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Access to Hematology Care in an Age of Innovation

The 91������Ʒ (ASH) believes that all individuals should have access to and be able to afford high-quality, clinically appropriate care, including innovative therapeutics. Hematologists treat patients with malignant hematologic disorders such as leukemia, lymphoma, and multiple myeloma, as well as non-malignant conditions such as sickle cell anemia, thalassemia, bone marrow failure, venous thromboembolism, and hemophilia. Many of these diseases are relatively rare, requiring typically high-cost, highly specialized diagnostic and therapeutic services. High cost alone, however, is not always indicative of high value and proof of benefit must always be demonstrated to the patient and the treating physician.

Advances in science have led to a recent surge of approvals for treatments for hematologic diseases and disorders. Chimeric antigen receptor T-cell (CAR-T) therapy, approved in 2017 for certain patients with lymphoma and leukemia, is a potentially life-saving cellular therapy for patients who have exhausted all other options. Several new CAR-T products and additional indications (e.g. multiple myeloma) are expected in the next few years. In addition, gene therapies are anticipated for beta thalassemia and sickle cell disease, offering potential cures for diseases with previously limited treatment options. These innovative treatments, however, come at a high cost, creating potential barriers to access. The principles outlined below are supported by ASH to help ensure that all individuals have access to high-quality, specialized care, including innovative therapeutics.

1. Treatment decisions should be made between a physician and the patient.

Treatment decisions should be made between a physician and the patient; yet many times, policies, put in place by the Federal government or insurance companies, can impact these decisions.

Step therapy (ST) and prior authorization (PA), widely used amongst insurers as a way to reduce costs, both have the potential to delay patient access to necessary, high-quality medication and to increase administrative burden for physicians. Unfortunately, these utilization management techniques are even used for commonly prescribed, evidence-based treatments, considered to be the standard of care, and many times limit the ability of the physician to prescribe the best/evidence-based standard of care for their patients. Many individuals with sickle cell disease, for which recurrent severe acute painful crises and chronic daily pain are the most common complications, are forced to switch from long-acting pain medications they have been taking for years because of failed PA attempts or a new PA must be filed every month for the same medication, placing an unnecessary administrative burden on the physician. Additionally, PA often delays care for both bone marrow transplants and cellular therapy, causing additional toxic therapy to be administered despite clear indications for these other treatments. And a patient with Waldenstrom’s macroglobulinemia, a type of non-Hodgkin lymphoma, can have thrombosis or stroke because the proteins in their blood increase while waiting to start the therapy to reduce them. ASH supports the outlined by the American Medical Association and reforming ST and PA policies to promote timely access to clinically appropriate therapies.

“Non-medical switching” is another strategy used by insurers to control costs. The term “non-medical switching” refers to changing a patient’s medications for reasons other than medical ones.1 With non-medical switching, patients are forced to change their current medication to an alternate one selected by the insurance company; such a change could have detrimental effects on patients’ health and compliance with their recommended treatment plan.2 For example, in 2022, hematology patients taking direct-oral anticoagulants (DOAC) – drugs critical for the treatment of venous thromboembolism (VTE) – faced challenges accessing their elected DOAC due to non-medical switching to an alternate DOAC. The DOAC that had been removed from the formulary had a lower bleeding risk, and studies showed that it is safer in patients with advanced age, cancer, and chronic kidney disease.3 The removal of the preferred DOAC from the formulary caused significant clinical and safety concerns related to patients being forced to switch. Decisions regarding any medication choice, and all treatment choices more broadly, should be based on clinicians’ expertise, scientific evidence, as well as patients’ preferences and values, and are made through a shared decision-making process. Non-medical switching circumvents the patient-physician decision-making process to determine the most effective and culturally appropriate treatment plan and ignores hematologists’ expertise. This is neither a safe nor effective way to practice medicine. Non-medical switching by insurers should not occur without the advance knowledge of the patients and their treating physicians. In addition, medical considerations should supersede non-medical ones; patients and their physicians should have the option to reject a change based on non-medical switching if the switch undermines the medical interests of the patient.

ASH believes that patients should have access to the most effective treatment option, whether that be a brand name drug or a generic drug. Proposed policies requiring generic drugs to be prescribed first over brand name drugs may be detrimental to patient outcomes in certain circumstances. Currently, there is a lack of comparative data on the difference between some generic and branded drugs that treat hematologic diseases. For example, very little data exists on the difference between generic and branded imatinib, used to treat certain types of leukemia, and some of the studies that have been done demonstrate that individuals treated with generic imatinib experience higher rates of treatment failure at three months and lower overall survival, progression-free survival, and event-free survival at 24 months.4

Allowing patients to receive the most efficacious treatments in a timely manner can increase the likelihood of lower health care costs in the future, by enabling patients to achieve their optimal health outcomes.

2. Patients should have access to innovative therapeutics and physicians should be equitably reimbursed for their delivery. The high cost of these therapies should not be shifted to physicians.

Innovative therapeutics, such as cell and gene therapies, offer potential cures for diseases and disorders, which previously may have had limited treatment options. Many of these therapies are designed to be a one-time treatment, preventing the need for other, ineffective treatments, or a lifetime of maintenance therapies. But they come at a high cost and innovative payment solutions, which have been slow to develop, are needed to address current barriers and prevent future barriers to access. As seen with CAR-T therapy, payment policies and solutions have not kept pace with the science and approvals, and the lack of a sustainable payment solution has left a chilling effect on patient access to care for this potentially life-saving therapy.

ASH supports policies that will make these therapeutics attainable to the patient populations in need of them, and, at the same time, provide equitable reimbursement for the physicians who deliver them. Physicians should not be held liable for the high cost of these therapies. ASH encourages the development of value-based payment arrangements, especially for innovative therapies, as long as they are evidence-based and clinically appropriate. These arrangements must recognize the physician services associated with the provision of the treatment, including the cost of obtaining, storing, and direct administration of these therapies.

3. Medical considerations, not cost, should drive the determination of the site of care for providing high cost treatments.

ASH supports a site neutral payment policy for drugs so that medical considerations, not cost, drive the determination of site of care. For example, there is a significant disparity between inpatient and outpatient reimbursement for CAR T-cell therapy. The majority of ASH members currently consider the inpatient setting to be most clinically appropriate for the average patient, due to the likelihood of adverse events occurring in the days post-infusion; however, institutions may lose over $100,000 for each patient treated in the inpatient setting compared to those treated in the outpatient setting where drug and patient care costs are covered by a drug payment of the average sales price (ASP) plus six percent as well as a separate payment for the care delivered.

A treatment plan for an individual with a hematologic disease or disorder should be the result of discussion between the patient and the physician and should allow for timely, affordable access to both standard of care and innovative therapies if determined to be the most appropriate for the patient. Without a change in how our health care system reimburses these treatments, access will be limited, and future research for similar therapies likely slowed, if not halted. ASH will continue to work to ensure that patients with hematologic diseases and disorders are able to access and afford high-quality, clinically appropriate care, including innovative therapeutics.

As an organization, ASH also continues to identify and advocate for ways to limit patient out-of-pocket expenses. Click here to read the ASH Policy Statement in Support of Patient Access to safe and Effective Drugs.  

Originally published November 22, 2019. Revised April 2, 2024.