Patient Participation in Clinical Research- as early as possible!
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EORTC Patient Course 2014 The trials we need- Patient involvement in clinical trial design Bettina Ryll, MD/PhD Melanoma Independent Community Advisory Board, m-icab Melanoma Patient Network Europe, MPNE
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EORT patient course Feb 2014- where should patients get involved in the clinical research process? The earlier patients get involved in the clinical research process, the larger the potential beneficial impact for them. Involving patients at a time point when all parameters of a trial are set misses the real opportunity of added value through patient involvement. Doing the right thing instead of only doing things right! You can also watch the talk under http://goo.gl/KS3VyF.
Transcript of Patient Participation in Clinical Research- as early as possible!
EORTC Patient Course 2014
The trials we need-Patient involvement in clinical trial design
Bettina Ryll, MD/PhDMelanoma Independent Community Advisory Board, m-icab
Melanoma Patient Network Europe, MPNE
Declaration of conflict of interests
11/6/2014
BR is not receiving any personal remuneration/ honoraria/ compensation for her involvement in Melanoma patient advocacy.
Honoraria received for consultancy activities (Amgen, GKS, Novartis) are paid into an m-icab/MPNE fund held by RCT in Belgium and go without exception towards m-icab/ MPNE activities.
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Patients
• carry the risk to theirs lives in clinical trials• carry the major burden of ‘the risk of not
taking risks’- before future patient generations-if trial design is merely confirmative instead of learning
• will be prevented from accessing drugs due to high cost to the health care system
The recurrent debate ‘what do patients contribute to clinical trial design’…..
citationBMJ 2014;348:g368published 24th Jan 2014
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….is outdatedas patient emancipation is already a fact.
Melanoma-Independent Community Advisory Board
11/6/2014
www.m-icab.org
Melanoma Patient Network Europe
11/6/2014 www.melanomapatientnetworkEU.org
trial management, e.g. consent, patient
information leaflets, trial adherence
patient recruitmente.g. providing information on clinical trials, advertising trials
clinical trial design
Clinical trials- where to get involved?
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clinical question/ problem
trial management, e.g. consent, patient
information leaflets, trial adherence
patient recruitmente.g. providing information on clinical trials, advertising trials
clinical trial design
Where to get involved?
11/6/2014
clinical question/ problem
current patient involvement
trial management, e.g. consent, patient
information leaflets, trial adherence
patient recruitmente.g. providing information on clinical trials, advertising trials
clinical trial design
Where to get involved?
11/6/2014
clinical question/ problem
current patient involvement
impact on patients
trial management, e.g. consent, patient
information leaflets, trial adherence
patient recruitmente.g. providing information on clinical trials, advertising trials
clinical trial design
Where to get involved?
11/6/2014
clinical question/ problem
current patient involvement
impact on patients
doing the right thing doing things right
trial management, e.g. consent, patient
information leaflets, trial adherence
patient recruitmente.g. providing information on clinical trials, advertising trials
clinical trial design
Where to get involved?
11/6/2014
clinical question/ problem
Asking the right questions-
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CONFIRMING versus LEARNING trials
progress ‘the cure’
risk for the non-patient
unknown risk to the patient
known risk to the patient
The design of a trial-
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• is not a law of nature and therefore evolvable• what looks like good science is not always good
for patients
‘randomized, double-blind, placebo-controlled’
Alternatives- Adaptive licensing
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‘….. ‘adaptive’ approaches to marketing authorization that have been proposed and discussed since the 2004 Report. Such approaches are known under various names (including staggered approval, adaptive approval, progressive authorization and adaptive licensing) and have been proposed by key opinion leaders in the EU and the United States.1,2 These adaptive approaches are all based on the premise that knowledge about medicines is not binary but continues to evolve over time. The proposal is to replace the single transition from non-approval to approval with a series of approval stages with iterative phases of evidence gathering and regulatory evaluation. The use of adaptive approaches, which also incorporate elements of existing pathways, should be seen as a holistic option in the future regulatory system.
Source: WHO- Priority Medicines for Europe and the World 2013 Update 1 Eichler HG et al. Adaptive licensing: taking the next step in the evolution of drug approval. Clin Pharmacol Ther, 2012, 91:426-437. 2 Executive Office of the President, President’s Council of Advisors on Science and Technology. Report to the President on propelling innovation in drug discovery, development and evaluation. Available at http://www.whitehouse.gov/sites/default/files/microsites/ostp/pcast-fda-final.pdf Last accessed 25 February 2012.
http://www.ema.europa.eu/docs/en_GB/document_library/Presentation/2012/04/WC500124930.pdf
http://www.ema.europa.eu/docs/en_GB/document_library/Presentation/2012/04/WC500124930.pdf
http://www.ema.europa.eu/docs/en_GB/document_library/Presentation/2012/04/WC500124930.pdf
Potential advantages for patients
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• timely access to novel promising therapies• continuous testing and re-evaluation will give a
better picture of efficacy and safety ‘real world effectiveness’
• coordination with HTA will reduce the time gap between drug authorization and reimbursement
M-icab Conference 2014 Patient participation in Melanoma Clinical Research
www.melanomapatientnetworkEU.org
Summary
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• focus on maximum impact on patients’ lives• learning over confirming trials• respect for a patient’s perception of risk• assess the humane impact of science• make full use of existing regulatory alternatives• take the EMA by their promise to enable
innovation!
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only a patient knows what it is like to be a patient-
we as advocates need to ensure that it gets noted
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For us as patient advocates, this means
• assuming our responsibility• educating ourselves• accurately capturing patients’ needs• participating as equals in the general
discussion • working on constructive solutions
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We as patients are stakeholders in our own right and need to represent ourselves.
• Expertise defined by personal experience rather than professional standing.
• Heterogeneous• Highly motivated• Desperate• Sense of urgency• Few conflicting interests
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The need for education-
Affecting change is only possible when knowing the constraints under which a system operates, we therefore need to• collaborate effectively on educational needs
and exchange know-how and expertise• make use of existing resources, e.g. EUPATI or
professional bodies like ASCO, TOPRA, IMI…• ‘professionalize’: the patient expert
……but at the same time
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Accurate capture of patients’ needs
• patients are a heterogeneous group and perceived are not always real needs
• respect for ‘the lay patient’s perspective’
need for data
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Engage as equal partners-the value of the patient perspective
Drugs’ ultimate reason of being is to benefit patients. Patients are experts in what it means to live with their condition.
We as society ignore the existing expertise at our own peril.
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Being part of the solution….
as only constructive solutions will make a difference to patients.
• Collaborative approaches based on mutual respect and understanding
• Critical but constructive• Focus on outcomes
because
• we are the experts on our condition.• we can ensure that a drug is trialed in a way
that is relevant to patients. • we can ensure that a drug adds value to a
patient’s life.
‘doing the right thing, not only things right’
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Thank you for your attention and please keep in touch!
Bettina
www.m-icab.orgwww.melanomapatientnetworkEU.org
