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Research & Reports

Searching in Biopharma (1580)

Multiple Myeloma (Relapsed/Refractory)

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

Questions Answered in This Report:

  • Delaying disease progression and improving overall survival are key goals in the treatment of R/R myeloma (i.e., second and subsequent 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 2011 major-market sales leader for R/R myeloma. What weaknesses in its profile would 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 will offer the clinical improvements over currently available therapies that surveyed MCO PDs seek from new therapies?

  • Overall survival and progression-free survival are key drivers of physicians’ prescribing decisions and/or are the focus of drug development for new R/R myeloma therapies. What trade-offs across 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 do physician preference and prescribing likelihood vary across different target product profiles for R/R myeloma?

  • Based on its clinical profile, bortezomib plus lenalidomide and dexamethasone (the VRD regimen) is the current clinical gold standard in our Drug Comparator Model. What attributes do thought leaders believe differentiate this therapy from competing current therapies and emerging therapies? Will any therapies in development challenge VRD as the gold standard in 2016 or 2021?


Attributes included in conjoint analysis-based assessment of target product profiles for R/R myeloma (second and subsequent lines of therapy):

- Median overall survival

- Median progression-free survival

- Overall response rate

- Hematological toxicity: rate of grade 3/4 thrombocytopenia/neutropenia

- Rate of peripheral neuropathy

- Drug formulation

- Price per 28-day cycle

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

- Effect on overall survival

- Effect on progression-free survival

- Effect on hematological toxicity

- Effect on peripheral neuropathy

Physicians surveyed: 60 U.S. and 30 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 (Takeda/Janssen-Cilag/Janssen’s Velcade) + dexamethasone [BD]

- Bortezomib + lenalidomide + dexamethasone [VRD]

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

- Pomalidomide (Celgene’s Pomalyst) + dexamethasone

Emerging Therapies

- Carfilzomib + Rd [CRd]

- Pomalidomide + BD

- MLN9708 (Takeda/Millennium) + Rd

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

- Panobinostat (Novartis) + BD