1. Medical Research BADRAG meeting Jan 2013 Dr H Sari-Kouzel

2. Overview Why do we need research How it it done Different types of research New drug development Taking part in research Questions

3. Why do Research? Understand disease process Develop medicines Prevent disease development – vaccines Reduce burden of disease

4. The Cycle of Research

5. Design Research Study Research question Consult R&D Discuss sponsorship Develop Protocol

6. Planning Research Develop protocol consultation patient / participation groups Risk management Consent / confidentiality

7. Planning Research Planning and funding Funding secured Study file Finalise protocol

8. Seeking Ethical Approval Research Ethics committees – Group of members of public and experts. – Review research proposals – Independent – Ethical standards; respects the dignity, rights, safety, and well being of the people who take part

9. Types of Research Clinical trials – medicines – devices Non clinical trials – Lab based; animal models, human tissue, genetic studies, etc – Epidemiology; smoking tobacco is cause of lung cancer. – Clinical research; quality of life,

10. Lab based research Animal models of disease similar to human. Human tissue; surgery, post mortem, live samples, synovial fluid (joint aspiration) Genetic research; blood or saliva samples

11. Human Tissue Act 2004 An Act to make provision with respect to activities involving human tissue; to make provision about transfer of human remains from certain museum collections; and for connected purposes. [15 th November 2004]

12. Human Tissue Act 2004 illegal Removing, storing or using human tissue with out consent. DNA “theft”- taking and testing DNA without consent. Storing tissue or organ for a purpose not stated. Organ trafficking

13. New Drug Development Pre clinical ; Lab, animal, toxicity Phase I Healthy Volunteers Phase II first patients, dose ranging Phase III more patients marketing application Phase IV post marketing studies

14. Phase III clinical trials Involves patients Randomised controlled clinical trial; Gold standard – blind, double blind. Proposal with full details of the trial protocol. Ethical approval

15. Results Results published in a peer-reviewed journal. Standard for reporting results. Analysis is scrutinised by other researchers. License to market the medicine MHRA (UK), in other countries FDA, European.

16. Dissemination of Results Presentations at conferences - abstract Publication in Medical journals Newspapers Headlines

17. Dissemination The Lancet British Medical Journal etc

18. National institute of clinical excellence NICE Independent organisation Provides national guidance and standards Appraisals for new drugs/ devices Cost effectiveness new drugs Makes recommendations TAG ensure equal access for patients across England Input from experts as well as public

19. Phase IV Post Marketing MRHA reporting system for new drugs, black triangle▼ Any side effects should be reported. professionals and public are encouraged to report Yellow card system

20. Post Marketing Databases set up to monitor the drug in the real world. 2000 first biologics - anti TNF were licensed for use Concerns about the long term effect Biologics register – BSRBR

21. BSRBR Based in Manchester University Funded by a number of pharmaceutical companies Independent NICE approval recommended entry to register Patient consent was required Baseline questionnaire, 6 monthly

22. BSRBR Support from patients and rheumatologist phenomenal Important information UK wide collected and reports published regularly. Similar registries in other countries has provided real experience of biologics

23. National Databases Rheumatology BSR BR-AS INBANK in progress other

24. Non Clinical trials Ethical approval required Patient consent Researcher – theory – Design’s a study to answer a question – Pilot study – test the water – Expand

25. Research is fundamental to the NHS “Research is vital in providing the new knowledge needed to improve health outcomes” the White Paper 2010

26. National Institute for Health Research National body Established April 2006 Framework National Research Facility Improving the health and wealth of the nation through research.

27. Role of NHS Every Trust has a dedicated Research and Development R&D team Most trusts have a clinical trial unit/ research facility to run clinical trials Dedicated research nurses and admin team Run commercial and non commercial studies

28. Taking Part in Research Doctors and allied health professionals in training learn about research Do projects Learn to appraise published papers Good clinical practice- training updates for doctors

29. Patients taking Part in Research Patients invited Patients provided information about the project Opportunity to discuss Patient consent Enrolment

30. Standards in Research Ethical standards to protect people 1947Nuremburg Code 1964 Declaration of Helsinki 1996 international conference of harmonisation GCP 1996 Medical Research Council GCP 2004 European Directives update 2013 2005 Research Governance Framework

31. Patients taking part in Research www.NHS choice.co.uk/clinical trials www.healthtalkonline.org/medical research/ www.healthtalkonline.org/medical

32. From Serendipity to Science Gold injections – 1928 injecting Gold salts – 1961first controlled trial Methotrexate – 1951use described – 1978 formal clinical trial Biologics – 1995 Clinical trials – 2000 Licenced used clinical practice

33. Future of Research “The more you know, the more you find out that you do not know”

34. Question time