Niche Markets and Rare Diseases

December 2012

Niche Markets and Rare Diseases: Soft Tissue Sarcoma

Report Authors
Joanne Graham, Ph.D.
Niamh Buckley, Ph.D.
Benjamin Guikema, Ph.D.
Lade Ayodele, M.B.B.S., M.P.H.

Introduction:

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?

Scope:

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, leiomyosarcoma, sarcoma not otherwise specified, fibroblastic/myofibroblastic sarcoma, tumors of uncertain differentiation, synovial sarcoma, tumors of peripheral nerves, vascular tumors, rhabdomyosarcoma, 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.


Search Reports

Mentioned in this report:

  • - Ajinomoto
  • - Amgen
  • - Ariad
  • - AstraZeneca
  • - Bayer
  • - Bristol-Myers Squibb
  • - CytRx
  • - Eisai
  • - Genentech
  • - GlaxoSmithKline
  • - Lilly
  • - MEI Pharma
  • - Merck Serono
  • - MorphoTek
  • - Novartis
  • - Oncolytics
  • - Onyx
  • - Pharmacyclics
  • - PharmaMar
  • - Pfizer
  • - Roche
  • - Sanofi
  • - S*Bio
  • - Threshold
  • - TopoTarget
  • - ZioPharm
Decision Resources Group brands include: