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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)
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
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
~6000
Rare
Diseases
203
Cancers
~1
86
RA
RE
CA
NC
ER
S
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
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
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)
Standard Inferior therapy
52%
48%
75%
25%
Non-study clinic (n= 68)
3 times higher
chance to
receive a better
therapy!
Source: Standard therapy in ,FIGO I-IIA: Study- vs Non-Study clinic, German Quality program QIII 2001 AGO OVAR
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
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
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)
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
?
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
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)
Where to find trials?
Research networks websites
EU Clinical Trials Register https://www.clinicaltrialsregister.eu
Doctors & Patient organisations
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
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…
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."
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 participation in cancer trials
• Improve informed consent
• Make trial results widely known
• Provide insight on adherence, CAM use, QoL
• Fundraise to make research happen
Research subject
Information provider
Advisor
Reviewer
Co-researcher
Driving force
PatientPartner
FP7 Project (2010)
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
The Patients’ Academy: empowering patients on medical R&D
Rare Cancers: Rare, but not alone.
Jan Geissler
Twitter @jangeissler
http://www.patientsacademy.eu
http://www.cmladvocates.net