What Is a Sarcoma ‘Specialist Center’? Multidisciplinary Research Finds an Answer

1. Introduction

Over the course of the last 20 years the treatment of sarcoma in the western, more wealthy, world has improved through the introduction of multidisciplinary management and development of specialist centers. In some areas a network approach has evolved with the multidisciplinary team based in different healthcare settings and hospitals, but retaining the multidisciplinary meeting using information and communications technology.

Much of the world however has not been able to adopt these principles. The population of the world is about 8bn and that of Europe and North America combined is only about 17% of that. Although data are not available it is certain that sarcoma care and treatment standards now delivered in the wealthy world are rarely available beyond it. Patients and patient support groups report this anecdotally. The areas of the world from which patients most frequently reach out for advice about treatment are Eastern Europe, Middle East, South-East Asia, Africa, South and Central America. Doctors providing such advice are mostly from USA, Europe, UK, Canada and Japan.

The development of the Sarcoma Patient Advocacy Global Network (SPAGN) has brought a patient focus onto this challenge and consequently advocacy consideration of how sarcoma specialism can be encouraged to develop where it does not currently exist, and further develop where it does exist has become a priority.

The Need for Specialist Care

Sarcomas are generally poorly understood. They account for slightly more than 1% of all cancer incidence so are among the rarest, and often least considered, of all cancers. The American Cancer Society’s 2021 GLOBOCAN analysis of worldwide cancer incidence, which has over 46,000 citations, only mentions the specific Kaposi’s sarcoma, ignoring the majority of soft tissue sarcoma and bone sarcoma altogether [1].

Even with effective primary treatment (usually surgery) the reported 5 year survival varies between about 65% in the western world [2,3] to around 50% on a worldwide estimate. To complicate the challenge of understanding rarity is the fact that there are over 100 different sarcoma histologies, tumours can be found almost anywhere in or on the human body, and tumours can become quite large and are often painless until they impact on organs or on patient function.

When sarcoma becomes advanced treatment options are few. International guidelines rely on toxic chemotherapies such as anthracyclines for many sarcomas, although research with immunotherapies is making small steps forward [4]. Genetic understanding is improving but advances are on a histology-by-histology basis and matching genetic characteristics to available therapies is a highly specialist medical requirement [5]. This latter area of development is exclusively in the more wealthy world [6].

Steps are being taken to improve data capture, collation and analysis. It is recognized that registries offer opportunities to understand and identify areas for improvement. Only in western Europe are there national registries which can present a comprehensive picture and return analytical data to the front-line, whether for policy-making or individual treatment. Even here a Common Data Model (CDM) [7] is not identified and cross-border data collation and analysis is unrealized although the ambition is recognized and that work is happening [8]. How this can be extended to the developing world is an unknown, although there is growing experience with data integration in areas such as financial services.

Project Rationale

Management of sarcomas in specialist centers is associated with significant benefits for patients [9]. Even though the evidence is limited, it is undisputed that sarcoma patients should be treated by experts in specialist centers and is regarded as essential in published clinical guidelines [10,11].

Individual patients and the Sarcoma Patient Advocacy Global Network (SPAGN) have used the terms specialist and expert frequently in reviewing sarcoma care and treatment. Some interpretation of the terms can be inferred in these papers [12,13] but a definition has not been addressed.

Although widely used, the terms ‘specialist center’ or ‘expert center’ are rarely defined. They have even become controversial and are being challenged in the light of comments from various parts of the world [14,15,16].

Patient advocacy groups from countries traditionally seen as “third world” also report the absence of specialist care. It is an ambition which needs a means to express the aspiration, introduce the intial standards required and to explain the benefit and value such care can offer.

SPAGN worked with an international group of expert sarcoma doctors, to:

• identify features and principles which define what is needed to manage sarcoma patients optimally
• and to: find a term that could be applied in any area of the world.

We believe that these will help healthcare systems and treatment providers understand the importance of specialist care for people with sarcoma.

2. Methods

A discussion paper was distributed to those attending the 2023 SPAGN Annual Conference in Dublin, Ireland, followed by a plenary conference debate, attended by approximately 75 delegates. This resulted in an abstract presentation at the Connective Tissue Oncology Society (CTOS) Conference 2023. Further wider consultation with patient groups and professionals across the world followed. This paper is the result of that work.

