UKCRC Clinical Trials Booklet PDF

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A guide to understanding Clinical Trials...

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U nderstanding C linical T rials

The UK Clinical Research Collaboration (UKCRC) is a partnership of organisations working to establish the UK as a world leader in clinical research, by harnessing the power of the NHS. The aim is to revitalise the environment for clinical research in the UK, benefiting patients and the public by improving national health and increasing national wealth. The partnership includes the main UK research funding organisations, academia, the NHS, regulators, industry and patients.

One of the key aims of the partnership is to increase public awareness and understanding of clinical research and to promote active patient and public involvement in the research process.

www.ukcrc.org

Although every effort has been made to ensure accuracy, the UKCRC and its advisors cannot accept any liability in relation to the information in this booklet. It is not a substitute for professional medical care. Readers are strongly advised to discuss the information provided and seek personalised advice from their doctor or health care professional

Th e UKCRC wel co mes any feed back that can help to impr ove our publ ic atio ns. If you would l ike to make any c omments on thi s bo ok let pl ease c ontact the UKCRC b y emai l at i nf o@ uk cr c. or g or i n wr iting to :

UK Clinical Research Collaboration 20 Park Crescent London W1B 1AL United Kingdom Tel: +44 (0)207 670 5452 Fax: +44 (0)207 637 6067 [email protected] www.ukcrc.org

U nderstanding C linical T rials

1st editi on

© U KCR C 2 006 Thi s b ookl et is the co pyr igh t o f t he U KC RC. It can be do wnl oad ed, pr int ed out, copied , us ed and di str ibu ted fr ee of c har ge for no n-c omm erc ial pur pos es. No alt era tion or ad apt ati on of thi s b ookl et may be ma de wit hou t t he perm iss ion of UKC RC. Wh ere it is copied , u sed or di str ibu ted, th e s our ce of the ma ter ial mus t b e id ent ifi ed and th e c opyright s tat us ack now led ged . Repro duc tio n o r di str ibu tio n o f m ate ria ls eith er in who le or in par t for a com mer cial pu rpo se is pro hib ite d w itho ut the pr ior wr itt en per miss ion of UKC RC, 20 Park Cr esc ent , L ond on, W 1B 1AL .

Contents

Introduction

4

Wha t a re clinical tria ls?

5

Why are c lin ic al trials imp ortan t?

6

How are trials set up ?

7

Who c an take par t in clinic al trials?

8

How are p eop le rec ruited to a trial?

9

What are the risk s an d b enefits of trials?

10

How are trials su p p or ted?

12

How a re tria ls de signe d a nd run? Are there d ifferen t types of trial?

13 13

Why d o some trials need man y p eop le?

17

Why d o trials sometimes take man y years?

18

Are you thinking a bout j oining a tria l?

19

What is ‘in formed c on sen t’?

19

What hap p en s d u rin g a trial?

21

What hap p en s at the en d of a trial?

22

Will my in formation b e con fid en tial?

22

What hap p en s if somethin g goes wrong?

23

How c an I find ou t ab out trials that are happ enin g n ow?

23

What shou ld I ask b efore I join a trial?

24

Whe re ca n I find m ore inform ation?

30

Lin ks

30

Organ isation s

30

Fur ther read ing

32

3

Introduction If you are being treated in the NHS you may be asked to take part in a clinical trial. Clinical trials are research studies that involve patients or healthy people and are designed to test new treatments.

In this booklet we use the term ‘treatments’ to mean a wide range of health care approaches that can be tested in a clinical trial including drugs, vaccines, other approaches to disease prevention, surger y, radiotherapy, physical and psychological therapies, educational programmes and methods of diagnosing disease.

This booklet has been written to try to answer the many questions people ask about clinical trials. It explains what clinical trials are and why and how they are carried out. It is designed to give you the information to help you to decide whether to take part in a trial. It also includes some of the questions you may want to ask before you make a decision to join a trial.

4

What are clinical trials? Clinic al trials are medic al research stu dies in volvin g p eop le. They aim to test whether differen t treatmen ts are safe and how well they work . S ome trials in volve healthy members of the pu b lic . Others in volve p atients who may be offered the option of tak in g p art in a trial d uring their c are an d treatment. C lin ic al trials are c arried ou t to tr y to an swer spec ific q u estion s abou t health an d illn ess.

