Orphan Drugs & Personalised Medicine: The Patient Perspective

Transcription

1 Orphan Drugs & Personalised Medicine: The Patient Perspective Dr. Cees Smit, Inspire2Live/VSOP/EGAN DCTF, Ede, October 8, 2014

2 a Patient Advocacy Platform, bringing together patients, doctors, scientists and others to work on the challenge to get cancer under control.

3 Content Fundamental Research (Animal Research) ALS Ice Bucket Challenge Unmet medical needs Innovative Power of Patients Personalised Medicine EU New Clinical Trial Regulation Conclusion

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6 The other side of the coin 2004: Foundation Informatie Dierproeven (SID), founded by Patients: VSOP Scientists: Federa (FMWV) Animal lab scientists (NPV)

7 You Tube movie Dierproeven doe je niet zomaar 3 minuten

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10 De patiënt in 40 jaar Patiënt 1.0 Patiënt 2.0 Patiënt 3.0 Informatie, voorlichting Lotgenotencontact Belangenbehartiging Internet, social media, Wiki s, Patientslikeme, Mijn Zorgnet Samenwerking onderzoekers, patiënten en bedrijfsleven Drijvende kracht: Unmet medical needs

11 Dutch ALS campaign I m now deceased

12 treeway.nl & projectmine.com

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14 The ALS Catch-22 Challenge Every year in Europe new cases Every year in Europe deaths of ALS This is why we need as patients a fast and effective clinical trial regulation in Europe for unmet medical needs

15 Adaptive licensing New medicines for serious conditions: how patients would weigh benefits and risks Report form Genetic Alliance UK with Welsh Institute for Health and Social Care April 2014,

16 Unmet medical needs Unmet medical needs are the driving force for patient groups to become active in medical research

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19 Proportion of patients Hemophilia, age-distribution (n=338) 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 0% Year >40 years years years years 0-10 years Mauser et al, Van Creveldclinic, Utrecht, 2010, personal communication

20 The patient in 40 years Patiënt 1.0 Patiënt 2.0 Patiënt 3.0 Information, education, peer support lobbying for their interests Internet, social media, Wiki s, Patientslikeme, Mijn Zorgnet Cooperation patients, physicians, researchers and industry Driving force: Unmet medical needs

21 Trek or Pull model

22 Innovative power patients To bring together patients and their body material (DNA, tissue, etc.) Sharing of data for research and the organization of cooperation (EuroBiobank) Fundraising for biobanks and research (gene therapy), also for the long term (Téléthon) (Ref.: Van der Valk & Smit, NTvG, mei 2011)

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25 Genethon BioProd Genopole

26 Genopole, Paris The Genopole project was conceived in the late 1980s by Bernard Barataud (the then President of the French Muscular Dystrophy Association, organizer of France s first Telethon fund-raising event and the founder of Genethon, a hi-tech genomics and genetics research lab) and Daniel Cohen (Scientific Director of the Foundation) Now, 15 years on, the biopark is home to 71 innovative companies and 21 academic research laboratories.

27 Artritis research Patient reported outcome measures So far from scientists: pain Addition from patients: Fatigue Sleep disturbances Maarten de Wit (EULAR), thesis 2014

28 Six minute walking test Additions from the Duchenne community Small movies on relevant patient outcome measures for EMA (very well received)

29 Movie Duchenne

30 Personalised Medicine (1) For patient registries/biobanks as a research infrastructure tool we need appropriate long-term finance structures Good examples: HIV, HOVON, Parelsnoer For example: by a percentage of a DOT/DBC!

31 Personalised Medicine (2) The new EU Data Protection Regulation tends to ignore the negative effect the new Regulation could have on scientific and medical research A viable alternative could be to have a separate legislation specific to data protection in the areas of health and medical research. A precedent: Directive 2006/123/EC (the Bolkestein directive on services)

32 Personalised Medicine (3) Registration & Reimbursement needs of n = 1 products instead of n = needs rethinking (a paradigm shift) The same for therapeutics & diagnostics The rare disease example: the need for EUwide databases and procedures (legislation)

33 NOS Journaal, Juli 2012

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35 Meaningful Patient Involvement Value +, European Patient Forum (EPF) Patient Partner, EFGCP, EGAN, ECRIN, EPF Summer School Eurordis, LSE EUPATI, the EU Patient Academy

36 EU Regulations EU Clinical Trial Directive (2000) EU Clinical Trial Regulation ( )

37 EU CTrR Art. 2, point 11 Ethics committee means an independent body established in a Member State in accordance with the law of that Member State and empowered to give opinions for the purposes of this Regulation, taking into account the views of laypersons, in particular patients or patients organisations

38 EPF opinion We believe the review of the legislative framework presents an opportunity for reform towards more patients involvement throughout the research process; greater trust and public confidence in medical research; and improved participation rates.

39 Input from patient groups (1) Nationally, groups of patients could be trained and assist CA/EC in giving their views (disease-specific or disease-exceeding) Before application however, timely consultation of relevant patient groups is essential to discuss pros and cons of proposal There are not so many cultural differences for patients within the EU MS (access) Number of EC s in EU is too big to be effective

40 Input from patient groups (2) In some EU MS there is already experience with pat reprs in EC s, like in the UK National Policy of NHS: Involve In some EU MS patient participation in EC s is forbidden by law (The Netherlands, Germany) But in an optimal ethical review in the next decade, there is no space for paternalism!

41 We are positive, but Patients wants faster access to medicines & therefore a well balanced administrative burden New EU regulation animal research New EU clinical trial regulation New EMA procedures on transparency New EU Data Protection Directive Growth in HTA calculations Difficult ICT mechanism for CTrR EU strong in procedures/processes, but weak in vision! (ref.: Jonathan Holslag, The Power of Paradise, 2014)

42 be more competitive! My estimation is that all the requirements to fullfill all clinical trial requirements and reimbursement procedures in all EU Member States make up percent of the developments costs of new medicines. That s too much to make the EU competitive & that was the reason for the revision of CTrR

43 UK discussion: merge Part 1 & 2

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45 In conclusion European patient groups are in principle positive about the new regulation, but we hope we will be taken serious and really involved in the implementation at the level of the CA/EC of national Member States In the past decade EU Patient Groups have proven to be serious partners in the development of therapies and medicines

46 For more information Dr. Cees Smit