Hematologic cancers continue to gain awareness for payers. This is a result of the number of novel therapies recently approved by the US Food and Drug Administration to treat cancers that have few alternatives, as well as the growing number of expanded indications and use of combination therapies.
At the 2013 American Society of Hematology (ASH) meeting, data were presented supporting the clinical value of novel therapies and existing treatments. And just as important was the emphasis on cost-effective tests, treatments, and procedures. Several retrospective analyses evaluated cost-effective treatments through various lines of therapies.
One of the more popular takeaways was the list of unnecessary and potentially harmful treatments.
Evidence-Based Recommendations Focus on Waste
Last year, ASH released specific hematology-related recommendations referred to as “Choosing Wisely” (Hicks LK, et al. Blood. 2013;122:3879-3883). An ASH task force evaluated suggestions from many committee members and examined systematic reviews to compile a list of management-related recommendations.
A total of 5 recommendations were presented at ASH 2013, all targeting the practicing of efficient, cost-conscious medicine by avoiding unnecessary procedures and treatments, including:
- Do not infuse more than the minimum number of red blood cells necessary to relieve the symptoms of anemia or to return a patient to a safe hemoglobin range
- Do not test for thrombophilia in adults with venous thromboembolism (VTE) occurring in the setting of major transient risk factors
- Avoid using inferior vena cava filters routinely in patients with VTE
- Do not administer plasma or prothrombin complex concentrates for nonemergent reversal of vitamin K antagonists
- Limit surveillance computed tomography scans in asymptomatic patients after curative intent treatment of aggressive lymphoma.
ASH will continue to develop evidence-based guidelines that will likely influence the quality of care; these guidelines will be useful for payers in medical technology and in pharmacy and therapeutics assessments.
Site of Service Affects Cost
Identifying the most appropriate site of service is common among payers. Given a choice to cover chemotherapy and supportive care within an office or clinic or in a hospital, the majority of payers will choose the office or clinic as the optimal site, based on cost alone. Carolina Reyes, PhD, evaluated the cost differences by site of service in a retrospective analysis specific to rituximab (Rituxan) infusions. With the use of medical and pharmacy claims, this study examined the use and cost of rituximab infusions in the treatment of diffuse large B-cell lymphoma. Of the 491 patients evaluated, 65% received rituximab in the office and the remaining 35% received infusions in the hospital setting. The follow-up extended through 30 days after the last infusion and included at least 7 months of therapy.
The average number of infusions administered to patients in a hospital was actually less than for patients given infusions in a clinic (4.92 vs 6.52, respectively). Of the patients treated in the clinic, 87% received granulocyte colony-stimulating factor (G-CSF) compared with 77% in patients treated in the hospital.
These findings are similar to the rates of patients receiving combination therapy (ie, rituximab plus chemotherapy), which were 93% in the clinic compared with 85% in the hospital. From this, one may assume that with more rituximab infusions, more chemotherapy, and more G-CSF given in the clinic compared with the hospital, the cost would be substantially greater for patients treated in the clinic.
However, the average unadjusted daily infusion cost was higher in the hospital than in the clinic setting ($12,481 vs $5834, respectively), and the total per-member per-month costs were also higher for the patients treated in the hospital than those treated in a clinic ($22,325 vs $15,541, respectively).
These data emphasize the importance of aligning appropriate contractual arrangements. As more centers adopt accountable care models within oncology through various payer-provider arrangements, site of care will become an even more important cost-containment measure.
Advances in Therapy
At the previous meeting, much focus was placed on new treatments and investigational treatments for chronic myelogenous leukemia (CML). There has been a lot of postmarketing activity for second- and third-generation drugs for CML in the past 12 months. There has also been a lot of emphasis regarding appropriate testing at 3-month intervals, which may influence drug choice. At ASH 2013, one focus area was the pipeline for non-Hodgkin lymphoma (NHL).
Several PI3 kinase (PI3K) inhibitors are nearing approval. PI3K inhibition reduces B-cell survival. Phase 2 data have demonstrated a high response rate persisting a year with idelalisib monotherapy in patients with indolent NHL refractory to both an alkylating agent and rituximab.
Idelalisib, given orally twice daily, has also demonstrated a strong overall response rate in patients with follicular lymphoma, small lymphocytic lymphoma, marginal zone lymphoma, and Waldenström’s macroglobulinemia. The most frequent adverse events with idelalisib monotherapy were diarrhea and transaminase elevations.
According to Martin Dreyling, MD, copanlisib may demonstrate the quickest response of the PI3K inhibitors. Copanlisib, given as a weekly infusion, produced significant response rates in patients with follicular lymphoma, chronic lymphocytic leukemia, mantle-cell lymphoma, peripheral T-cell lymphoma, and diffuse large B-cell lymphoma. Common adverse events included gastrointestinal toxicity and metabolic side effects.
SAR 245409 is an oral inhibitor of PI3K and mTOR. This agent has demonstrated efficacy as monotherapy in the treatment of relapsed or refractory follicular lymphoma in patients who had received a median of 3 previous regimens. Common adverse events included diarrhea, rash, and liver toxicities.
These PI3K inhibitors may bring welcomed treatment options to patients with lymphomas. Payers will need to evaluate these new options among themselves, as well as compared with existing options, to determine their true value.
In addition to presenting data on new and existing treatments, several cost-efficacy analyses were presented at the 2013 ASH meeting. ASH should also be applauded for its “Choosing Wisely” recommendations, and its continued work to produce evidence-based guidelines.