Guideline development in rare cancer:

a combined patient-physician effort

in sarcoma

Peter Hohenberger

Div. of Surgical Oncology & Thoracic Surgery

UMM Mannheim

University of Heidelberg, Germany

Guideline development in rare cancer:

a combined patient-physician effort

in sarcoma

……. with the experience derived from sarcoma

to be used for other rare cancers

Problems of rare cancers

� Unexperienced physicians outside expertise centers

� Expert patients often have more expertise than

unexperienced physicians

� Well-trained patients

(knowledge about methodology, data on toxicity

not officially reported, PROs)

� Not much data from randomised studies available

� Radiation therapy

� Chemotherapy

� Radio-Chemotherapy

� Isolated limb perfusion

� Chemotherapy + hyperthermia

AJCC stage III sarcomas: neoadjuvant treatment options

Facts

� In sarcoma, there is not much (good) Cochrane

evidence

� Not many prospective randomized phase III

studies

� How good are guidelines ?

� LoE ? Best level: big randomized phase III studies

Even if we think:

EORTC 62012: No patients beyond 60years

Age distributionof sarcomas in Germany, epidemiological data

Zeissig et al. 2017, submitted

Main aspect

What would an experienced, well-trained, well-

informed patient:

� - accept as a treatment and

� - recommend to other patients

� - important contribution

Since 2007

FP6 & FP7 projects on sarcoma & RC

� Analysis of current practice

� Description of weaknesses

� Comparison between countries

� Expectations of patients

JARC working packages

Peter Hohenberger, MD

Div. of Surgical Oncology & Thoracic Surgery

Mannheim University Medical Center

University of Heidelberg, Germany

WP 6 Clinical practice guidelines

WP 6 Clinical practice guidelines

� Actions to assess the scenario of existing

clinical practice guidelines on RCs

� To provide recommendations on

how to ensure best production,

dissemination and implementation of

clinical practice guidelines on RCs

� at local level and within future ERNs

� This WP will consider all the already available and ongoing

efforts in the field of guidelines on cancers& rare cancers

� Will collaborate with key players in this areas: European

Society of Medical Oncology (ESMO),

State of Art in oncology (START),

European Cancer Patient Coalition (ECPC),

EURORDIS,

OECI,

Orphanet,

Istituto Superiore di Sanità (ISS - Rare Best Practice), M

MAPPING_NCD (http://www.ncd-map.eu.)

� Experiences available outside Europe will be also considered.

WP 6 Clinical practice guidelines

Objectives:

� to map available clinical practice guidelines at the European level on all families of rare cancers and identify gaps;

� to provide a quality evaluation of existing clinical practice guidelines for rare cancer subtypes

� to identify open issues about implementation of clinical practice guidelines at the local level

� to work out solutions on how to incorporate clinical practice guidelines within ERNs

� Not: to create guidelines

WP 6 Clinical practice guidelines

� Explore the existing database of clinical practice guidelines at

INSERM (Orphanet).

� Collect the existing clinical practice guidelines produced by

European societies/efforts/etc, such as ESMO, START

considering the work already undertaken by RARECAREnet.

� Carry out a survey within OECI among its member

institutions about which clinical practice guidelines are used

in clinical practice.

� Carry out a survey within the Network of Comprehensive

Cancer Centers in Germany, INCa in France, Cancer Research

UK and its member institutions in the UK.

� Lead partner: INSERM

WP 6/ T6.1 Available clinical practice guidelines

at the European level on all families of RCs

� Assess accessibility of existing CPGs for physicians and patients - online

and printed

� Assess the degree of updating of CPGs over time.

� Validation process of guidelines (consensus mechanisms, peer-review, ... )

� Assess the contribution of partners during guideline development (uni- vs.

multi-centricity, uni- vs. multi-disciplinarity, involvement of PAGs, etc).

� Finally, score CPGs according to available quality criteria.

� Summaries referring to guideline availability and quality per family of RC

� Patients Advocacy Groups (PAGs) will contribute by assessing the impact of

patients to influence the development of guidelines

� Lead partner: DKG.

