Despite the comparatively low incidence of chronic myeloid leukemia (CML) in the United States, patient management requires extended durations of therapy, which in turn translates into a disproportionately large size market. Market dynamism is enhanced by the occurrence of resistance or intolerance to any one agent, resulting in many patients receiving three or more lines of therapy. This environment presents marketers with multiple opportunities to expand market share. CML treatment consists almost entirely of six targeted therapies, principally oral tyrosine kinase inhibitors (TKIs). First-to-market Novartis’s Gleevec (imatinib) is entrenched as the most commonly prescribed agent in newly diagnosed CML patients but battles against two other TKIs—Bristol-Myers Squibb’s Sprycel (dasatinib) and Novartis’s Tasigna (nilotinib)—in this setting. Market dynamism has been further stimulated by accelerated approvals of three agents in 2012: two oral TKIs, Pfizer’s Bosulif (bosutinib) and Ariad Pharmaceuticals’ Iclusig (ponatinib), as well as Teva’s Synribo (omacetaxine) for subcutaneous infusion.

Both physicians and payers expect CML treatment to enter a period of turbulence over the next two years following the anticipated launch of generic imatinib, which surveyed MCOs indicate will have a dramatic effect on tier placement of drug therapies. Managed care organization pharmacy directors/medical directors (MCO PD/MDs) will look for the demonstration of a clear efficacy advantage when making their decisions on formulary inclusion and tier status in CML, and marketers should look to address any weaknesses in their clinical dossiers. With adverse changes in tier placement likely to occur for most CML agents, the increasing cost burden borne by patients will be brought into even sharper focus. This coming period presents a window of opportunity for marketers to review their pricing strategies and patient payment assistance programs to counter an increasingly hostile climate.

Questions Answered in This Report:

  • Clinical practice, prescribing trends, and reimbursement considerations for established TKI first-line CML therapies: Which prognostic scoring systems do office- and hospital-based hematologic oncologists use for risk stratification in newly diagnosed patients, and how does this approach affect patient management? What percentage of newly diagnosed chronic phase (CP) CML patients receives Gleevec, Sprycel, or Tasigna, and how does this usage vary between office- and hospital-based physicians? How do physicians profile TKIs on key attributes and on safety and tolerability? What is the average duration of therapy in first-line CP CML? What restrictions are placed by payers on treatment durations? What are first-line therapy discontinuation rates? Which factors discourage compliance to first-line therapy? What are the key reasons for discontinuation of first-line therapy? What are first-line drug therapy patient shares for acute phase (AP) and blast phase (BP) CML, and what are the reimbursement hurdles? What is the extent of current coverage by surveyed MCOs for first-line CP CML patients, and how is this coverage expected to evolve over the coming year?

  • Clinical practice, prescribing trends, and reimbursement considerations for second- and later-line CML therapies: What percentage of CP CML patients receive specific targeted agents as second- and third-line therapy? How do targeted agents rate on key attributes, and what are the key drivers of therapy choice for second-line and third-line CP CML? What are treatment patterns for patients intolerant or resistant to first-line therapy and in patients progressing to AP or BP CML? What constraints do payers impose on prescribing by physicians in second-line and third-line CML? What is the extent of MCO current coverage in second-and later-line CP CML patients, and how is this coverage expected to evolve over the coming year? What are MCO perceptions of the performance of targeted agents in CML? Which cost control measures are imposed on CML prescribing by MCOs? How prevalent are bundled payment programs? Which acquisition routes are used by physicians? What are the reasons for the use of specialty pharmacies for CML?

  • Impact of FDA label changes on product uptake in CML: What was the response of hematologic oncologists and MCOs to the 2013 change in Iclusig’s label? How did the label changes affect management of Iclusig-treated patients? What restrictions did payers impose following the label change? What are physicians’ future intentions for Iclusig prescribing? What was the impact of the full regulatory approval for Synribo on prescribers, nonprescribers, and MCOs?

  • Evolving prescribing trends and formulary access considerations in CML: What are current sequential treatment regimens in CP CML, and how are these regimens expected to evolve by 2016? What are key drivers in the uptake of new therapies? How are preferred lists in commercial plans likely to change over the next 12 months? What is the rationale for non-preferred status on commercial plans, and what are the drivers of change away from this status? What do physicians consider will be the impact of the prospective launch of generic imatinib? What is the current and expected tier status of targeted therapies in commercial plans? What are the most important factors behind formulary inclusion and favorable tier status in CML? What are MCO attitudes toward patient payment assistance programs, and what has been the impact of such programs on reimbursement decisions?


This U.S. Physician & Payer Forum report contains insights from a survey of 100 physicians and 30 MCO pharmacy and medical directors regarding the dynamics that affect prescribing practices and formulary coverage of treatments for CML in the United States. We explore the factors that shape current and future treatment patterns as well as reimbursement trends in the CML space. Additionally, this report reveals physicians’ and payers’ perceptions of targeted therapies in the United States.

Markets covered: United States.

Primary research: Online survey of 100 physicians (58 office-based and 42 hospital-based hematologic oncologists) and 30 MCO directors (20 PDs and 10 MDs).

Author(s): Philip White