Patient participation in clinical research. Melanoma Bridge meeting Naples 3 rd Dec 2014 Melanoma Patient Network Europe Bettina Ryll, MD/PhD www.melanomapatientnetworkeu.org
Jul 17, 2015
Patient participation in clinical research.
Melanoma Bridge meetingNaples 3rd Dec 2014
Melanoma Patient Network Europe
Bettina Ryll, MD/PhD
www.melanomapatientnetworkeu.org
potential conflict of interests
At this time,
Advisory function to the following Pharma: Amgen, GSK, Merck-Serono, Novartis, Roche.All honoraries received are handled by the Anticancer Fund, Belgium, on MPNE’s behalf and used for the work of MPNE.
MPNE projects are funded by grants/ sponsorships from the pharmaceutical industry: funding is always balanced and does not entail any rights to influence the concept/ faculty/ program. (Amgen, BMS, GSK, MSD, Merck-Serono, Novartis, Roche)
WMA Helsinki DeclarationEthical Principles for Medical Research involving Human Subjects- 1964, last amendment 2013
3. The declaration of Geneva of the WMA binds the physicians with the words ‘The health of my patient will be my first consideration’ and the International Code of Medical Ethics declares that, ‘A physician shall act in the patient’s best interest when providing medical care’.
…
8. While the primary purpose of medical research is to generate new knowledge, this goal can never take precedence over the rights and interests of individual research subjects.
….
26. …The potential subject must be informed of the right to refuse to participate in the study or to withdraw consent to participate at any time without reprisal. ….
Show me the evidence-
• unethical trials violating equipoise, inappropriate use of blinding and placebos
• confirming rather than learning trials • ‘Pharmageddon’- ever-decreasing ROI in Pharma• increasing drug prices- resulting in access problems
for our patients
… all in the name of evidence-based medicine.
“Avoidable waste in the production and reporting of research evidence”Iain Chalmers, Paul Glasziou, The Lancet, 15 June 2009, doi:10.1016/S0140-6736(09)60329-9
Over 30% of trial interventions not sufficiently described
Over 50% of planned study outcomes not reported
Most new research not interpreted in the context of systematic assessment of other relevant evidence
Unbiased and usable report?
85% research waste = over $85 billion / year
Low priority questions addressed
Important outcomes not assessed
Clinicians and patients not involved in setting research agendas
Questions relevant
to clinicians & patients?
Over 50% studies designed without reference to systematic reviews of existing evidence
Over 50% of studies fail to take adequate steps to reduce biases, e.g. unconcealed treatment allocation
Appropriate design and methods?
Over 50% of studies never published in full
Biased under-reporting of studies with disappointing results
Accessible full
publication?
1 2 3 4
slide courtesy J. Geissler
Only patients know what patients know- 1
Detecting Myeloma, ways to shortening an often painful and tedious patient odyssey: results from an international
survey. Myeloma Euronet (2009). 314 physicians & nurses, 260 patients & carers, 43 countries
slide courtesy J. Geissler
Only patients know what patients know- 2
Questions:
Forget medication
Miss for other reason
Stopped because felt worse
Forget when travelling
Take yesterday?
Stop when under control
Inconvenience
How often difficulty remembering
Low: 21 %
Medium: 47 %
High: 33 %
Adherence score classifies patients
into adherence groups:
F1/ base = all respondents (n=2546) Low (n=528), Medium (n=1185), High (n=833) - Please answer ‘yes’ or ‘no’ to each question based on your personal experience with your CML medication. F2/ base = all respondents (n=2546) Low (n=528), Medium (n=1185), High (n=833) - How often do you have difficulty remembering to take all your medications?Use of the ©MMAS is protected by US copyright laws. Permission for use is required. A license agreement is available from: Donald E. Morisky, ScD, ScM, MSPH, Professor, Department of Community Health
Sciences, UCLA School of Public Health, 650 Charles E. Young Drive South, Los Angeles, CA 90095-1772.
original slide courtesy J. Geissler
Adherence Study by the CML advocate network- MEMS®, Morisky Adherence Scale
2546 CML patientsin 79 countries
DATA NOT OPINIONS
• Everyone is biased.
• Patients’ preferences are as diverse as the preferences of any other stakeholder group-there is no single medical KOL voice either.
• We need scientific tools to account for bias and diversity.
trial management, e.g. consent, patient
information leaflets, trial adherence
patient recruitmente.g. providing information on clinical trials, advertising trials
clinical trial design
Patient involvement in clinical trial design
12/4/2014
clinical question/ problem
current patient involvement
impact on patients
doing the right thing doing things right
http://www.informed-scientist.org/presentation/the-role-of-patient-groups-in-the-clinical-trial-process B. RYLL
Current scenario:
Post-licensing, treatment population grows rapidly; treatment experience does not contribute to evidence generation
Adaptive Licensing:
after initial license, number of treated patients grows more slowly, due to restrictions; patient experience is captured to contribute to real-world information
Eichler HG et al. Clin Pharmacol Ther. 2012
11
Innovative Drug Licensing Models-e.g. Adaptive Licensing
original slide courtesy F. Pignatti
Melanoma patients need
• ethical trials on the basis of Helsinki respecting patients’ real and not perceived or projected interests.
• a sustainable drug development system ensuring innovative, effective and safe enough drugs at an affordable price.
• sophisticated models for trial design- e.g. Bayesian designs-and innovative drug development models- like Adaptive Licensing, break-through designation- to account for the specific condition of high unmet need and highly promising new therapeutical developments.
Because evidence-based medicine is the tool, not the goal.
MPNEwww.melanomapatientnetworkeu.orgWe are also on facebook, linkedin, twitter and youtube
Contact Bettina [email protected]
Next MPNE conference‘The risk of not taking risks in Melanoma’
24-26th April 2015, Brussels
Thank you
for your attention