Analysis of a Solution

Any terms we choose should recognize that there are national and regional variations beyond the control of sarcoma diagnosis and treatment resources. These differences can be significant and will be impacted by such factors as:

• Healthcare provision and planning, taking account of population centers or population distribution in rural areas, for example
• Budgets, whether these are determined by national provision, hospital constraints or insurance providers
• Availability of specific sarcoma expertise (eg surgical sub-specialties) even when general oncology expertise is available
• Access to specialist resources (eg ablative therapy, proton beam)
• General awareness of sarcoma as a malignancy
• Awareness of the need to develop and support specialist services

The meeting attendees agreed that we are looking for high level core principles based on the multidisciplinary approach which can be expressed simply and be readily understood. These should be supported by ‘recommended’ or ‘desirable’ factors which allow nationally specific terms to be used in expressing them.

One non-negotiable core principle was accepted by the debate: the treatment and care of all patients diagnosed with a sarcoma should be under the supervision and management of a multidisciplinary team of sarcoma experts with consensus medical decision-making determined at a team meeting or Board.

Accreditation

The challenge of how to certify provision meeting the definition was discussed. There are two elements which are challenging.

• Inspection. Even using today’s remote systems and with publicly available data inspection is time-consuming and expensive.
• Withdrawal of a certification, as such decisions have financial implications and may result in contested decisions.

The Sarcoma Foundation of America has been using a scheme for some years which relies on internal audit, self-certification against a checklist, with centers being reminded that they need to audit afresh when staff change [17].The Netherlands uses a system which limits validity of accreditation to 5 years. At that point an application to renew has to be submitted with required documentation including support from patient groups and specialized references.

Networks vs. Centers

There was discussion about ‘networks’ as opposed to ‘centers’. It was recognized that long-standing networks such as those in Scotland or France [18] offer a model which contrasted with the ‘expert single-site’ model used in countries such as Scandinavia, the Netherlands, and the USA. It was also noted that England had started with an ‘expert single-site’ model [19] which has evolved into a mixed system with ‘surgical center plus local network’ in some regions.

This interdependence of ‘centers’ and ‘networks’ was recognized, the terms do not compete. Different types and forms of networking were also accepted, ranging from formalized structural relationships to functional and process-led collaborations. Geography may also affect networking decisions. A network approach enables a single clinician with highly specialist skills to work with more than one network if that ensures the best outcomes for patients. It also allows for distributed expertise which enables consultations closer to home for patients for initial diagnostic work, potentially for some treatment and for post-treatment follow-up.

Definition of Expertise

There was a need to define the expertise which should be available and to provide supporting ideas about how quality can be evaluated and described. Experience and knowledge built through a caseload underpins the learning process which every doctor undergoes. The question of how this can be collectively maximized for patient benefit is a key feature of multidisciplinary patient care.

There is value in a ‘reference network’, with the experience of EURACAN [20] opening up cross-border access to relevant expertise being an example. The point was also made that unintentional effects of regulatory decisions can affect the treatment of rarer diseases. In the USA medical licensing does not allow the practice of medicine outside the state in which a clinician is licensed, limiting the way that out-of-state specialists can support colleagues or advise patients directly even when they have unique experience to offer. Instead, the patient has to travel to the state where the sarcoma expert is licensed, creating a disparity for those with low economic capabilities, small children, or disability precluding travel. The value of being able to garner advice from a clinician with particular expertise or experience when a specific problem in treatment or care emerges was pointed out, with patients as well as their clinicians wanting to have direct access to such advice.

The role which new technology can play in resolving the challenges of specialist provision is quite clear. Digital pathology and radiology are already positively acting on the diagnostic process. Great steps have also been made as a direct result of the pandemic. A cultural shift which accepts non-face-to-face consultation is apparent in many places reducing the impact of transport time and costs as a barrier to expert care, although clearly many clinical interventions still require travel.

Initiatives such as EURACAN also allow a cross-border consideration of the opportunities opened up by digital technology [7]. Specialist centers and networks generate valuable data and co-ordinated registries using common data models allow collective analysis, which in a rare disease adds value. Supporting the development of expertise in this way is an important part of the clinical future for sarcoma care.

