Forskningsseminar hos KORA: "Hvordan involverer vi patienter i medicinsk forskning – og hvad betyder det for forskningen?" Den 02.03.2017

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Patient involvement:
When, Why and Who?
Prof Steffen Thirstrup, MD, PhD

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• MD 1993
• PhD 1999
• Hospital employments (Internal medicine)
• Specialist in Clinical Pharmacology and Therapeutics 2002
• Chief Medical Officer, Danish Medicines Agency 2004
– Danish CHMP-member: 2004-09
– Danish CAT-member: 2008/09
• Head of Institute for Rational Pharmacotherapy: 2009-11
• Head of Licensing Division, Danish Medicines Agency: 2011-13
• NDA: 1. March 2013 

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Medicinal product - definition
• Any substance or combination of substances presented as having properties for
treating or preventing disease in human beings; or any substance
or combination of substances which may be used in or administered to human beings
either with a view to restoring, correcting or modifying
physiological functions by exerting a pharmacological, immunological
or metabolic action, or to making a medical diagnosis.
Includes blood, blood derivaties, substances of animal or vegetable origin, immunologicals, allergens,
‘advanced therapies’ (stemcells, genes, engineered tissue etc), hoemeopathics and more
3

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European Medicines Agency’s interaction with patients, consumers, healthcare professionals and their organisations
Annual report 2015. Published 16 June 2016 on www.ema.europa.eu (EMA/727872/2015 )

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European Medicines Agency’s interaction with patients, consumers, healthcare professionals and their organisations
Annual report 2015. Published 16 June 2016 on www.ema.europa.eu (EMA/727872/2015 )

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Tysabri® (natalizumab) – PML cases
• Tysabri licensed June 2006
• Two post-marketing cases of PML triggers CHMP review in
September 2009
• MS patient’s organization consulted
 Warnings and precautionary measures introduced in the SmPC

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European Medicines Agency’s interaction with patients, consumers, healthcare professionals and their organisations
Annual report 2015. Published 16 June 2016 on www.ema.europa.eu (EMA/727872/2015 )

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European Medicines Agency’s interaction with patients, consumers, healthcare professionals and their organisations
Annual report 2015. Published 16 June 2016 on www.ema.europa.eu (EMA/727872/2015 )

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European Medicines Agency’s interaction with patients, consumers, healthcare professionals and their organisations
Annual report 2015. Published 16 June 2016 on www.ema.europa.eu (EMA/727872/2015 )
Healthcare professionals

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European Medicines Agency’s interaction with patients, consumers, healthcare professionals and their organisations
Annual report 2015. Published 16 June 2016 on www.ema.europa.eu (EMA/727872/2015 )

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www.ema.europa.eu

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Patient involvement – recent examples (1)
• Bisphosphonates and risk of necrosis of the jaw
– PRAC consulted patients on the educational material incl. patient reminder card
• Humalog® (insulin lispro) 200 U/ml
– Concerns regarding introduction of a new high insulin strength and how to ensure its safe
and correct use
 PRAC consultation with patients to obtain input on how best to minimize potential risk of medication errors
• Input received prompted the committee to request further changes to the labelling (differentiations of strengths)
• The company subsequently amended the labelling and other measures in the risk minimization plan
• Implantable mini-pump for continuous s.c. insulin in children with T2DM
– PDCO consulted patients on their view if this device could:
• Help ensure that young type II diabetic patients take their medicine as prescribed
• The need for such alternatives in this age group
• The likelihood that these patients would be willing to accept to have this implant.

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Patient involvement – recent examples (2)
• Review of new information on risk of long-term developmental problems in
children whose mothers took Valproate
 Patient meeting included epilepsy, bipolar disorder and migraine patient organisations and organisations
representing the patients, families and carers affected by valproate
– Very constructive exchange of information; patients shared their personal experiences and provided input on
how best to raise awareness for all concerned; In turn allowed PRAC to explain the assessment process
– Followed by written consultation on educational and communication material to raise awareness &
understanding of risks
– Input used by the PRAC in its final recommendations
• Significant benefit for a medicine for Haemophilia A
– COMP asked patients to help addressed the questions on significant benefit by providing critical information
on major contribution to patient care, sharing both their personal experiences as well as the patients they work
with

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Conclusion
• New medicines are developed for patients
– Logical to involve patients in both development and approval processes
• Patients can act both as individual experts and experts on behalf of a
patient’s organization
– Conflict of interest should be handled as for any other expert or KOL
• The European regulatory system has actively and increasingly
involved patients over the past 10 years
– Increased transparency
– Bilateral improved understanding