The y a im to find the best w a ys to:



preven t d isease an d redu c e the nu mber of p eop le who bec ome ill



treat illness to improve su r vival or inc rease the n umb er of people c u red



imp rove the qu ality of life for p eop le living with illn ess, in c lu d in g redu c in g symptoms of disease or the sid e effec ts of other treatmen ts, su ch as c anc er c hemotherapy



diagn ose diseases and health p rob lems.

Clinic al trials c over a b road ran ge of d ifferen t typ es of researc h. For examp le, trials are of ten u sed to test n ew med icin es or vac cin es bu t c an also be used to look at n ew c omb ination s of existing med ic in es. They can also be used to test whether givin g a treatmen t in a d ifferent way will make it more effective or red uc e an y sid e effec ts. Some trials are d esign ed to tr y ou t ways to p reven t a p ar ticu lar d isease in p eop le who have n ever had the d isease, or to p revent a d isease from retu rn ing. The

5

treatmen ts b ein g tested in these typ es of stu d ies can in clu de vac cin es, b u t may also in volve dru gs or dietar y su p plemen ts su c h as vitamin s an d min erals.

Clinic al trials are n ot always ab out testin g medic in es, they c an be used to test ‘in ter ven tion s’ aimed at mod if yin g a p erson’s behaviou r or lifestyle. This c ould in clu de an edu c ation al programme d esigned to imp rove a p erson’s un d erstan d in g of their med ic al c ond ition and help them to man age it more effec tively, or a p syc hological treatmen t, suc h as the u se of cognitive b ehaviou ral therap y for the treatmen t of an xiety or dep ression.

Why a re cl inical tria ls imp or tant ?

Clinic al trials are the b est way to c omp are d ifferen t ap proac hes to preven tin g and treatin g illn ess an d health p rob lems. Health professionals and p atien ts n eed the evid en c e from trials to k n ow which treatmen ts work best. Withou t trials, there is a risk that people c ould b e given treatmen ts whic h have n o ad van tage, waste resou rces an d might even be harmfu l. Man y treatmen ts that are now in c ommon u se in health care were tested in clinic al trials.

Some typ es of c lin ical trial are d esign ed to look at a treatment at an early stage of its development. Researchers and regulators will look at the in formation they have gathered an d d ec ide whether it is safe an d ap propriate to c ontinu e the d evelop men t of that treatment. If the treatmen t has no ben efit or has seriou s

6

sid e effects, it may n ot be developed fu rther.

Du rin g the later stages of d evelop men t of a treatmen t researchers will report on the b enefits an d risk s so that doc tors can d ecid e whether or how b est to use it. I t is important that the resu lts of clin ic al trials are p u blished so that others c an u se the in formation to help them make d ec isions abou t treatment an d health c are. C lin ical trial resu lts also form an imp or tan t part of the eviden ce used to d ec ide whether a particu lar treatmen t will be p rovid ed through the NHS .

How a re tr ials s et up ?

Clinic al trials are d esigned b y d octors an d other sp ecialists with inp u t from a wid e variety of people, inc reasingly inc lud ing patien ts. They work together to d ecid e what q u estion s n eed to be an swered . First of all they look c arefully at the resu lts of the trials that have already been d on e to find ou t what is alread y kn own. This is c alled a syste ma tic re vie w . A systematic review provid es more acc u rate an swers than ind ivid ual trials an d also help s to iden tif y imp or tan t q uestion s that still n eed to be an swered throu gh fu rther researc h.

Doc tors, nu rses, p atients an d researc hers work together with statistic ian s, trial managers an d rep resen tatives from pharmaceu tic al comp an ies if relevant, to d esign the b est possib le trial. The d esign for the trial forms the b asis of the tria l

protocol .

7

When the trial protoc ol is read y it is sen t to a rese arch e thics

com mitte e , an ind ep en d en t grou p of p eop le that in c lu d es doc tors, nu rses, other medic al staff, members of the pu b lic an d sometimes lawyers. They d ec ide whether the trial is ethical. I n particu lar they c hec k whether:



the p oten tial b en efits of a new treatmen t are likely to ou tweigh the side effects



the in formation p rovid ed to help p eop le to dec ide whether they wan t to p articip ate in a trial is c lear and satisfac tor y



the way in whic h p eop le will b e asked to take part in a trial (recru ited) is ap p rop riate



there will b e c omp ensation for people in the trial in the un likely even t that somethin g goes wrong



travel expen ses will b e offered to p eop le who take part.

