Health related quality of life measurement in clinical trials
Submitting Institution
University of Central LancashireUnit of Assessment
Psychology, Psychiatry and NeuroscienceSummary Impact Type
HealthResearch Subject Area(s)
Medical and Health Sciences: Cardiorespiratory Medicine and Haematology, Public Health and Health Services
Summary of the impact
Ensuring that a new medicine improves or maintains a person's quality of
life is important. Abbott's longstanding collaborative relationship with
health professionals and service users has enabled the development of two
patient-reported, quality of life instruments (CFQoL, LupusQoL). Abbott's
expertise has contributed to the development of policy/guidelines
concerning quality of life measurement in clinical trials for the European
Medicines Agency and European Cystic Fibrosis Society. The instruments
have been adopted internationally with the LupusQoL providing a global
business opportunity for a US translation company. At the request of
pharmaceutical companies, the LupusQoL is translated into the numerous
languages required for use in their multi-national clinical trials of new
medicines.
Underpinning research
When deciding if a medicine is of benefit it is important to obtain the
patient's perspective, allowing service users a voice in their healthcare.
Health-related quality of life (HRQoL) measurement is able to do this and
refers to the physical, psychological and social wellbeing as perceived by
the individual. HRQoL Questionnaires (instruments) can provide a
standardised, valid and reliable way of gaining the patients' perspective
as to the benefits, limitations and safety of a specific intervention.
Regulatory authorities, who approve the licensing of medicines, now
require the inclusion of HRQoL in clinical trials in addition to clinical
outcomes and therefore quality of life information is becoming important
in drug labelling claims.
Abbott's HRQoL research (undertaken at UCLan 1995-date) involves the
development and validation of two disease-specific health-related quality
of life instruments: the Cystic Fibrosis Quality of Life questionnaire
(CFQoL)1 and the LUPUSQoL2,3, an instrument to
evaluate the quality of life in people with systemic lupus erythematosus.
Key collaborators include Professor Webb, Consultant Physician, South
Manchester NHS Trust, Dr Conway, Consultant Physician, Leeds NHS Trust and
Dr Teh, Consultant Physician, East Lancashire NHS Trust, with peer
reviewed grants from NHS Executive North West R&D Directorate, Lupus
UK and Arthritis UK. The original papers, reporting the development and
validation of these instruments, were published in high quality medical
journals (CFQoL: Thorax [Impact Factor 8.38, 120+ citations];
LupusQoL: Arthritis Care Research [Impact Factor 3.73, 65+ citations]).
The CFQoL was developed prior to the regulatory bodies requiring the
inclusion of patient-reported quality of life instruments in clinical
trials, but the development and evaluation process and sustained
multidisciplinary research endeavour using the CFQoL, enabled Abbott to
develop expertise in quality of life measurement. The subsequent
development of the LupusQoL, at a time when HRQoL measurement was becoming
obligatory, enabled commercial activity.
Abbott's longstanding reciprocal relationships with multidisciplinary
health professionals (who use HRQoL questionnaires in their clinical
practice and research) and service users (whose completion of the
questionnaires indicate improved or deteriorating quality of life) enabled
methodologically rigorous research. This joint effort at every stage of
the development and evaluation process (with patient and health
professional users contributing a wealth of expert knowledge and
experience) has resulted in comprehensive instruments in which each item
is important and meaningful for people with the condition. The
psychometric properties of the instruments have been demonstrated to be
robust with excellent levels of reliability and validity1,2.
International collaboration, and global interest from industry, has
resulted in these instruments being translated into numerous languages.
Further research has identified demographic, clinical and coping variables
that explain aspects of patient reports and aid data interpretation3,4.
For example, Abbott has shown that aspects of quality of life can predict
survival in cystic fibrosis5. This not only demonstrates the
significance of patient-reported outcomes in the clinical management of
the disease, but illustrates the importance of selecting such outcomes in
clinical trials. To aid the interpretation of the CFQoL by clinicians,
Abbott has subsequently evaluated the natural reporting of quality of life
over many years providing a benchmark to judge clinical relevance6.
Similarly, in collaboration with clinicians, a current national,
multi-centre study aims to provide information to help them easily
interpret the LupusQoL scores.
References to the research
The research underpinning the impact was published in high quality
international journals as evidenced below.