The importance of the MDM/Board principle in sarcoma care is best evidenced by treatment delays when a multidisciplinary approach is not in place. The evidence indicates that diagnostic delays can be partly attributed to patient ignorance but they are not patient-driven once concern has been raised [21,22]. The impact of clinical systems and procedures, often involving formal referral, is that they delay patient access to treatment which the MDM/Board approach reduces [23]. Pressure for faster, accurate diagnosis is a message from patient groups everywhere [24]

A number of further issues for a specialist network or center were raised. They included:

• Mentorship and training
• Communication with the patient is fundamental
• Accepting feedback from the patient
• A national ‘Gold Standard’ offering a set of ‘base values’
• Focus on outcomes and process - well-designed processes reduce treatment delay
• Success is multifactorial

3. Results

The proposed Term for a Specialist Sarcoma Service

Following this discussion, it was clear that the characteristics of the term/phrase we propose adopting mean that it:

can be a co-located specialized team which involves all core specialties, which draws in other specialties when needed and meets as an MDM/Board

and

may develop a network of providers to reach distant patients which it is confident can deliver appropriate care/treatment under guidance

or

can be a dispersed group of individual expert practitioners who come together regularly to discuss patients (MDM/ Board) with ad hoc associated specialists (e.g. from a Reference Network)

The creation of the over-arching term ‘intelligent specialist network’ goes beyond ‘reference’ and ‘expert’. The word ‘network’ indicates that provision of care does not have to be at a single-site. The word ‘intelligent’ reflects the fact that in multi-site provision there is sometimes the need for highly specialized knowledge or skills not usually required and therefore not immediately available within the local network. The type or form of a network will be influenced by local considerations and it is therefore not defined further. At the same time, the term recognizes that many specialist single-site MDTs will continue to use the term ‘specialist center’ and the choice of ‘intelligent specialist network’ does not disqualify that description.

We therefore propose that the generic term should be:

Sarcoma Intelligent Specialist Network

The context in which it is used will be similar to: “people with sarcoma should be treated in a sarcoma intelligent specialist network”.

The Core Principles

A patient’s primary need is for accurate and timely diagnosis followed by safe high-quality treatment, with curative intent if possible, as close to home as is practicable.

Therefore, the primary and over-arching core principle is that:

The treatment and care of all patients diagnosed with a sarcoma should be under the supervision and management of a multidisciplinary team of experts.

This may be provided through a single meeting or multiple meetings within a team of clinicians acknowledging that they work with one another. All relevant skills which address the needs of the patient(s) being discussed should be present. Presence may be in-person or ‘virtual’ (by phone or video). There should be a regular formal meeting which may be called a Sarcoma Board, Multidisciplinary Team Meeting (MDT Meeting), Team Meeting or similar. We are using the term MDM/Board in this paper. It is appropriate that where extra-specialist knowledge or skills (e.g. exceptional surgery, treatment of rare anatomic locations, proton beam, nuclear medicine) are required a relevant member of the team may consult with a provider of such experience/skills bringing them into the meeting if relevant to do so.

The key characteristic of multidisciplinary management is that formal referral between clinicians is not a requirement, it is a function of team management.

If the meeting is to function as intended it requires imaging and pathology slides and reports, with presence of the specialist at the meeting if required. This is indicated in NCCN Guidelines [25] and ESMO Guidance for the treatment of sarcoma [26]. Therefore, there are two further Core Principles:

Imaging modalities must be available with MRI as appropriate.

An experienced sarcoma specialist pathologist should either be the primary reviewer of biopsy/tissue sample, or the provider of a second/confirmatory opinion.

Who Should be a MDM/Team Member?

The multidisciplinary team approach relies on all patients being discussed and consensus treatment decisions reached through the consideration of all relevant clinical expertise. This is especially important at the initial stage following diagnosis when curative intent is the primary aim and adjuvant or neo-adjuvant therapies are considered.

Multidisciplinary patient management is demonstrated through formal meetings of the whole team on a regular basis with individual patient decisions being recorded and the ‘next step’ clinical responsibility decided. This MDM/ Board meeting will have a locally recognized name and will observe clinical guidelines that have national or international recognition. There will be instances where smaller less formal meetings between clinicians make certain treatment decisions but these should be brought to the full formal meeting for confirmation.

