From Specialty Pharmacy News

Specialty Pharmacies Can Bring Unique, High-Touch Hemophilia Management

Reprinted from SPECIALTY PHARMACY NEWS, a monthly newsletter designed to help health plans, specialty pharmacies, pharma companies, providers and employers contain costs and improve outcomes related to high-cost specialty products. Subscribe today!

By Angela Maas, Managing Editor
October 2017Volume 14Issue 10

Hemophilia is a unique condition that’s unlike many therapeutic categories treated with traditional specialty therapies. Patients need individualized care, and coordination and communication among an array of stakeholders are crucial. Specialty pharmacies can help facilitate that coordination in order to ensure people receive the individualized, high-touch care they need, as well as keep costs under control for a condition in which annual spending easily can top $1 million for a single person.

“Due to its predictably high costs, variant severities, episodic nature and high risk of inhibitor formation, hemophilia presents a classic disease management opportunity,” asserts John Louis, director of category management at Diplomat Specialty Infusion Group. “It is also a disease with decades of pharmacy management history to learn from.”

According to Louis, “Health plans might spend as little as $5,000” per year on one member with hemophilia. However, “In the case of an inhibitor, they could spend millions of dollars.” This occurs when a person’s immune system thinks that the factor used to treat hemophilia is a foreign substance and develops inhibitors to attack it. According to the Centers for Disease Control and Prevention (CDC), this happens in about one in five people with hemophilia A and three in 100 with hemophilia B.

In fact, Louis tells AIS Health that “We have come across a case of a health plan struggling with the case of a patient who was exceeding $10 million. It was an extremely rare case where the patient was affected by both hemophilia A and von Willebrand disease, plus an inhibitor, hepatitis B and HIV. There were also multiple hospitalizations and emergency room visits.” The CDC notes that people with inhibitors “are twice as likely to be hospitalized for a bleeding complication, and they are at increased risk of death.”

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“Hemophilia is one of the most costly therapeutic classes to manage with an average cost around $150,000 per patient and...upwards of $1 million in patients who have developed inhibitors to treatment,” explains Haita Makanji, Pharm.D., vice president of clinical strategy and programs at Magellan Rx, the PBM unit of Magellan Health, Inc. And, points out Mesfin Tegenu, president of PBM PerformRx, “The cost of treatment only increases with age due to weight-based adjustments and severity of hemophilia.”

When it comes to managing costs associated with hemophilia, “While some cost-management strategies can be applied at a health plan level, the most effective ones might ultimately be found at an individual patient level,” Louis tells AIS Health. “For example, while a young patient might appear to be adherent to his prophylactic infusion regiment, it might be discovered through routine telephone contact that a parent has been infusing in the evenings due to a rushed morning schedule. This means the patient’s factor levels are suboptimal during the daytime, when the patient is most active. Thus, the patient is at increased risk of a bleeding episode, which could require additional infusions to resolve.”

Hemophilia “is managed to enhance patient quality of life and to maximize clinical outcomes,” says Tegenu. “Without education and support, a hemophiliac patient can become non-compliant or stop taking prophylactic doses, which can lead to increased frequency of bleeds, ER visits and costly hospitalizations.” Education, he says, can help patients “understand signs and symptoms of bleeds and ways to care and prevent them.”

Stockpiling Factor Can Lead to Wastage

Tegenu tells AIS Health that “A comprehensive hemophilia management program includes monitoring of factor supply, which is important to ensure that patients have necessary drug on-hand as well as avoid stockpiling which leads to expiration and waste of resources.” Stockpiling, says Makanji, “is a major issue in the hemophilia community that may potentially lead to large amounts of wastage.” For this reason, she says, “It is always important to be aware of the patient’s inventory prior to dispensing; however, this information is not often collected, documented and reported by many pharmacies. Increasing the visibility into these processes through data collection will allow us to have more insight into which physicians and pharmacies are consistently providing quality and cost-effective care.”

But when patients don’t have factor on hand while experiencing an acute bleed, Louis says, they “usually end up in the emergency room or a nearby treatment center that carries factor, both which likely charge much more for the drug — in addition to service and facility costs.” He notes that because of the high cost to stock factor, many hospitals often may not carry it.

“The goal of therapy for a patient with a bleeding disorder is to provide education and support to allow the patient to manage factor infusions at home,” says Kirstin Schmidt, national clinical director – bleeding disorder program at infusion service provider Option Care, part-owned by Walgreens. “This allows the patient to self-administer therapy at the time of a bleed, which is both more convenient for the patient and family and cost-effective for the payer.”

Other issues within the hemophilia space make it a condition ripe for management. For one, says Makanji, “There is a significant lack of transparency between stakeholders in this category. Many products to treat hemophilia share the same medical benefit drug billing codes (HCPCS), which can make claims analysis difficult to interpret. Furthermore, there is concern around how hemophilia products are dosed and dispensed to the patient. Patients are often dosed based on standardized dosing protocols or ‘population averages’ rather than on how they personally metabolize the drug, which can lead to less than ideal dosing regimens. Even once a dose is determined, there are discrepancies between the dose prescribed and the dose dispensed by pharmacies.”