1. Gee L, Abbott J, Conway S, Etherington C, Webb AK. (2000). Development
and validation of a disease specific health related quality of life
measure for adults and adolescents with cystic fibrosis. Thorax, 55,
946-954.
2. MacElhone K, Abbott J, Teh LS. et al. (2007). The development and
validation of a disease- specific quality of life instrument for adults
with systemic lupus erythematosus. Arthritis Care and Research, 57,
972-979.
3. McElhone K, Abbott J, Gray J, Williams A, Teh LS. (2010). Patient
perspective of Systemic Lupus Erythematosus in relation to Health-related
Quality of Life Concepts. A qualitative Study. Lupus, 19, 1640-1647.
4. McElhone K, Castelino M, Abbott J, Bruce IN, Ahmad, Y, Shelmerdine J,
Peers K, Isenberg D, Ferenkeh-Koroma A, Griffiths B, Akil M, Maddison P,
Gordon C, Teh LS. (2010). The LupusQoL and associations with demographic
and clinical parameters in patients with Systemic Lupus Erythematosus
(SLE). Journal of Rheumatology, 37, 2273-2279.
5. Abbott J, Hart A, Morton AM, Dey P, Conway SP,
Webb AK. (2009). Can health-related quality of life predict
survival in adults with cystic fibrosis? American Journal of Respiratory
and Critical Care Medicine,17, 954-58.
6. Abbott J, Hurley MA, Morton AM, Conway SP. (2013). Longitudinal
Association between Lung Function and Health-Related Quality of Life in
Cystic Fibrosis. Thorax, 68:149-154.
Details of the impact
Policy development for clinical trials
The portfolio of sustained research activity has facilitated the building
of relationships with regulatory bodies, professional societies, user
groups and industry (e.g. European Medicines Agency, European Cystic
Fibrosis Society, UK Cystic Fibrosis Advocacy Service, Corporate
Translations Inc.). This has led to Abbott's expertise in quality of life
measurement being in demand. Abbott has been invited to participate in
continuous contributions to the UK Cystic Fibrosis Trust (Clinical Trials
Advisory Group) and the European Cystic Fibrosis Society (Scientific
committee 2009-date). Programmes of longitudinal research have
subsequently highlighted the importance of HRQoL measurement and allowing
service users a voice in their healthcare5,6,a. This research
has aided the development of European policy in relation to the use and
interpretation of quality of life data in clinical trials. In 2007, Abbott
contributed to the European Cystic Fibrosis Society consensus report
concerning clinical trials in cystic fibrosis. Subsequently, as part of
the European Framework 6 programme (LSHM-CT-2005-018932, EuroCareCF),
Abbott led the European quality of life group to produce the guidelines
for the measurement of quality of life in clinical trials in cystic
fibrosisb. Currently, Abbott is a member of an International
multidisciplinary group (invited by the US Cystic Fibrosis Foundation and
European Cystic Fibrosis Society) to develop and implement guidelines for
the assessment and management of anxiety and depression in cystic
fibrosis.
Additionally, Abbott was invited, by the European Cystic Fibrosis
Society, to join an interdisciplinary group of nine world experts on
Cystic Fibrosis to work with the European Medicines Agency (EMA). The EMA
is the body that regulates licences and constantly monitors the safety of
medicines across the European Union. Together with European drug
regulators, representatives from international pharmaceutical companies,
and patient representatives, Abbott discussed and compiled current
scientific evidence on outcome measures for evaluating medicines targeting
CF lung disease. She provided expert advice concerning patient-reported
outcome measures in clinical trials and contributed to the revision of the
EMA guidelines on `the clinical development of medicinal products for the
treatment of cystic fibrosis'. The proceedings were observed globally by
the US Food and Drugs Administration, Pharmaceuticals and Medical Devices
Agency, Japan and Health Canada via broadcastingc. This EMA
policy development has informed further research given the impetus to
develop a brief global instrument to measure patient-reported improvement
and treatment burden in cystic fibrosis clinical trials. Hence, impact is
a reciprocal two-way process.
