Global Patient Involvement in Sarcoma Care—A Collaborative Initiative of the Connective Tissue Oncology Society (CTOS) & Sarcoma Patients EuroNet (SPAEN)
2. Discussion Areas
2.1 Optimizing Clinical Care in Sarcoma
Early and Accurate Diagnosis
2.2. Overcoming Healthcare System Challenges for the Management of Sarcomas
2.2.1. Specialist Care of Sarcomas within a Multidisciplinary Team
- The need for a stronger body of evidence to demonstrate the status and benefits of MDT management of sarcomas. What different systems are operating globally? What impact does this have on prognosis? Performance data may be difficult to obtain but the structures operating within countries could be better understood and the examples of best practice could be showcased.
- The need to facilitate cross border collaborative healthcare with MDTs to support better patient care and also learning for healthcare professionals.
2.2.2. Awareness/Education Amongst Healthcare Professionals
2.3. Working on Cross-Border Challenges for Sarcoma Patients
2.3.1. Background of Global Cancer Realities
2.3.2. Background of Sarcoma Realities
2.3.3. Background for Future Solutions
- Solutions should be applicable on a regional/local basis; however, they need strong international cooperation and collaboration;
- A long-term solution could be the establishment of at least one sarcoma center (or sarcoma core team) per country or for neighboring countries. This would require knowledge transfer from experienced sarcoma centers to newcomers;
- For this type of cooperation across borders, digital health solutions will be crucial, such as solutions for diagnosis (pathology, radiology), remote surgery, data exchange, virtual tumor boards, or translation of medical reports;
- Existing initiatives such as EURACAN (European Reference Network (ERN) for rare adult solid cancers) in Europe for the exchange and discussion of patient cases or international financed projects such as SELNET (Sarcoma European & Latin American Network) for the exchange/transfer of experience and knowledge will become important;
- We also need better solutions for cross-border research such as digital (virtual) studies and registers;
- At a European level, investment should be placed in the development of training and educational activities for healthcare professionals and medical students in the usage of already existing tools that facilitate cooperation and knowledge-sharing within the medical community in a cross-border context, such as the Clinical Patient Management System (CPMS) .
2.4. Driving Research in Sarcomas
- There Are No Clinical Trials in Large Parts of the World
- The Incorporation of the Patient Voice in Planning Research/Clinical Trials
- Industry leading research with a strong focus on drugs and not enough research on other techniques or strategies.
- New drugs owned and directed by the pharmaceutical and biotech industry.
- Researchers and advocates can provide valuable information to the industry on sarcoma and encourage seeking approval for this rare disease.
- While factoring in industry business needs, partner to identify mutually beneficial goals for all.
- Funding for research for multi-modality treatment is limited.
- Generally, multi-modality research is driven by academic cooperative groups as interest across the industry has been more focused on drug development. Advocates can leverage their voices to lobby for more support.
- Drug Development
- Too much research for Me-too drugs?
- Once a target has proved beneficial in a disease, other similar compounds are studied with the aim of seeking approval.
- What are the downsides of having several drugs with similar mechanisms/targets for the same subtype?
- Given there are limited funding resources, does this divert funding from other subtypes?
- Looking for the lowest bar in drug development results in minimally beneficial drugs that are costly.
- It can make business sense to have low bars to meet business goals of approval so a drug can be marketed. However, what is the measure of benefit and who defines this? Is this an opportunity for incorporating patient perspectives?
- Cost can be in dollars and side effects. Both need to be explored along with approval.
- Drug repurposing
- In theory, there should be a shortened timeline to making a “repurposed” drug available if effective. This assumes that the dose and side effect profile will be the same in the patient population (may not be so). What is the potential for repurposing?
- Identifying sound rationale for the pursuit of a particular drug for sarcomas may be challenging.
- Patient Reported Outcomes (PRO) in rare diseases—the “right” PRO for evaluation of new therapies
- This field is evolving with several sarcoma groups developing subtype specific PRO tools. The European Organisation for Research and Treatment of Cancer (EORTC) and the Patient Reported Outcomes Measurement Information System (PROMIS) have validated tools that can be adapted.
- Is there an opportunity to have treatments approved with PROs as a primary endpoint?