According to Louis, “Assay management is a strategy commonly employed by specialty pharmacies to control costs. Factor medications are unique. Due to the manufacturing process, the amount of factor in each vial will contain 10% more or less than the quantity labeled on the packaging. A prescription for factor, therefore, is usually written with a ±10% discretionary allowance for the dispensing pharmacist. That means dispensing pharmacies may bill up to 10% more (or less) than the prescribed ‘target’ dose (the middle of the given range). Dispensing assay combinations closest to the target dose ensures therapeutic efficacy while preventing unnecessary costs.”

Magellan Rx, Makanji says, supports “full transparency” as an effective management strategy, because “insight into prescribed doses, dispensed doses and bleed history allow providers and managed care organizations to successfully identify appropriate areas to intervene.” In addition, when patients may find it challenging due to geographic restrictions to travel to a hemophilia treatment center (HTC), “better data collection will allow Magellan Rx to leverage national key opinion leaders to provide second opinions on challenging cases.”

“HTCs play a tremendously important role in this space as they are able to get the most cost-effective drug pricing for hemophilia products due to their 340B status,” Makanji explains, referring to the federal government program through which manufacturers provide outpatient drugs at lower prices to participating entities. “Today, more than ever in the specialty space, there is talk of limited networks and preferred specialty pharmacies. This can lead to HTCs not being included in dispensing networks and therefore be unable to dispense drug. Without drug dispensing margins, many HTCs would struggle to remain solvent. Magellan Rx does not advocate pulling patients away from HTCs, but instead favors a more standardized, quality measure-focused approach in which all providers, whether they are HTCs or not, are held to the same standards.”

“At Option Care, we see ourselves as an extension of the HTC team,” says Schmidt. “Our focus as the [specialty pharmacy] is on providing care in collaboration with the HTC to meet the goals and needs of the patient.”

‘Each Case Is Different’

People with hemophilia also require highly individualized care. “Each case is different,” maintains Louis. “The needs of a grown adult are worlds apart from those of a baby and its new parents. Therefore, a carefully constructed care plan would consider a patient’s individual medical history, disease severity, age, education and training needs, caregiver support, inhibitor status, financial circumstances, psychosocial needs, intravenous access, nursing requirements, orthopedic complications, comorbidities, physical disabilities, emergency preparedness and the home environment.”

Because each patient is different, “We cannot simply place these members in predefined programs because the severity level of their disease fluctuates greatly,” says Tegenu. “Many of these members also have multiple co-morbidities that further complicate the treatment of their other conditions. The risk of ER visits/hospitalizations is higher with the hemophilia population than patients treated with ‘traditional’ specialty medications.”

Hepatitis and HIV are two co-morbid conditions seen within the patient population due to infection from tainted blood years ago. “The good news is that manufacturing processes and treatments have significantly improved, and the risk of new transmission of such diseases through blood product is non-existent today,” explains Makanji. “Still, patients are able to live much longer lives with hemophilia than ever before, and because of that there are many adult patients that were transmitted these viruses years ago. At the end of the day, these are complex patients with unique needs, and they need holistic care. One of the primary value propositions of hemophilia treatment centers is they provide a coordinated team-based approach to treatment that can help handle these complexities. Through data and outcome collection, Magellan Rx ultimately wants to demonstrate the value of this approach.”

“One of the greatest challenges that specialty pharmacies face with HIV- or hepatitis-infected hemophilia patients is adherence to medication,” says Louis. “Oral medications for HIV coinfection require a tremendous amount of treatment discipline to keep the disease at bay. Patients might also view coagulation products as less critical to sustaining life and, consequently, use them with less urgency.”

And before the newer hepatitis agents came onto the market the last few years, “treatments had punishing side effects and sub-optimal success,” he says. “A hemophilia patient who was unsuccessful in previous treatments might be reluctant to endure treatment again. A pharmacy’s role is to educate patients on the success rates and minimal side-effect profiles of newer drugs and to encourage continued treatment. For patients who are not treating their coinfections, it is often due to significant challenges in maintaining bleed prevention. They are prone to less active lives and less controlled diets. They might often need help with psychosocial matters affecting depression, which affect treatment.”

Specialty Pharmacies Serve ‘Vital Function’

“Managing hemophilia requires personalized medical treatment and pharmacy care plans,” says Louis. “Parents of a newly diagnosed patient might require extensive support from a local HTC staffed with specialists in pediatric hematology, nursing, orthopedics, physical therapy, psychology, genetics, dentistry and social work.” Specialty pharmacies, he contends, “serve a vital function in the ongoing management of patients. Between comprehensive care visits to an HTC (often only once per year), a specialty pharmacy is typically the only stakeholder in routine contact with the patient. Specialty pharmacies monitor drug utilization and home product inventory and, among other things, can deploy in-home nursing (as well as provide self-infusion training), provide educational resources, help patients secure gap insurance or copay assistance, counsel on therapy options and report vital information to the treating physician.”

“In order to provide personalized, high-quality care for effective management of hemophilia, the specialty pharmacist must work collaboratively with the patient, caregivers, prescribers and HTCs to provide individualized care,” says Schmidt. “It is crucial for the [specialty pharmacy] to ensure the therapy provided is safe and accurate and all team members have a good understanding of bleeding disorders.”


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