WP 6/ T6.2 Quality evaluation of existing clinical

practice guidelines for rare cancer subtypes

Table to Task 6.2

Cancer

family

H

&

N

Thoracic Male

GU

Female

GU

Neuro-

endocrine

Endocri

ne CNS Sarcoma

Guideline1 subpar Guideline2 ok Guideline3 good Guideline4 superb Respon-

sibility of

assessment

DKG, ECPC, SIOP, GPOH, EURORDIS combine their expertise in assessing quality of guidelines in the rare cancer

families

Intended result: map of guidelines and their quality

WP 6 Milestones

M6.1 Results of the surveys of guidelines within OECI, Network of

Comprehensive Cancer Centers Germany, INCa and Cancer Research UK

and other interested stakeholders

(Task 6.1, M12)

M6.2 List of criteria to evaluate the clinical practice guidelines available

(Task 6.2, M15)

M6.3 Results of the “pilot assessments” of the interplay between clinical

practice guidelines, practicing physicians and local reimbursement

mechanisms

(Task 6.3, M20)

M6.4 Report on possible solutions for assessing the effect of improving

cancer care by clinical guidelines implementation at population level

(Task 6.4, M24)

WP 6 Deliverables

D6.1 Report and survey on availability of clinical practice

guidelines for all rare cancer families

(M18)

D6.2 Report on the assessment of the quality of existing

clinical practice guidelines

(for each family of rare cancers, M24)

D6.3 Recommendations on optimal implementation of

clinical practice guidelines on rare cancers (M30)

D6.4 Recommendations on how to integrate clinical practice

guidelines into existing healthcare networks, with special

reference to ERNs (M36)

� Special regard: relation with local reimbursement mechanisms

� To study innovative models to provide value-based RC care

� study the interplay between clinical practice guidelines and local

reimbursement mechanisms with “pilot assessments”

� assess accessibility of available clinical practice guidelines and

awareness thereof by the oncology community.

� identify gaps in available guidelines at the European level with

regard to some rare cancers.

� provide recommendations to improve quality of guidelines and to

make them more broadly available (language, dissemination)

� try to identify target “communities”, with a view to multi-

disciplinarity (involving all professional oncology societies).

� Lead partner: DKG

WP 6/ T6.3 Open issues about implementation of clinical

practice guidelines at the local level

� Consider disease outcomes on a country basis, in collaboration with

the WP4 and European cancer registries,

� Use results of the RARECAREnet high resolution and pilot studies to

evaluate to which extent the use of CPGs could improve.

� Review data about adherence to guidelines and its impact on disease

outcome in rare cancers.

� Study models to integrate clinical practice guidelines into existing

healthcare networks, with special reference to ERNs.

� Discuss with European cancer registries possible ways to assess the

implementation of CPGs at population level (collab: Task5, WP4).

� Lead partner: DKG.

WP 6/ T6.4 Solutions on how to incorporate

clinical practice guidelines within ERNs.

We did that already ! And it works.

� Chordoma

� Desmoid

We did that already ! And it works.

� Chordoma

� Desmoid

ERNssince Dec 16, 2016

1 BOND ERN on Rare Bone Disorders

2 CRANIO ERN on Rare craniofacial anomalies and ENT disorders

3 Endo-ERN ERN on Rare Endocrine Conditions

4 EpiCARE ERN on Rare and Complex Epilepsies

5 ERKNet European Rare Kidney Diseases Reference Network

6 ERN-RND ERN on Rare Neurological Diseases

7 ERNICA ERN on Rare inherited and congenital anomalies

8 ERN-LUNG European Reference Network on Rare Respiratory Diseases

9 ERN-SKIN ERN on Rare and Undiagnosed Skin Disorders

10 EURACAN ERN on Rare Adult Cancers (solid tumors)

11 EuroBloodNet ERN on Rare Hematological Diseases

12 EURO-NMD ERN for Rare Neuromuscular Diseases

13 ERN-EYE ERN on Rare Eye Diseases

14 ERN GENTURIS ERN on GENetic TUmour RIsk Syndromes

15 GUARD-HEART Gateway to Uncommon And Rare Diseases of the HEART

16 ITHACA ERN on Rare Congenital Malformations and Rare Intellectual Disability

17 MetabERN ERN for Rare Hereditary Metabolic Disorders

18 PaedCan-ERN ERN for Paediatric Cancer (haemato-oncology)

19 RARE-LIVER ERN on Rare Hepatological Diseases

20 ReCONNET Rare Connective Tissue and Musculoskeletal Diseases Network

21 RITA Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases

22 TRANSCHILD ERN on Transplantation in Children (incl. HSCT, heart, kidney, liver, intestinal, lung and

multiorgan)

23 VASCern ERN on Rare Multisystemic Vascular Diseases