Meadow Green co-wrote this post with Christina Corridon.
The 2016 American Society of Hematology (ASH) Annual Meeting was rich with clinical updates and innovations to advance care and improve life for patients with hematologic cancers. However, as life is prolonged among chronic and acute diseases, payers, providers and patients are wrestling with the cost of therapy over months and even years—a topic that came up many times during various sessions at ASH.
When discussing therapeutic choice, it’s hard to avoid the topic of cost. During Dr. Jaqueline Barrientos’ session, “Sequencing of Chronic Lymphocytic Leukemia (CLL) Therapies,” she mentioned this consideration in CLL, where an explosion of new treatments has transformed patients’ prognosis and out-of-pocket costs. When referring to chronic treatment with high-cost drugs, Barrientos says that it’s one thing if the patient is elderly, but it’s a bit different when the treatment choice is impacting a 40-year-old who will likely need to pay for those drugs over a much longer time period. According to Barrientos, cost will be a key issue to address moving forward.
The data to support treatment discontinuation already is starting to accumulate in some malignancies. For example, the session “Cessation of Tyrosine Kinase Inhibitors Treatment in Chronic Myeloid Leukemia Patients With Deep Molecular Response: Results of the Euro-Ski Trial” highlighted the results of a longitudinal European database study, which demonstrated that stopping TKI therapy did not compromise high molecular recurrence free survival rates, especially if patients have been treated with imatinib for close to six years and had a molecular response. The study also estimated, without the use of a true economic model, that patients who had discontinued treatment while in study (n=317) had generated a savings totaling about 22 million euros.
Currently, the jury is still out for durable response and treatment cessation (and the potential for eliminating recurring costs over time) for other therapies. The topic did recur in Dr. Barrientos’ discussion, which highlighted the potential for a “drug holiday” with Venclexta (venetoclax) in CLL. It’s also at the heart of some of the excitement around CAR-T therapies, a technology that produces complete responses in some patients and raises the interesting question of response duration. It’s interesting to imagine a crowded landscape where the length of treatment or recurring high costs drive treatment choice between a product that's used until progression versus a drug that offers a drug holiday or a “cure.” This may mean that the therapeutic choice may be different for a young patient, or for a Medicare patient comparing the recurring out-of-pocket cost for Part D specialty tier drugs or the out-of-pocket cost for a one-time infusion on Part B.
Moving forward, the presence or absence of data supporting treatment discontinuation and a durable response will likely be a key issue in multiple hematologic malignancies. On one hand, payers may question paying a premium price on a drug that just offers the “promise” of a durable response without the long-term follow-up data to support it, and that data will be years in the making. In contrast, providers and patients will be increasingly attracted even to the concept due to sensitivity to accruing costs over time and increasingly high out-of-pocket costs.
Manufacturers in the early pipeline and clinical development planning stage should consider if there is a scientific rationale for creating stopping criteria linked to response rather than progression, and how to operationalize this within the product’s study design. Although manufacturers have often dismissed the creation of such data in hematologic oncology for many valid reasons, it would be foolish to not address the impending availability of such data from competitors or from the market, itself. For those in the launch planning and go-to-market stage, it’s important to understand the likelihood of the clinical community exploring discontinuation or durable response, and address the risks and opportunities it may pose. For drugs in market, consider whether treatment discontinuation is a feasible opportunity and a compelling value driver, which may position your product as a preferred option for an increasingly cost-sensitive payer, patient and provider community.