Access to Clinical Trials International Initiatives

Transcription

1 Access to Clinical Trials International Initiatives Stefan Bielack Cooperative Osteosarcoma Study Group COSS Stuttgart Cancer Center Klinikum Stuttgart Olgahospital Zentrum für Kinder-, Jugend- und Frauenmedizin Pädiatrie 5 (Onkologie, Hämatologie, Immunologie) s.bielack@klinikum-stuttgart.de

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3 AYA: (Some) reasons for prognostic disadvantages diagnostic delays => tumor load

4 AYA: (Some) reasons for prognostic disadvantages diagnostic delays => tumor load distinct tumor biology distinct pharmacology

5 AYA need not experience more toxicity than children! RMS (COG, n=657) AYA => less marrow & mucosal tox., more neuropathy (Gupta et al., Cancer. 2012) Ewing (EURO-E.W.I.N.G. 99, n=851) AYA => less marrw & mucosal tox, vomiting (Juergens et al., Pediatr Blood Cancer 2006) Osteo (Livestrong metaanalysis, n=4,838) AYA => less thrombopenia, neutropenia, DOC (Collins et al., J Clin Oncol 2013)

6 AYA: (Some) reasons for prognostic disadvantages diagnostic delays => tumor load distinct tumor biology distinct pharmacology compliance treatment infrastructures

7 Pediatric n DFS Adult n DFS FRALLE % (6 years) LALA % (4 years) FRALLE % (5 years) LALA % (5 years) CCG % (6 years) CALGB % (6 years) AIEOP % (OS, 2 years) GIMEMA 95 71% (OS, 2 years) DCOG % (5 years) HOVON 44 37% (5 years)

8 AYA: (Some) reasons for prognostic disadvantages diagnostic delays => tumor load distinct tumor biology distinct pharmacology compliance treatment infrastructures lack of trial participation

9 AYA: Trial participation Bleyer WA. The adolescent gap in cancer treatment. J Registry Manage 1996; 23: 114-5

10 Age and survival improvements Bleyer, Med Pediatr Oncol 2002

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15 Distinct AYA outcomes Lack of trial participation why? (Non-) Availability Lack of recruitment if available

16 AYA Lack of available trials / industry sponsored Orphan diseases with limited industry interest Traditional adult trials only start at age 18 Pediatric phase I/II trials often - start late (after adult license) - based upon disease instead mode of action - stop at age 18 ( PIP ) - don t recruit very well

17 AYA trials Recent initiatives to bridge the 18 age gap

18 AYA trials Recent initiatives to bridge the 18 age gap

19 AYA trials Recent initiatives to bridge the 18 age gap

20 Distinct AYA outcomes Lack of trial participation why? (Non-) Availability Lack of recruitment if available

21 AYA Lack of trial recruitment Example: Osteosarcoma an AYA cancer

22 European and American Osteosarcoma Study COG Childrens Oncology Oncology Group Group COSS COSS Cooperative Osteosarcoma Study Group Cooperative Osteosarcoma Study Group EOI EOI European Osteosarcoma Intergroup European Osteosarcoma Intergroup SSG Scandinavian Sarcoma Group SSG Scandinavian Sarcoma Group eligible: 40 years at osteosarcoma diagnosis

23 EURAMOS Recruitment 04/05 06/ patients 326 institutions from 17 countries in Group Freq. Percent Cum COG 1, COSS EOI SSG Total 2, Thanks to EURAMOS-CDC, MRC London

24 AYA Lack of trial recruitment Example: Osteosarcoma an AYA cancer

25 European and American Osteosarcoma Study EURAMOS Age-specific recruitment

26 EURAMOS: Age-specific recruitment 1,4 different 1,2 1 SEER EURAMOS 0,8 0,6 Medical Oncology missing (ca. 600 pts) 0,4 0,

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28 France: Different trial, same lack of adult participation 80% Ped 20% Med

29 Would it be feasible to perform such trials only in selected large centers?

30 EURAMOS Patients per center Pts/year centers pts <1 185 (56.4%) 487 (22.5%) (27.4%) 688 (31.7%) (10.4%) 485 (22.3%) (4.3%) 280 (12.9%) (0.6%) 53 (2.4%)

