Dr Mary Baker MBE President, European Brain Council

1. Scottish Medicines Consortium 10 Year Anniversary Conference The Patient Perspective 21 March 2013 Royal College of Physicians of Edinburgh Dr Mary Baker MBE President, European Brain Council Patron, European Parkinson’s Disease Association

2. Costs of disorders of the brain In Europe 2010: • €798 billion across 30 countries and 19 groups of disorders • Has increased from €386 billion in 2004 European Neuropsychopharmacology (2011) 21, 718-779

3. Distribution of costsTotal disorders of the brain European Neuropsychopharmacology (2011) 21, 718-779

4. Distribution of costsNeurological & Mental disorders Neurologicaldisorders Mental disorders European Neuropsychopharmacology (2011) 21, 718-779

5. The world is living longer A baby girl born in Japan has a 50/50 chance of living 100 years

6. Global ageing in the 21st century ‘The world stands on the threshold of a social transformation - even a revolution - with few parallels in humanity's past.... perhaps two-thirds of all people who have ever reached the age of 65 are alive today.’ Peter G. Peterson

7. The changing role of women In the 1920’s, a couple in their 80’s had 44 female relatives - 14 of these did not work outside the home environment In the year 2000, a couple in their mid 70’s had 13 female relatives - 3 of these did not work outside the home environment What is the situation in 2013?

8. Fewer to care for more:a declining dependency ratio By courtesy of Dr Panos Kanavos, London School of Economics

9. Polypharmacy and co-morbidity

10. Provision of culturally relevant care The challenge of migration

11. Time with the doctor .....12 minutes

12. Access to medication A very uneven playing field

13. Counterfeit Medicines 2011: in EU CMs risen to over 27million WHO estimate CM sales in developed countries is 1% & 10% in developing countries • 2008 ‘Project Medifake’ European Customs initiative discovers… • 3 million packs captured over two months • 4 MHRA batch recalls in 2007 • EAASM found 62% of medicines bought from internet to be fake or substandard (Counterfeit Superhighway - 2008) EU Falsified Medicines Directive due to be adopted by Member States within 3 years of January 2013

14. In production GMP Colombian style Ingredients include: Boric Acid Lead-based road paint Shoe polish Brick dust Profit before patients!

15. Way forward Trust Innovation

16. A quick fix

17. Safety versus innovation Education of benefit/risk Quote from cancer patient: ‘I am already in an unsafe world, a little more unsafeness doesn’t worry me’ Dr Albert Jovell, Barcelona

18. Benefit/Risk: the need to know The difference between side effects and adverse effects Side effects: can often be tolerated where there is overall benefit to health Adverse effects: is it worth taking medication if the ‘cure’ is worse than the disease?

19. Transparency HMA/EMA Guidance Document on the identification of commercially confidential information and protection of personal data within the structure of the marketing authorisation (MA) dossier – release of information after granting of a marketing authorisation The challenge ... effective communication

20. New approach to clinical trials

21. Clinical trials – the facts • 30% of all clinical trials fail to recruit a single person • 85% of clinical trials face delays due to limited participation • Fewer than 10% of Parkinson's patients ever take part in trials, despite overwhelming interest in working with scientists to help speed treatment breakthroughs

22. Family Planning & Pregnancy in chronic disease A frequent issue not heard, not talked about and not seen… By courtesy of Dr Lode Dewulf, UCB Pharma GmbH

23. A health tragedy struck50 years ago • Thalidomide is a sedative drug introduced in the late 1950s that was used to treat morning sickness and aid sleep. • It was sold from 1957 until 1961/2, when it was withdrawn for this indication after being found to be a cause of major birth defects. (Thalidomide is currently used for the treatment of multiple myeloma). • The thalidomide tragedy led to much stricter testing being introduced for drug and pesticide licensing • It also caused a halting of research in children and in pregnant ladies By courtesy of Dr Lode Dewulf, UCB Pharma GmbH

24. Co-existence of disease and pregnancy (plans) • After 50 years, the pregnancy issue remains: • This issue is frequent and on the rise • e.g. Estimated 1 in 1000 pregnant women is diagnosed with cancer during their pregnancy. • e.g. Lupus (90% of which occurs in women) starts before the age of 30 in 1 in every third diagnosedwomen. • e.g. RA (prevalence: 1 in every 250 women) starts before the age of 30 in 1 in every fifth diagnosed women. • eg. 25% of women with IBD conceive for the first time after diagnosis • Societal trend to delay family planning increases the incidence of co-morbidity during pregnancy • Earlier diagnosis and better treatment for severe and/or chronic disease allow women to consider having children • The issue is broader than the pregnancy itself, and also includes the decision to conceive, the ability to conceive and the post partum (lactation, vaccination) • The issue is not only medical, but also emotional and societal By courtesy of Dr Lode Dewulf, UCB Pharma GmbH

25. Patient reported outcomes Develop a measurement acceptable to European Medicines Agency (EMA) and Food & Drug Administration (FDA) in partnership with: • Academia (University of Oxford - Ox-PAQ project) • Clinicians • Regulatory, payers, patients and their families

26. Patient reported outcomes

27. The Regulator and the Payer Professor Guido Rasi Chief Executive Officer European Medicines Agency (EMA) Sir Michael Rawlins Chairman National Institute of Health & Clinical Excellence (NICE)

28. Lifestyle choices 60% of NHS budget is spent on lifestyle issues: • Ageing • Infertility • Sexual health • Smoking • Drug Addiction • Alcohol • Obesity

29. Distress is not enough Patient advocacy groups need to: • Establish credibility • Collect evidence • Contribute to discussions • Understand health technology assessments

30. HTA Summer School for Patient Groups Collaboration between EFNA and London School of Economics: • September 2009 • May 2010 • June 2010 • September 2010 • June 2011 • September 2011 • February 2012 • September 2012 Working for people living with brain disorders

31. HTA Summer School for Clinicians Proposal under development for 2013

32. The Patient’s journey Information is needed from: • Clinician – about the disease • Industry – about the medication • Patient NGOs – about the fellow travellers

33. A major challenge – societal engagement • How do we involve patients/carers in research? • An informed patient is a cost effective patient (Wanless) • (

34. Working together Patients have a voice and need: Join the team • to be heard • to be listened to • to be inclusive

35. The future of medicine Today Future In the future we diagnose and treat based on biology and select medication based on an objective evaluation of the benefit/risk for the individual patient Today we diagnose and treat based on symptoms and a subjective interpretation of symptoms

36. Telemedicine and eHealth • Telemedicine and eHealth will play a major role in the future • The question is not if it will happen, but when

37. From the bench to the bedside New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years Rx

38. From the bench to the bedside New drug application & review ~ 2 years New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years HTA review ~ 2 years Rx

39. From the bench to the bedside New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years Rx

40. From the bench to the bedside New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years Rx

41. From the bench to the bedside New drug application & review ~ 2 years Preclinical & clinical development ~ 8 years HTA review ~ 2 years Rx

42. Management of long termchronic illness • The ability to adaptand self manage • To be able to participate in social activity despite all limitations

43. What matters most ... ‘It is not the strongest of the species that survives nor the most intelligent that survives. It is the one that is most adaptable to change.’ Charles Darwin1809 - 1882