Estimating the Economic Impact of Adding Panobinostat to a U.S. Formulary for Relapsed and/or Refractory Multiple Myeloma: A Budget Impact and Cost-Benefit Model

Abstract

Funding for this study was sponsored by Novartis, East Hanover, New Jersey. Bloudek and Kish are employees of Xcenda, a consulting company contracted by Novartis to conduct this analysis. Roy, Globe, and Kuriakose are employees of Novartis. Siegel is on the advisory boards and speaker's bureau of Celgene, Onyx/Amgen, Millennium/Takeda, and Novartis and is on the advisory boards of Merck. Jagannath is a consultant to Sanofi, Bristol-Meyers Squibb, and Celgene. Orloski is a contractor to Xcenda and provided medical writing support, which was funded by Novartis. Study design and concept were contributed by Bloudek, Roy, and Kish, assisted by Globe. Bloukek took the lead in data collection, along with Kish, and data interpretation was performed by Siegal, Jagannath, Globe, and Kuriakose. The manuscript was written primarily by Orloski, along with Roy and Kish, and revised by Roy, along with Siegal, Jagannath, Globe, Orloski, and Kuriakose.

Cost-effectiveness of Pomalidomide, Carfilzomib, and Daratumumab for the Treatment of Patients with Heavily Pretreated Relapsed–refractory Multiple Myeloma in the United States

“…However, DSAs showed that the model results were relatively insensitive to changes in AE rates. Thus, including costs similar to those considered by Bloudek et al 20,23 are unlikely to have changed the results.…”

“…The model methodology used in this case was appropriate for making a comparison across treatments not included in the same trial, which was not necessary in the model by Borg et al 22 One other study has estimated the cost of RRMM therapy in the US. In the analysis by Bloudek et al, 20,23 the estimated monthly total costs (Medicare perspective, including drug costs, administration, and grade 3 or 4 AE prophylaxis and management) per patient treated with POM-d and CAR were $14,733 and $13,438, respectively. Inspection of the cost per treatment year reported by Bloudek et al, 20,23 shows that differences from estimates of this model ($11,966 and $12,489, respectively) were mostly due to the additional AE costs considered ($2,171/mo with POM-d and $1,303/mo with CAR versus $83/mo with POMd and $122/mo with CAR in this model).…”

“…In the analysis by Bloudek et al, 20,23 the estimated monthly total costs (Medicare perspective, including drug costs, administration, and grade 3 or 4 AE prophylaxis and management) per patient treated with POM-d and CAR were $14,733 and $13,438, respectively. Inspection of the cost per treatment year reported by Bloudek et al, 20,23 shows that differences from estimates of this model ($11,966 and $12,489, respectively) were mostly due to the additional AE costs considered ($2,171/mo with POM-d and $1,303/mo with CAR versus $83/mo with POMd and $122/mo with CAR in this model). However, DSAs showed that the model results were relatively insensitive to changes in AE rates.…”

“…Although several economic evaluations have been conducted in patients with RRMM, none have considered heavily pretreated patients in the US. [20][21][22][23] The very similar patient characteristics and clinical settings of the aforementioned trials make an evaluation between these 3 regimens feasible. The objective of this study was to assess the clinical and economic implications of treatment with POM-d versus DARA and versus CAR in heavily pretreated patients with RRMM.…”

The sustainability of a community nurses programme aimed at supporting active ageing in mountain areas

“…This is essential even if the innovative services are outsourced to social entrepreneurs on the local health market, according to the planned sustainability approach. The current literature proposes several frameworks for budget impact analysis to provide a comprehensive economic assessment of health care interventions, which can be used by those who manage and set health care budgets, such as the administrators of national and/or regional PHCSs . However, sustainability is not only a financial issue but something more complex and policy makers have to involve all relevant stakeholders and consider the context in which the innovative approach is introduced.…”