have not required an inpatient hospital stay or needed to visit an emergency room in 13 years. Georgia is fortunate to have many dedicated hemophilia experts practicing here. Listen to them, lean on their expertise and respect their medical advice.

And third, lastly, I understand that the price of these anti-hemophilic drugs can be high. But people with hemophilia are relatively small in number. Please do not let costs be the primary consideration in prescribing a hemophilia therapy. Allow physicians to determine the best course of treatment for their patients without too many burdensome constraints or time-consuming prior authorization processes. When treatment decisions are left in the hands of doctors and their patients, when doctors and their patients with bleeding disorders can freely and easily choose from all approved anti-hemophilic drugs, I know from personal experience that you will see the best outcomes. I believe that today you are considering adding a new hemophilia medication to the preferred formulary. Just know that while this new medication may or may not be my silver bullet for hemophilia treatment, it most certainly is someone’s. Because of the differences in how hemophilic drugs work for each individual, the same is true for every hemophilia drug. Prescribing decisions are best left with the doctor, not the payor.

In closing, thank you for taking the time to listen and thank you for considering my experiences and my testimony. I respect and appreciate what you are trying to accomplish not only as someone with hemophilia but as a proud resident of Georgia.

Jeff Cornett’s Testimony

My name is Jeff Cornett, and I am the Vice President of Research and Public Policy for Hemophilia of Georgia. Since 1973, Hemophilia of Georgia has advocated on behalf of people with hemophilia and other inherited bleeding disorders in our state.

I am not here today to speak on behalf of any particular drug. Instead, I want to stress the importance of patients having access to the full range of anti-hemophilic drugs. We strongly believe this is necessary and medically sound due to the pharmacokinetics of these biologics and their routes of administration.

Since the 1970s, the treatment for hemophilia has been to inject a solution containing the missing clotting protein directly into the bloodstream. Infants and others with poor venous access often must have central lines implanted so they can receive the medication. They run the risk of infection and clotting within the line.

In the home, parents are taught to find a vein, access it with a needle, and slowly infuse the medication. Some parents must begin this when their child is a toddler. Two or three mornings a week they must reconstitute medication, prepare the injection equipment, and hold down a child while trying to find a suitable vein. Any medication that reduces or removes the need for frequent, intravenous administration is a godsend for these families.  Hemophilia of Georgia is steadfast in our belief that Georgia Medicaid should consider quality-of-life issues when granting approval for medications for these children.

As injected factor concentrate is a foreign protein, it carries the risk of stimulating the immune system to create inhibiting antibodies. The development of an inhibitor is dangerous and costly. We ask that Georgia Medicaid allow the use of products with little to no risk of inhibitor development and avoid requiring patients to switch medications, as this has been theorized to increase the risk of inhibitors.

As you know, there are no generics in the anti-hemophilia drug class. Factor concentrates differ in the manufacturing process. Some are derived from human plasma; others are created using recombinant technology. In the recombinant products, some are created using baby hamster kidney or Chinese hamster ovary cell lines. Others use human cell lines. Some are pegylated. Others are fused with human immunoglobulin or albumin. These differences can interact with an individual’s physiology to create unique pharmacokinetics.  What works well in one patient may not in another. Even one or two breakthrough episodes of joint bleeding each year can set a patient up for long-term joint damage, especially if trough levels (that is to say, if periods of low blood factor levels due to the medication’s half-life) are allowing microbleeding into joints. Hemophilia of Georgia believes the right medication for the patient is the one that gives the greatest efficacy with the least risk for adverse reactions, not necessarily the medication that is cheapest for Medicaid.  A medication that prevents all bleeding and does not require an indwelling port or frequent venipuncture can avoid costly complications down the road.

Hemophilia of Georgia is aware of the high cost of hemophilia medications. Fortunately, this is a rare disorder and there are relatively few patients with hemophilia on Georgia Medicaid.  We believe that Georgia Medicaid can achieve its goal of being financially prudent without restricting the ability of physicians and patients to find the hemophilia medication that is going to achieve the best outcome. We ask the Drug Utilization Review Board to join us in pushing for policies and procedures to that end.

Thank you.