The trial c an only go ahead when it has been ap proved b y an ethic s c ommittee.

Who c an ta ke par t in c linica l tri als?

All trials have gu idelin es abou t who can take p art. These are called e ligibility crite ria . Eligibility c riteria are u sed to en sure that trials in c lu d e the sor t of p eop le who may b en efit from the treatmen t, and to make su re that p eop le who take part are not exposed to avoidab le risks. This means that there may n ot

8

always b e an ap prop riate or su itab le clinic al trial for you to take part in.

The inclusion criteria help the researc hers to d ec id e who

can

take p art in the trial. S ome trials only in c lu d e p eop le in a certain age group , or of on e sex, or at a p ar tic ular stage of their illness.

The exclusion crite ria state who

cannot

take p ar t in the trial.

For examp le, many dru g trials d o n ot allow pregn ant women to take p art as there may b e a risk to the u nb orn bab y. Peop le who are already tak in g particu lar med ic ines may also be exc lu d ed as these may affec t the trial treatment.

Before you go into a trial you may have to have some extra tests to see if you are eligib le or to ensu re that you are not likely to be at risk of b ein g harmed b y the treatmen ts in the trial. For examp le, if a poten tial sid e effect of a n ew dru g is that it in c reases b lood p ressu re you may have your blood p ressu re checked to see if you are eligib le to join the trial.

How a re pe ople r ecrui ted to a tr ial?

A c lin ic al trial is often run in a n umb er of d ifferent hospitals or health c entres. The d octor or n urse who ask s you to take part in a c lin ic al trial may n ot be the p erson who design ed and set u p the stud y, espec ially if it is ver y large. However, they will have been fully b riefed ab out the stud y b efore agreein g to b ecome

9

in volved . They can give you all the information you n eed an d will b e able to an swer your qu estion s.

What are t he ris ks an d bene fits of tri als?

Clinic al trials are c arefully d esign ed to min imise the risk s and maximise the b enefits to all who take par t, whatever treatmen t they rec eive. S ome trials will have ver y little risk involved. However, the risks of a trial may b e greater when less is k n own ab out the treatmen t b ein g tested. Before an y d rugs are first given to people, they will have b een developed in a lab orator y an d c hec ked for safety in an imals.

In all trials the treatment may c ause side effects that doc tors can not p red ict an d that you may n ot b e exp ectin g. These may be un p leasan t and ver y rarely c an b e life-threaten ing. You should b e told ever ythin g that the researchers kn ow ab ou t an y possib le risks an d sid e effects an d why the trial is nec essar y so that you can make an in formed c hoic e abou t whether to take part.

If you take p ar t in a trial you will be mon itored regularly d u rin g an d after the stu d y. You will have regu lar tests an d you may be asked some extra qu estion s ab ou t how you are feelin g. You may also be asked to fill ou t q u estion naires or to keep a diar y. Sometimes this mean s goin g to you r hosp ital or GP more often than you wou ld normally, so b ear this in min d b efore you agree to take p art. Ask how man y extra visits will b e n eed ed and

10

con sid er how c onven ien t this will be for you. Usu ally there will be mon ey available to help with an y extra c osts you have.

The b enefit to you of this extra attention is that an y c han ges in you r health, whether or n ot they are related to the treatment you are having, are freq u en tly pic ked up an d ac ted up on earlier than if you were n ot in a trial. However, some peop le fin d that the extra atten tion makes them worr y more abou t their c on d ition an d p revents them from ‘gettin g on with their life’.

It is imp or tan t to rememb er that not ever yone rec eives a n ew treatmen t in a clin ic al trial. A c lin ic al trial n eed s to c omp are a n ew treatmen t with the stand ard treatmen t alread y in u se, if there is on e. S ome people in a trial will therefore rec eive the stan d ard treatmen t b u t, un til the resu lts of the trial are an alysed , n o on e will kn ow whic h treatment is b etter. does n ot always mean

‘New’

‘better’ an d you may not b e worse off if

you d o n ot rec eive a n ew treatmen t.