Commercial applications outside academia
The LupusQoL has attracted global interest from the pharmaceutical
sector. Inclusion of a patient-reported quality of life instrument in
clinical trials is essential and will enable a pharmaceutical company to
gain approval to market a drug and make licensing claims as to the
effectiveness of the drug from the patient perspective. Driven by demand
from pharmaceutical companies to translate the LupusQoL into numerous
languages so that the instrument could be used in multinational drug
trials, a US translation company, Corporate Translations Incd,e,
have developed a new business opportunity by translating and culturally
validating the LupusQoL. This involves a lengthy and rigorous
harmonisation process and cognitive debriefing with patients to assess the
clarity and acceptability of each translation. They report the instrument
has been used successfully in 51 countries in 77 different languagesf.
UCLan's licensing agreement is for Corporate Translations Inc. to be the
sole worldwide distributor of the LupusQoL and as such they have developed
and maintain the websitee. The LupusQoL is currently being used
in several phase 111 clinical trials (predominantly in North America) to
evaluate whether a specific drug can improve a patient's quality of lifeg.
If the products are shown to be effective the pharmaceutical company will
be able to generate new and increased revenue streams (and patients will
benefit from a drug that can improve their quality of life).
Commercial exploitation of the LupusQoL by the University
Through UCLan's Innovation and Enterprise Unit and Strategic Development
Services a new business model was identified and negotiated with partners
(East Lancashire NHS Trust, Corporate Translations Inc.) to allow the
sharing of Intellectual Property incomeh. The LupusQoL is free
to non-commercially funded research projects and for use in clinical
practice, but there is a charge of £5.00 per patient entered into a
commercially funded research study. The licence income received from
pharmaceutical companies is approximately £32K; from the administration of
approximately 6400 LupusQol questionnaires in various international
clinical trials.
Education and Capacity building
The research programmes have contributed to individual career development
and the delivery of highly skilled people to the NHS and society. Dr Gee
(PhD student and Research Fellow involved in the CFQoL) operates a
clinical psychology consultancy service and Dr. McElhone (PhD student and
Research Associate involved in the LupusQoL) is employed as a Specialist
Lupus Research Nurse (East Lancashire NHS Trust) and Senior Research
Fellow (University of Manchester). They have acquired specialist knowledge
in quality of life measurement and patient-reported outcomesi.
Sources to corroborate the impact
Example of media coverage
a. American press — patient-reported quality of life predicts survival in
cystic fibrosis.
http://www.medpagetoday.com/Pulmonology/CysticFibrosis/12283
http://www.physorg.com/news149750128.html
Policy development — Consensus Reports for the European Cystic
Fibrosis Society
b. Abbott J, Hart A, Havermans T, Matossian A, Goldbeck L,
Barreto C, Bergsten-Brucefors A, Besier T, Catastini P, Lupi F & Staab
D. Measuring health-related quality of life in clinical trials in cystic
fibrosis. Journal of Cystic Fibrosis 2011;10,Suppl 1:S82-85.
Policy development in Clinical trials
c. European Medicines Agency (evidence of expert)
http://www.ema.europa.eu/docs/en_GB/document_library/Agenda/2012/07/WC500129307.pdf
http://www.ema.europa.eu/docs/en_GB/document_library/Report/2012/12/WC500136159.pdf
Commercial applications outside academia
d. Contact 1: Mary Gawlicki, President, Corporate Translations Inc.,
North America.
e. Corporate Translations Inc. host the LupusQoL website —
www.lupusqol.com
f. Evidence of language translations:
http://www.corptransinc.com/Sites/LupusQoL/Translations/Available-Translations.aspx
g. Evidence of the LupusQoL being used in clinical trials (all trials are
registered with
clinicaltrials.gov):
http://www.corptransinc.com/Sites/LupusQoL/Instrument-Information/Clinical-Trials.aspx
Commercial exploitation of the LupusQoL by the University
h. External evidence of income from the LupusQoL Contact 2: Catherine
Gedling, Research Governance Manager, East Lancashire NHS Trust
Education, capacity building and delivery of highly skilled people to
the NHS
i. PhD. Quality of life in adults with Cystic Fibrosis. (Louise Gee,
Awarded 1999, University of Central Lancashire).
http://www.pearceandgee.co.uk/services
PhD. Disease characteristics, treatment adherence and Quality of Life in
SLE. (Kathy McElhone, Awarded 2006, University of Central Lancashire).