The membership of the formal multidisciplinary team should be disclosed and available to patients. In each case below the * (starred) member should be an experienced sarcoma specialist and is a mandatory requirement. They are:

• *Surgeon(s) with specialist experience in treating sarcoma
• Surgeon(s) with special interest e.g. retroperitoneal, head & neck, endoprosthetics, thoracic
• *Histopathologist
• *Radiologist – imaging/diagnostic
• *Oncologist with radiotherapy expertise
• *Oncologist (medical) with drugs/medicines expertise
• Oncologist (pediatric) with experience treating children/Adolescents & Young Adults (AYA)
• Radiologist – interventional
• Palliative care specialist physician
• Sarcoma specialist nurse (see Note1 below)
• Physiotherapy/rehabilitation practitioners
• Fertility and sexual health especially with AYA
• Psychologist

Other clinical expertise should be brought in as required if they are not among the special interests or experience of those above. These areas of expertise may include (not an exclusive list):

• Ablative therapies
• Medical physics/nuclear medicine (including PET)
• Surgical sub-specialties and expertise (including robotics)
• Novel therapies (including immunotherapy)
• Genetics

Recommendation

The Sarcoma Intelligent Specialist Network must maintain contact with providers of clinical services which are used relatively infrequently, regularly review such contacts and have clear methods for bringing in such expertise.

Leadership of an MDM/Board will be a senior clinician. Succession planning is not always a feature of clinical management and quite often succession is left to chance. For stability and for ensuring that a Network continues to meet all the requisite Principles and Features, we have a further Recommendation.

Recommendation

The Specialist Network can identify suitable locum expertise and when a specialist member of the clinical team moves on to quickly fill the role and ensure that the level of expertise offered to patients does not diminish.

Note1: The value of the sarcoma nurse specialist has been shown and patients regard sarcoma nurse specialists as essential. One of the roles is to support patients through the diagnostic and primary treatment process. Where there are national practice issues which make such nursing provision problematic this role should highlighted as essential and its provision should be an aspiration wherever possible.

Recommendation

Sarcoma specialist nurse(s) are in post and seen as key members of the team.

Additional Features of a Sarcoma Intelligent Specialist Network

It became clear during the discussion that patient needs can be defined in four features of a sarcoma intelligent specialist network:

1. Expertise

2. Knowledge

3. Resources

4. Access

The label of Sarcoma Intelligent Specialist Network should feel aspirational. The following are Recommendations which may be adapted to address national, regional or local considerations, especially when budgets are considered.

1. Expertise

It is accepted that the caseload a Network handles will underpin the way its expertise develops and grows [27,28]. Therefore, a high case volume should be a mandatory requirement for Sarcoma Intelligent Specialist Networks.

The incidence of sarcoma is poorly understood. Overall incidence rates of 70-80 per million of population come from Europe while the USA records about 40 per million. Patient stories tell us that there will be late diagnoses with advanced disease, some patients will come through referral after treatment which reveals sarcoma (e.g. uLMS), some will be GIST going straight to a medical therapy from a GI specialist, so it is not easy to estimate the sarcoma caseload for a Network and the scale of population which would support it.

In the USA the Sarcoma Foundation uses a caseload of 50 patients per annum as the minimum to establish expertise. The same applies in Germany with the threshold defined by the German Cancer Society [29]. To reach this would probably require a population of 2-2.5 million. The economic calculations used for England indicated a base of 5 million population to justify a specialist center, although this is distorted because some of its centers have a national as well as a regional/local role. It set a minimum standard at 100 cases per year and one center which had been initially authorized was closed after five years for low numbers. In Italy the national recommendation is for 100 soft tissue and 50 bone cases each year.

There have been data presented in TARPSWG (Transatlantic Australasian Retroperitoneal Sarcoma Working Group) meetings on surgery for retroperitoneal sarcoma that support a caseload of 24 per annum to define expertise in treating this specific sarcoma patient group. Such work supports our approach to using caseload as an indicator of expertise. Local decisions on caseload for pediatric units would need to be made.

It is unlikely that single institutions in small countries with a small population will be able to treat this number of cases so thresholds should be realistic but not reduced to accommodate more Centers with less expertise. Thus, a nation with a population <5m could be appropriately served by one Network structured to meet its geographical realities.

