What Attributes Will Distinguish Emerging Therapies in the Eyes of Hematological Oncologists and Payers?

The introduction of the proteasome inhibitor bortezomib (Millennium/Takeda/Janssen’s Velcade) and the immunomodulatory agents thalidomide (Celgene’s Thalidomide/Thalomid, Fujimoto Seiyaku’s Thaled) and lenalidomide (Celgene’s Revlimid) has revolutionized the treatment of multiple myeloma over the last decade; however, patients with relapsed/refractory (R/R) disease remain particularly difficult to treat. The FDA awarded carfilzomib (Onyx Pharmaceuticals/Ono Pharmaceutical’s Kyprolis) and pomalidomide (Celgene’s Pomalyst/Imnovid) accelerated approval in July 2012 and February 2013, respectively; both agents are expanding existing treatment options and have the potential to further improve treatment outcomes when combined with lenalidomide or bortezomib. Several other therapies, including agents from novel drug classes, are also in development for R/R myeloma. Owing to the high level of unmet need in this setting, significant clinical and commercial opportunity remains for therapies that can provide greater survival benefits compared with current standards of care.

Questions Answered in This Report:

  • Improving overall survival and delaying disease progression are key goals in the treatment of R/R myeloma (i.e., second and third lines of therapy). What are the key primary and secondary clinical trial end points with which new therapies are evaluated? How do U.S. and European hematological oncologists weight specific efficacy end points and other drug attributes in their prescribing decisions for R/R myeloma?

  • Lenalidomide plus dexamethasone (the Rd regimen) was the 2012 major-market sales leader for R/R myeloma. What weaknesses in Rd’s clinical profile might allow emerging therapies to gain traction in the market? Have emerging therapies demonstrated strengths on the attributes that surveyed hematological oncologists indicate are most important in their prescribing decisions? Which emerging therapies are likely to offer clinical improvements over currently available therapies?

  • Overall survival and progression-free survival are key drivers of physicians’ prescribing decisions and/or the focus of drug development for new R/R myeloma therapies. What trade-offs (if any) in these and other clinical attributes are U.S. hematological oncologists willing to make when considering the use of emerging therapies for the treatment of R/R myeloma? Based on the trade-offs in price and performance across key drug attributes that U.S. hematological oncologists are willing to make, how does physician preference and prescribing likelihood vary across different target product profiles for R/R myeloma?

  • By 2017, pomalidomide in combination with BD (bortezomib plus dexamethasone) will emerge as the gold-standard therapy in our Drug Comparator Model because of its superior clinical profile over the key current therapies we evaluated. On what clinical attributes is the regimen pomalidomide plus BD most differentiated from its competitors? Which current therapies are at greatest risk of being replaced by pomalidomide plus BD?


Attributes included in conjoint analysis based assessment of target product profiles for relapsed/refractory multiple myeloma:

- Median overall survival (months).

- Median progression-free survival (months).

- Overall response rate (% of patients).

- Hematological toxicity: rate of grade 3/4 neutropenia (% of patients).

- Rate of peripheral neuropathy—any grade (% of patients).

- Drug formulation.

- Price per 28-day cycle.

Attributes included in assessment of U.S. payers’ receptivity to new therapies for relapsed/refractory multiple myeloma:

- Improved effect on overall survival (OS).

- Improved effect on progression-free survival.

- Lower incidence of grade 3/4 neutropenia.

- Lower incidence of peripheral neuropathy.

Physicians surveyed: 60 U.S. and 31 European hematological oncologists.

Payers surveyed: 20 U.S. MCO PDs.

Comprehensive List of Therapies Included in Our Research and Modeling:

Current Therapies

- Lenalidomide (Celgene’s Revlimid) + dexamethasone (Merck & Co.’s Fortecortin, Visufarma/Banyu Seiyaku’s Decadron, generics) [Rd]

- Bortezomib (Millennium/Takeda/Janssen’s Velcade) + dexamethasone [BD]

- Bortezomib + lenalidomide + dexamethasone [VRD]

- Carfilzomib (Onyx Pharmaceuticals/Ono Pharmaceutical’s Kyprolis) monotherapy

- Pomalidomide (Celgene’s Pomalyst/Imnovid) + dexamethasone

Emerging Therapies

- Carfilzomib + Rd [CRd]

- Pomalidomide + BD

- Daratumumab (Genmab/Janssen Biotech/Janssen-Cilag’s HuMax-CD38)

- Ixazomib citrate (Millennium/Takeda’s MLN-9708) + Rd

- Elotuzumab (Bristol-Myers Squibb/AbbVie) + Rd

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