Report to the NHS Central Research & Development Committee
Annex 9 - Justifications of Priorities

JUSTIFICATION B
The organisation and evaluation of services, including medical, psychosocial, counselling and other services, to meet the needs of those found to be at high risk of cancer due to a genetic susceptibility

Background

During the past few years a number of genes, which in mutant form confer an increased risk of cancer, have been identified and characterised. These include genes for retinoblastoma, Wilm's tumour, neurofibromatosis, multiple endocrine neoplasia II, adenomatous polyposis coli, von Hippel-Lindau syndrome and the p53 gene, which when mutated in the germline predisposes to a variety of cancers. It is technically feasible to test for the presence of constitutional mutations in these genes in persons suspected to be carriers. However, such mutations are rare in the general population. Genes which have been located but not yet identified include the BRCA1 gene, which predisposes to breast and ovarian cancers, two genes for hereditary non-polyposis colon cancer, and the ataxia telangiectasia (AT) gene. Persons heterozygous for the AT gene appear to be at increased risk for a number of cancers including breast. At present it is possible to assess carrier status for these genes by linkage analysis in large informative families or by inference (i.e. obligate carriers) in certain other families. Once these genes are identified and characterised it will be possible to test individuals for carrier status regardless of family size and structure.

For those more common genes such as BRCA1, the gene for AT, and genes for hereditary colon cancer, the issue of general population screening arises. The identification of carriers of cancer susceptibility genes could be of great public health benefit, subject to the availability of efficient techniques for early detection of cancers which offer increased chance of cure, and advice on lifestyle habits to reduce cancer risk. As an alternative to general population screening, selected groups could be targeted e.g. all women with a first degree relative with breast cancer diagnosed under age 50 could be screened for BRCA1. In addition, individuals with a family history of cancer, or members of families with cancer predisposition syndromes, may present themselves to be referred for counselling, testing and screening.

As more genes are identified and with advances in molecular biological techniques for detecting mutations, there is likely to be an increasing demand for genetic testing. The management of those found to be carriers of high risk genes is uncertain. Screening for early detection of specific cancers is frequently advocated, but for some cancers e.g. premenopausal breast, this is of unknown effectiveness. The social and psychological effects of being found to be a carrier are also unknown.

Objectives

In order to avoid uncoordinated piecemeal services being set up for this group of people, various questions need to be answered by research.

Justification

Identification of individuals with high risk of cancer due to genetic susceptibility and their management is a major challenge to the NHS requiring appropriate choices of management and service provision.

JUSTIFICATION C
Factors influencing delayed presentation by patients (e.g. psychosocial) and variations in onward referrals by physicians to oncology specialists

Background

Delay in responding to symptoms may be associated with later stage at presentation (1) and shorter overall survival (2). The psychological factors underlying the variability in time to presentation are poorly understood, although suggestions have been made (3). If risk factors within the population who delay seeking medical attention could be identified, proactive interventions in primary care could be developed.

Studies in the international literature on cancer survival in relation to patterns of care indicate improved outcomes are associated with specialised care for uncommon cancers (4). Some studies also suggest this association exists for the more common cancers. The care of cancer involves the primary, secondary and tertiary services. Variations in onward referrals between clinicians will result in variation of care.

Objectives and justification

Understanding why such variation in referral occurs may allow interventions which should lead to consistency of better care.

References

JUSTIFICATION D
Factors influencing the accrual of patients into cancer trials

Background

Without doubt randomised clinical trials are needed to evaluate new and much needed treatments for the benefit of future cancer patients. Such trials have been highly influential in changing the clinical management of common cancers. Also, there is evidence to suggest that the outlook is better for those patients who are included in clinical trials. However, except for the childhood cancers, the leukaemias and some of the rare tumours, a small proportion of eligible patients are entered into trials. Adequate support for clinicians entering patients, failure to obtain consent and inadequate infrastructure for trials are all suggested to contribute to this shortcoming.

Objectives

Understanding more about what inhibits the accrual of patients into randomised trials may help point to ways which will influence a desirable increase.

Justification

Clinical trials are a major tool in evaluating cancer care. Methods to increase accrual into studies, when appropriate questions have been identified, are urgently needed.

References

JUSTIFICATION E
Studies designed to explain variations in disease outcomes, particularly in relation to variations in patterns of practice

Background

There is considerable geographical variation in standardised mortality rates from certain cancers both within the UK and internationally. It is thought to be unlikely that these differences in cancer survival are entirely due to known problems with the quality of data in cancer registries.

Studies usually do not include sufficient information to consider the patient and tumour prognostic variables which may relate to the observed outcome variations. A small number of studies point towards patterns of practice being relevant to outcome. This has been shown in ovarian and colorectal cancers, testicular cancer and paediatric oncology.

Objectives and Justification

Further studies are needed to extend this work into other tumour types and more importantly, to include studies designed to understand the genesis of the variations are needed and assess the quality of outcomes as well as survival.