Peop le who take p art in trials often feel that they are tak ing an ac tive p art in their health c are. They are also help in g others, and possib ly themselves, b y help in g to id entify the b est treatmen ts.

11

How a re tr ials s uppor t ed?

Man y d ifferent typ es of organisation su p por t clinic al trials. These in c lu d e:



the NHS



the Me dical Rese arch Council an d government dep ar tments or agenc ies



cha ritie s



pha rm a ce utical com panies .

All trials, no matter who fu nd s them, are c hec ked and mon itored in similar ways to make sure that the people who take p art are p rotected. Eac h trial also has a sponsor who is respon sib le for the c ond u ct of the trial. The spon sor may b e the organ isation fu nd ing the trial or the institu tion hosting the research, for examp le, a un iversity.

Man y of these organ isations in volve p atien ts to help dec id e what will b e researched in the fu ture. I t is essen tial that research takes into ac c ou n t the needs an d in terests of the people it is tr yin g to help. Sp ecialists are of ten aware of gaps in kn owledge ab ou t d iagn osis an d treatment bu t p atien ts an d their families may also see asp ec ts of c are that need fu rther researc h.

12

How are trials designed and run? Are t here differ ent t ypes o f tri al? Clinic al trials are c arried ou t in a nu mber of stages. Early stage trials u sually involve a small nu mber of patien ts or healthy p eop le. When p syc hological treatmen ts or edu c ation al programmes are bein g tested , these early stage stu dies c an be used to ‘ fin e tu ne’ the treatmen t b efore it is tested in a large group of people. For trials of medic in es and other treatments, early stage stu dies are c arried ou t in a small group of people to assess safety b y look ing for u n wan ted side effects. Later stage clinic al trials u su ally involve larger n umb ers of particip an ts an d are u sually ra ndom ise d tria ls . The p roc ess of ra ndom isa tion is exp lained later in this sec tion.

A good examp le of how the c lin ical trial p roc ess help s to answer imp or tan t q uestions is the development of new d rugs. These are first d evelop ed in the lab orator y to see whether they may be helpfu l in the p reven tion or treatmen t of a particu lar illness. They are then tested in animals to c hec k their safety an d to fin d out how they affect the b ody. I f they look like they may b e of ben efit and are likely to be ac c ep tab ly safe they will then be tested throu gh different stages of c lin ical trials. For dru gs, the differen t stages of c lin ical trials are kn own as pha ses :

Ea rly sta ge : Pha se 1

Phase 1 is the first stage and usu ally involves small grou p s of healthy p eop le or sometimes patien ts. Phase 1 trials are main ly aimed at fin din g ou t how safe a d rug is.

13

Pha se 2

By the time a d ru g reaches Phase 2, researc hers will kn ow q uite a lot ab out it.

Pha se 2 tria ls aim to:



test the new d rug in a larger grou p of p eop le to b etter measu re the safety an d side effec ts



see if the d ru g has a positive effec t in p atien ts.

La ter sta ge : Pha se 3

Phase 3 trials are large an d may inc lud e hu nd reds, or sometimes many thou san ds, of p atients from all over the UK, an d often from several c ou n tries.

Pha se 3 tria ls aim to:



compare the effec ts of n ewer d rugs with the stand ard treatmen t, if there is on e



fin d out how well the dru g work s and how long the effec ts last



fin d out more abou t how c ommon an d serious an y sid e effec ts or risk s are and ab out an y p ossible lon ger term problems that c ou ld d evelop .

14

Pha se 4

Phase 4 trials are carried out af ter a new d rug has b een shown to work and has b een given a licen ce.

Pha se 4 tria ls aim to find out:



how well the d rug work s when it is u sed more widely



the long- term risk s an d b en efits



more abou t the possib le rare side effec ts.

Phase 1 trials on ly p ick up ver y c ommon sid e effec ts. Phase 2 trials help to pic k u p less common side effec ts, b ut Phase 3 or 4 trials are n eed ed to p rop erly assess safety and risks.

If you are asked to take p art in a trial, there are a nu mber of terms which you may hear:



Controlle d tria ls

Controlle d tria ls are design ed to compare d ifferen t treatmen ts. Most c ontrolled trials c omp are a n ew treatment with the stand ard or u su al treatment by settin g u p two group s of p eop le. One group , k n own as the tria l group or
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