Recommendation

A Sarcoma Intelligent Specialist Network should have a caseload of 100+ sarcoma patients each year, with 50 newly diagnosed patients, to assume appropriate sarcoma expertise.

4. Knowledge

Knowledge is best gained through initial training and continuing education. The contact which a Network’s clinicians have with other sarcoma specialists through formal opportunities such as exchanges, fellowships, training, oncology conferences and other events is important. The active involvement of surgeons and oncologists undergoing training or immediately following initial qualification should be part of the Network’s routine function. Those wishing to follow a pathway to sarcoma specialty should be encouraged.

5. Resources

We do not define detailed provision of technical equipment, so we discuss the overall picture of emerging technology. Radiographic imaging in particular is rapidly evolving, so too is access to specialized techniques such as isolated limb perfusion, ablative procedures, or proton beam therapy. This technology will ensure a high level of quality care for sarcoma patients.

A Sarcoma Intelligent Specialist Network should have access to new technology and imaging techniques as well as to specialized treatment options

Genomic services: This is an area which is fast developing. The case for Whole Genome Sequencing at first diagnosis is being actively argued. There is a case for understanding the genomic mutations present in advanced disease as the science of treating by histology+mutation or by mutation alone, rather than by histology alone, is rapidly advancing. There is evidence in select subtypes (notably GIST) where NGS (Next Generation Sequencing) should be performed prior to initiation of systemic therapy. It is likely that the number of sub-types and relevant new therapies will grow.

There is also growing knowledge of germ-line mutations such as the P53 related Li Fraumeni syndrome which if diagnosed in a family has significant implications.

6. Access

Promoting good quality and safe care of patients across the Network should be a primary aim. While the Network will aim to assure the quality of its processes it is equally important to assure the quality of each patient’s journey through the care pathway. The EU Joint Action on Rare Cancers introduces the concept of the “network patient” who is treated according to the Network’s clinical practice guidelines, exploits the clinical expertise available in the Network and is registered in the Network database [30].

Similarly, the concept of ‘shared decision-making’ is increasingly recognized as important for the effective treatment of patients, especially when complex decisions which can impact quality-of-life are required [31].

Patient Experience

Patient Reported Outcomes are becoming generally recognized as adding value in research. The increasing availability of sarcoma relevant PRO tools is allowing evaluation of patient experience and satisfaction along the entire patient pathway. While we have no information that indicates these tools are currently being widely used in this way we believe that patient experience and assessment can exert a direct impact on a Network and help raise the quality of the outcomes it achieves.

Recommendation

A Sarcoma Intelligent Specialist Network collects and analyses data on patient experience and uses it within its self-audit and improvement planning.

Patient Support Group

There is real value to patients in a patient support group, or groups, associated with the Network. The quality of the patient experience is improved by exchanging experiences with those who have faced something similar. This maybe through social media but is enhanced by face-to-face contact.

Recommendation

The Network should facilitate patient support groups in the Network, while encouraging patient-leadership and management of such groups

Groups may come together at a national level to allow a single patient voice to be expressed in the national healthcare system and to build relationships with groups in other countries through membership of SPAGN. Ideally, each site or network should have a sarcoma-specific patient support group which can provide support to patients in the context of local needs, provide advice to both patients and to the MDM/Board when required, and help its national group in developing the patient voice.

Patient advocacy groups may also develop research projects, sometimes working with clinical professional partners, sometimes looking at non-clinical issues on their own. Patient-led research is a development which should be encouraged. There is also a growth of evidence-based advocacy which can bring patient-led data to the fore in decision processes and policy-making. A strong example is seen in the work of SPAGN and the Netherlands Cancer Institute on the PPRN Priority Setting Partnership [33].

Further Discussion Points

Further points were made in discussion which although not specifically addressed within the main text should be noted. They create part of the underlying ‘tone’ and ambition of this work.

These points:

The patient wants timely accurate diagnosis and to know/understand treatment options.

Patients should be told if there is a treatment center closer to home.

Look at outcomes not process but recognize that good process delivers good outcomes

Develop Intelligence through research, gathering and using data which is both patient and activity oriented

Be clear about the ‘added value’ specialists bring to patient care

Recognize realities, be practical and creative