31 Is it easy to run a trial -in many centers? -in your own center?

32 ICH Topic E6 Guideline for Good Clinical Practice

33 Documentation to be held by investigator/institution for clinical trials Investigators brochure (+ updates) or SmPC Protocol and amendments (signed) Information sheet and consent form (+ updates) Financial aspects Insurance statements Signed agreements between parties EC opinion and composition MRHA authorisation Investigators CVs Medical and laboratory tests, including normal ranges Medicine labels Instructions for medicine use Shipping records Certificates of analysis Decoding procedures Master randomisation list Monitoring reports (pre-trial, initiation, close-out etc) List of persons responsibilities delegated to (+ updates) CRFs and corrections SAE notifications from investigators and to EC and MRHA EC/MRHA annual reports and final reports Subject screening log Subject identification code list Subject enrolment log IMP accountability at site Record of retained tissues Documentation of IMP destruction Completed subject identification code list Audit certificate Clinical study report

34 How to proceed for better AYA care? Perform trials across the age ranges Take the effort and open (investigator-initiated) trials at your center! Enrol your (AYA) patients!

35 Access to Clinical Trials International Initiatives Stefan Bielack Cooperative Osteosarcoma Study Group COSS Stuttgart Cancer Center Klinikum Stuttgart Olgahospital Zentrum für Kinder-, Jugend- und Frauenmedizin Pädiatrie 5 (Onkologie, Hämatologie, Immunologie) s.bielack@klinikum-stuttgart.de

36 European Societies

37 Controversy session: Adolescent oncology: pediatric or adult territory? Istanbul, 1 October 2006, 13:45-14:45 Presentation 1: Pediatric view speaker Stefan Bielack, DE Presentation 2: Adult view speaker Ian Judson, UK Presentation 3: Advocate for multidisciplinary team of both pediatric and adult oncologists Gunnar Saeter, NO

38 EXECUTIVE SUMMARY OF SIOPE EUROPEAN CANCER PLAN FOR CHILDREN AND ADOLESCENTS Cancer in young people is rare, but it is still a major health issue in Europe. Each year, more than 6,000 young people in Europe die of cancer. There are more than 300,000 European childhood cancer survivors (in 2020, they will be nearly half a million): two-thirds of them have some late side effects of treatment, that are severe and impact on the daily life of half of those affected. Within the European Network for Cancer research in Children and Adolescents (ENCCA), SIOPE and the European paediatric haematology-oncology community have established a long-term sustainable Strategic Plan to increase the cure rate and the quality of survivorship for children and young people with cancer over the next ten years. The ultimate goal is to increase the disease- and lateeffect- free survival after 10 years from the diagnosis, and beyond. Seven medical and scientific objectives have been set up to achieve these goals: Innovative treatments:... Precision cancer medicine:... Tumour biology:... Equal access:... TYA: to address the specific needs of teenagers and young adults (TYA), in cooperation with adult oncology Quality of survivorship:... Causes of cancer... SIOPE will steer and coordinate the effective implementation of this Strategic Plan, together with the European Clinical Trial Groups (ECTGs) and the National Paediatric Haematology Oncology Societies (NAPHOS), in close cooperation with the parents, patients, and survivors advocates from the European Regional Committee of Childhood Cancer International (CCI)....

39 Cancer in Adolescents and Young Adults (AYA) Working Group

40 Cancer in Adolescents and Young Adults (AYA) Working Group Mission of the Joint ESMO-SIOPE Working Group on Cancer in Adolescents and Young Adults (AYA) The goal of the joint Working Group s to promote education in cancer topics specific to this population. It is a joint venture between the European Society for Medical Oncology (ESMO) and the European Society for Pediatric Oncology (SIOPE) created in 2016, following collaborative efforts in the educational arena between the two societies since ESMO-SIOPE AYA Activities and Responsibilities (1) Increase awareness amongst the medical and pediatric oncology communities & enhance knowledge on specific AYA cancer issues Serve as a primary resource for new CME opportunities through production of specific educational and scientific programs. Liaison with other ESMO committees when specific actions are needed, e.g. providing advice at policy level to optimize approaches to prevention, diagnosis, treatment and long term follow-up of these specific patient populations. Selected AYA Working Group Accomplishments Regular teleconferences & face-to-face meetings (Yes!!! Medical and pediatric oncologists talk to each other!) Dedicated space on the ESMO website Committee/Adolescents-and-Young-Adults-Working-Group e-learning modules on Cancer in adolescents and young adults Breast cancer in young women Colorectal cancer in young adults: The focus on hereditary cancer syndromes Development of further modules in progress Joint ESMO-SIOPE AYA symposia at ESMO conferences Agreement on producing a joint handbook on AYA cancer ESMO-SIOPE Survey on AYA Care

41 European Network

42 European Network

43 National activities by medical and pediatric oncologists. and many more

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45 Thank you! Stefan Bielack