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

Medical Research

BADRAG meetingJan 2013Dr 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.

[15th 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.

2000 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 2013 2005 Research Governance Framework

Patients taking part in Research

www.NHS choice.co.uk/clinical trials www.healthtalkonline.org/medical 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