3. Priority List of Projects to Be Worked on Together over the Next 5 Years
3.1. Minimum Requirements for Sarcoma Care and Sarcoma Specialist Centers and Empower Patients to Find Them
3.2. Digitally Enabled Multidisciplinary Networks and Stronger Body of Evidence for Benefits of MDT Sarcoma Management
3.3. Establish Sarcoma Expertise, Facilitate Access to Established Sarcoma Centers and Cross-Border Healthcare
3.4. Develop a Global, Collaborative Awareness Effort in Order to Achieve a Meaningful Impact
3.5. Global Access to Sarcoma Clinical Trials and Incorporating the Voice of Sarcoma Patients (Advocates)
3.6. Further Ideas and Potential Deliverables over the Next 5 Years
- The continuation of expert–patient advocate “consensus roundtables” for sarcoma subtypes
- A position paper on future optimal research and care for sarcoma patients
- Foster the establishment of sarcoma PAG in more countries worldwide
Conflicts of Interest
The CTOS & SPAEN Collaborative Group
|Agarwal, Manish, Mumbai, India; [email protected]|
|Basson, Mandy, Australia; [email protected]|
|Bickley, Sorrel, UK; [email protected]|
|Blay, Jean-Yves, Lyon, France; [email protected]|
|Bovée, Judith V.M.G., Leiden, The Netherlands; [email protected]|
|Casali, Paolo, Milan, Italy; [email protected]|
|Chacon, Matias, Buenos Aires, Argentina; [email protected]|
|Coutinho, Mariana, Portugal/UK; [email protected]|
|Fernandez, Natalia, Spain, [email protected]|
|Fernandez, Piga, Chile; [email protected]|
|Frezza, Anna Maria, Milan, Italy; [email protected]|
|George, Suzanne, Boston, USA; [email protected]|
|Gladdy, Rebecca, Toronto, Canada; [email protected]|
|Gonzato, Ornella, Italy; [email protected]|
|Graham, Ann, USA; [email protected]|
|Green, Phil, UK; [email protected]|
|Gupta, Vandana, India; [email protected]|
|Haas, Rick L., Amsterdam, The Netherlands; [email protected]|
|Jones, Robin L., London, UK; [email protected]|
|Kasper, Bernd, Mannheim, Germany; [email protected]|
|Kawai, Akira, Tokyo, Japan; [email protected]|
|Laakso, Annika, Finland; [email protected]|
|Lozinsky, Shannon, USA; [email protected]|
|Muñoz, Matías, Chile; [email protected]|
|O’Driscoll, Alyssa, USA; [email protected]|
|Oshida, Terumi, Japan; [email protected]|
|O’Sullivan, Maureen J., Dublin, Ireland; [email protected]|
|Painter, Corrie, USA; [email protected]|
|Portnoy, Marlene, USA; [email protected]|
|Pretorius, Lauren, South Africa; [email protected]|
|Quintin, Annick, Switzerland; [email protected]|
|Reed, Damon R., Tampa, Florida; [email protected]|
|Reinke, Denise, USA; [email protected]|
|Rothschild, Sara, USA; [email protected]|
|Schuster, Kathrin, Germany; [email protected]|
|Shimura, Takaaki, Japan; [email protected]|
|Stacchiotti, Silvia, Milan, Italy; [email protected]|
|Swallow, Carol, Toronto, Canada; [email protected]|
|Tap, William D., New York, USA; [email protected]|
|Thomas, David, Darlinghurst, Australia; [email protected]|
|Trent, Jonathan C., Miami, USA; [email protected]|
|van der Graaf, Winette T.A., Amsterdam, The Netherlands; [email protected]|
|van Oortmerssen, Gerard, The Netherlands; [email protected]|
|Wartenberg, Markus, Germany; [email protected]|
|Whiting, Jeanne, USA; [email protected]|
|Wilson, Roger, UK; [email protected]|
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Kasper, B.; Schuster, K.; Wilson, R.; Bickley, S.; Blay, J.-Y.; Reinke, D.; Wartenberg, M.; Haas, R. Global Patient Involvement in Sarcoma Care—A Collaborative Initiative of the Connective Tissue Oncology Society (CTOS) & Sarcoma Patients EuroNet (SPAEN). Cancers 2022, 14, 854. https://doi.org/10.3390/cancers14040854
Kasper B, Schuster K, Wilson R, Bickley S, Blay J-Y, Reinke D, Wartenberg M, Haas R. Global Patient Involvement in Sarcoma Care—A Collaborative Initiative of the Connective Tissue Oncology Society (CTOS) & Sarcoma Patients EuroNet (SPAEN). Cancers. 2022; 14(4):854. https://doi.org/10.3390/cancers14040854Chicago/Turabian Style
Kasper, Bernd, Kathrin Schuster, Roger Wilson, Sorrel Bickley, Jean-Yves Blay, Denise Reinke, Markus Wartenberg, and Rick Haas. 2022. "Global Patient Involvement in Sarcoma Care—A Collaborative Initiative of the Connective Tissue Oncology Society (CTOS) & Sarcoma Patients EuroNet (SPAEN)" Cancers 14, no. 4: 854. https://doi.org/10.3390/cancers14040854