Medical Research BADRAG meeting Jan 2013 Dr H Sari-Kouzel

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

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

The Cycle of Research

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

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

Planning Research Planning and funding Funding secured Study file Finalise protocol

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

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,

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

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]

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

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

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

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.

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

Dissemination The Lancet British Medical Journal etc

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

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

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

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

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

National Databases Rheumatology BSR BR-AS INBANK in progress other

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

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

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

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

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

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

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 Research Governance Framework

Patients taking part in Research choice.co.uk/clinical trials research/

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

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

Question time