"Access to treatment, care and clinical trials by patients with rare cancers", presented by Jan Geissler (Twitter @jangeissler) at ESMO congress in Vienna, 30 Sept 2012

1. Access to treatment,
care and clinical trials by
patients with rare cancers
Jan Geissler
Co-founder, CML Advocates Network
Vice President, Leukemia Patient Advocates Foundation
Secretary, European Forum For Good Clinical Practice
Member, EU Committee of Experts for Rare Diseases (EUCERD)
Director, European Patients‟ Academy on Therapeutic Innovation (EUPATI)

2. Rare but not alone? The journey of
(rare) cancer patients
 1 in 3 Europeans will get cancer
in their lifetime
 1/3 of them develop
a rare cancer
 ~261 cancers are rare
- and very different

3. Cancer, rare cancers, rare diseases
– is there a difference?
 Rare Cancers: often lost between 17 common cancers
and 1000s of rare diseases
 Rare Cancers share many challenges with rare diseases
~186 RARE
CANCERS
203 ~6000
Cancers Rare
Diseases

4. Patients with rare cancers
face specific challenges
1. Prevention and screening mostly irrelevant
2. Late or incorrect diagnosis very common
3. Experienced doctor not available locally,
lack of access to clinical expertise
4. Lack of appropriate therapies
5. Slowness of research (lack of trials & commercial
interest),
6. Facing stigma and inequity
7. Lack of information & local patient groups

5. Patients with rare cancers
face specific challenges
1. Prevention and screening mostly irrelevant
2. Late or incorrect diagnosis very common
3. Experienced doctor not available locally,
lack of access to clinical expertise
4. Lack of appropriate therapies
5. Slowness of research (lack of trials & commercial
interest),
6. Facing stigma and inequity
7. Lack of information & local patient groups

6. Rare Cancers: best to be treated in
an experienced center
 Misdiagnoses common before diagnosed appropriately
 Number of „cases“ in a center, conduct of studies:
Indicator for up-to-date expertise and treatment outcome
Study clinic (n= 56 pts) Non-study clinic (n= 68)
Higher chance to 25%
receive a better
52% 48%
therapy in 75%
study centers
(irrespective of trial
participation)
Standard Inferior therapy
Source: Standard therapy in ,FIGO I-IIA: Study- vs Non-Study clinic, German Quality program QIII 2001 AGO OVAR

7. Special challenges
crossing country borders
 Currently, crossing country borders for health services is
challenging (E112 form, prior authorization, unclear rules)
 „Patients‘ Rights in Cross-Border Healthcare“
Directive 2011/24/EU, in national law by Oct 2013
• To provide clarity and legal certainty on access to diagnosis &
healthcare in other EU Member States, especially when condition
requires particular expertise or resources (e.g. rare diseases)
• National contact points
• Rules for reimbursement
• Procedural guarantees
• Prior authorisation system & reasons to refuse & time limits
• Cooperation between health systems

8. Patients with rare cancers
face specific challenges
1. Prevention and screening mostly irrelevant
2. Late or incorrect diagnosis very common
3. Experienced doctor not available locally,
lack of access to clinical expertise
4. Lack of appropriate therapies
5. Slowness of research (lack of trials & commercial
interest),
6. Facing stigma and inequity
7. Lack of information & local patient groups

9. Unmet medical needs for most
patients with rare cancers
 “Success stories” available only to
small numbers of rare cancers
 Patients in urgent need of
therapeutic innovation
Source: RareCare (2012)

10. Barrier:
Public image of clinical trials
Lack of public confidence in research
?
leads to
 delayed / lack of trial recruitment
 delayed generation of meaningful clinical data
 slow progress or lack of research in Europe
 Good information and support of
patient groups can help to understand

11. Potential benefits of a clinical trial
 Personal benefit:
• Some new therapies only available in trials
• Closer monitoring
• Therapy optimization trials with more effective
or more tolerable regimens
 Altruistic reasons:
• Achieving progress – beyond our personal case

12. Understanding risks of clinical
trials
 Uncertainty always brings risks, but
trials are strongly regulated & assessed
 Risks of participation in trials
• Risks of the unknown: will it work? Will it do harm?
• Impact on quality of life, unpleasant/more diagnostics
• Protection from unauthorized use of data and tissue
 Good information is essential for
informed consent (of the whole family)

13. Where to find trials?
 Research networks„ websites
 EU Clinical Trials Register
https://www.clinicaltrialsregister.eu
 Doctors & Patient organisations

14. Patient organisations are
‚navigators‘ for patients
 Most „official“ information portals
fail on rare cancers:
Budget for the "Top 17".
 Patient organisations inform, help, support
• Explanation of disease, therapy, side effects
• Managing anxiety about disease and doctor
• Information on clinical trials (centers, doctors, results)
• Working in partnership with researchers
• Trustable web resources

15. International Rare Cancer Advocacy
Organisations (just to name some)
 International Brain Tumour Alliance (IBTA) - http://www.theibta.org
 Sarcoma Patients Euronet (SPAEN) - http://www.sarcoma-patients.eu
 CML Advocates Network - http://www.cmladvocates.net
 International Kidney Cancer Coalition (IKCC) - http://www.ikcc.org
 European Cancer Patient Coalition (ECPC) – http://www.ecpc-online.org
 European Rare Disease Organisation (EURORDIS) - http://www.eurordis.org
 European Waldenström Network (EWMNetwork) - http://www.ewmnetwork.eu/
 Myeloma Patients Europe (EMP, http://www.emp-myeloma.eu and ME,
http://www.myeloma-euronet.org)
 Lymphoma Coalition - http://www.lymphomacoalition.org
 Myelodysplastic Syndromes Foundation - www.mds-foundation.org
 Carcinoid & Neuroendocrine Tumor Society - http://www.cnets.org
Carcinoid Cancer Foundation - http://www.carcinoid.org/
 International Confederation Of Childhood Cancer Parent Organizations
(ICCCPO) - http://icccpo.org/
…and many more…

16. Patients – as co-researchers?
 "More needs to be done: rare cancers will never be a
priority unless the patients make it one. Patients
themselves must therefore play a larger role in
driving forward the search for therapies. They are
able to see connections that have eluded scientists."

17. Patients have a complementary
expertise that is invaluable to research
 Patients experience is
complementary, not substitutive
 Examples
• What it means to live with cancer
• Explain the value and barriers of Driving force
participation in cancer trials Co-researcher
Reviewer
• Improve informed consent
• Make trial results widely known
Advisor
• Provide insight on adherence, CAM Information provider
use, QoL Research subject
• Fundraise to make research happen PatientPartner
FP7 Project (2010)

18. The Patients’ Academy:
empowering patients on medical R&D
 Launched Feb 2012, runs for 5 years,
29 consortium members,
PPP of EU Commission and EFPIA
 will develop and provide
objective, credible,
correct, up-to-date knowledge
about medicines R&D
 will build competencies
& expert capacity among patients & public
 will facilitate patient involvement in R&D to support
industry, academia, authorities and ethics committees

19. Rare Cancers:
Rare, but not alone.
Jan Geissler
 jan@patientsacademy.eu
 Twitter @jangeissler
 http://www.patientsacademy.eu
 http://www.cmladvocates.net