Soft tissue sarcoma (STS) is a
collection of rare and diverse cancers that have a range of histologies and can
occur at any location on the body. The highly fragmented nature of this group
of indications—comprising at least 50 different subtypes—has provided many
challenges to its diagnosis, treatment, and development of new therapies.
Clinical trials evaluating new agents have frequently included multiple STS
subtypes with differing outcomes, which has made demonstration of significant drug
effects particularly difficult. Experts point to the need to manage STS in
specialist centers of sarcoma expertise and to design clinical trials that
include selected disease subtypes only in an attempt to progress and improve
treatment outcomes, especially those of advanced disease.
The mainstay of treatment for
early-stage disease is surgery, with or without radiotherapy; data showing a
role of adjuvant drug treatment are conflicting, and thus there is no consensus
for medical practice for these patients. Currently, the key drugs used to treat
advanced recurrent/metastatic STS are doxorubicin and ifosfamide, although
other drugs, such as a taxane, may be preferred for certain histologies. The
European availability of trabectedin (PharmaMar’s Yondelis) has increased
treatment options for relapsed patients. U.S. experts hope that trabectedin
will be FDA-approved in the near future, although they currently can access the
drug through patient assistance programs. The 2012 FDA approval of pazopanib
(GlaxoSmithKline’s Votrient) signaled the first targeted therapy to enter the
treatment armamentarium for STS. However, in many situations pazopanib is not
preferred to chemotherapy.
The late-stage development
pipeline for STS is relatively sparse, and the most advanced drug candidates
are largely more chemotherapeutics, including novel formulations of existing
drugs. This is in contrast to the need highlighted by experts for a more
personalized and targeted approach to drug treatment. Consequently, unmet need
for this difficult to treat and fragmented and complex indication will endure
over the next ten years.
Questions Answered in This Report:
Several therapies are in development for advanced/metastatic STS.
Which of these agents are most likely to launch? Will any emerging therapy
have the potential to become the new standard of care? What are interviewed
experts’ opinions on emerging therapies in this setting?
Pazopanib has recently received FDA approval for advanced STS and
many other targeted agents are in development. Will additional small-molecule
antiangiogenics succeed for STS and challenge pazopanib for patient share? Which
other targeted agents are most likely to launch for treatment of advanced STS?
Interviewed experts state that there is a great need for focused
clinical trials that include only specific subtypes of STS. How are emerging
drugs being trialed? What other unmet needs do interviewed experts identify?
Drug development efforts have focused on unresectable advanced/recurrent/metastatic
STS. What is the size of this population? What proportion of STS patients are
eligible for treatment within these patient populations? Will any emerging
therapy have the potential to increase treatment rates?
Markets covered: United States, France, Germany, Italy,
Spain, United Kingdom.
Primary research: 9 country-specific interviews with oncologists
with expertise in treating STS.
Epidemiology: Diagnosed incidence segmented by stage (I,
II, III, IV) and anatomical site (extremities/trunk/head/neck and
peritoneal/retroperitoneal). Diagnosed incidence by histology (myofibrohistiocytic
sarcoma not otherwise specified, fibroblastic/myofibroblastic sarcoma, tumors
of uncertain differentiation, synovial sarcoma, tumors of peripheral nerves, vascular
chondro osseous sarcoma, other).
Population segments: adjuvant, recurrent/metastatic first-line,
recurrent/metastatic second-line, recurrent/metastatic third- and subsequent-line.
Emerging therapies: Phase II: 21 drugs; Phase III: 6 drugs.