References

JUSTIFICATION F
Comparison of care for common cancers in specialist and non-specialist treatment settings with respect to psychosocial and clinical outcomes; and the relative costs of managing each step of disease progression

Background

Patients with cancer may receive care in specialist or non-specialist care settings, or in a combination of both settings for specific active phases of treatment and for palliative care. For example, in advanced cancer, 19% of patients are admitted to a hospice at some point and 29% receive care from specialist community teams (1). However, these proportions vary greatly throughout the country (2). The benefits and disadvantages of these different treatment settings are poorly researched, especially with respect to psychosocial outcomes. Research is needed to compare clinical and psychological outcomes resulting from care in the different settings and to identify which types of patients and families will benefit most from care in the different settings.

Objectives

To compare clinical and psychological outcomes for patients cared for in different settings with different degrees of specialist involvement and evaluate cost effectiveness in the different settings. Studies may be appropriate in primary care, early management, specific surgical or active non-surgical treatment and palliative care.

Justification

To establish appropriate options for care and for service developments ensuring best clinical and psychological outcomes for cancer patients.

References

JUSTIFICATION G
The clinical utility of second-line chemotherapy in advanced common cancers, particularly its comparison to best supportive care

Background

The role of second-line chemotherapy in advanced cancer is understood to be palliative. Often treatments are given which have not been formally proven to be of benefit in the particular type of cancer being managed and these drugs have not been approved for such use by the licensing authorities. It is probable that these treatments are administered not infrequently more in hope than expectation of benefit, and because of a strong desire to be perceived as not having given up therapeutic hope.

Chemotherapy may be toxic and is potentially dangerous. It may lead to both a deterioration in the quality of life as well as a reduction of life. The option not to have a specific anti-cancer treatment is very difficult to define, present or measure. Surveys of attitudes suggest that the frequency of ineffective interventions is unlikely to be reduced unless the professional and lay view of best supportive care is more clearly defined.

Objectives

Research into the clinical utility of chemotherapy in these circumstances and how it compares with alternative management strategies such as best supportive care is required.

Justification

Better definition of clinical utility (or lack thereof) for chemotherapies in these settings and a clearer understanding of patients' reasons for wishing to receive it or not and of professionals' wish (or not) to deliver it, should lead to more informed choices which may improve outcomes and may reduce toxicity and cost.

References

JUSTIFICATION H
Effective ways of helping health professionals break bad news, elicit concerns and respond to patients and relatives for their best psychological adjustment

Background

The extent to which cancer patients perceive that information given about their illness and treatment was adequate to their needs appears crucial in determining their subsequent psychological adjustment (1). Those who feel they were given too much or too little information are much more likely to develop clinical anxiety and/or depression (2). Moreover, these affective disorders are more likely if patients' concerns about their predicament remain undisclosed and unresolved (3). Unfortunately, few health professionals have been taught effective ways of breaking bad news, eliciting and responding to patients' concerns (4). Yet this is a source of considerable stress for them and leads to emotional exhaustion (5).

Objectives

Ways of helping health professionals break bad news, elicit and respond to patients' concerns need critically to be evaluated. This would also have great utility in relation to treating other forms of bad news.

Justification

Improvements in professional skills in this area should reduce the psychological morbidity of cancer.

References

JUSTIFICATION I
Optimal management strategies for unrelieved symptoms, including pain and non-pain, in cancer patients

Background

Symptom control has improved greatly in recent years. Standard approaches escalating from simple analgesics to opiates will be successful in controlling pain in most patients but in some patients control may be unsatisfactory. Unrelieved pain may be a consequence of inadequate treatment or, with incident and neuropathic pain, it may result from apparent resistance to seemingly optimal treatment.

Anorexia and asthenia, dyspnoea and sometimes nausea and vomiting may be intractable to all available treatments. A knowledge of the incidence of these problems and a cost benefit analysis of simple versus more complex treatment strategies would allow the development of an algorithm to facilitate the management of these different symptoms.

Objectives

To assess accurately the scale of the problem of unrelieved symptoms in cancer patients and evaluate existing and new treatment approaches. These may include the introduction of new drugs and methods, better usage of existing treatments and the impact of new service configurations.

Justification

Symptom control is a useful determinant of quality of life in cancer patients.

References

JUSTIFICATION J
The most appropriate and cost effective model of service delivery and level of provision of palliative care services, including the role of nurse practitioners in hospitals and the community, and for the care of patients dying in acute hospitals

Background

Specialist palliative care is provided by hospices and NHS palliative care units, by hospital palliative care teams, home care and day care services. Some patients also require long stay care. The components and balance of a model system for a health care district have not been determined. Service provisions have been more influenced by local fundraising efforts than by needs assessment.

Interest is increasing in alternative mixes of skills in palliative care teams and in extending the scope of the role of the nurse. Routine, regular monitoring of patients with advanced cancer is critical, but costly. It is not yet known which health care professionals might be best used to support this group of patients.

Most patients who die from cancer, die in an acute hospital bed. The advantages and disadvantages of designated palliative care beds in an acute hospital setting are not known. There is a lack of evaluative information about the composition and effectiveness of hospital palliative care teams.

Objectives

Research to clarify the most appropriate and cost effective models for delivering palliative care services is needed. This should include testing alternative approaches including different mixes of health care professionals in teams and the value of designated beds for palliative care in an acute hospital.

Justification

Further appropriate developments in these services should reduce morbidity and increase patient and carer satisfaction. Equity of quality and access for these services is essential.

References

Next Page (Annex 9 - High Priority Areas)
CRDC Report: Contents Page