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Tackling treatment uncertainties together

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Research Priorities: top 10s

This page lists the top 10 research priorities, shared by patients, carers and clinicians, for each completed JLA Priority Setting Partnership.

1. What management strategy should be adopted for the treatment of acne in order to optimise short and long-term outcomes?

2. What is the correct way to use antibiotics in acne to achieve the best outcomes with least risk?

3. What is the best treatment for acne scars?

4. What is the best way of preventing acne?

5. What is the correct way to use oral isotretinoin (Roaccutane) in acne in order to achieve the best outcomes with least risk of potentially serious adverse effects?

6. Which lifestyle factors affect acne susceptibility or acne severity the most and could diet be one of them?

7. What is the best way of managing acne in mature women who may/may not have underlying hormonal abnormalities?

8. What is the best topical product for treating acne?

9. Which physical therapies including lasers and other light based treatments are safe and effective in treating acne?

10. How long do acne treatments take to work and which ones are fastest acting?

1a. What are the adverse effects associated with long term use of short and long actingbronchodilators; inhaled and oral steroids; and combination and additive therapies in adults? (N.B this includes children aged 12 years old and over)

1b. What are the adverse effects associated with long term use of short and long acting bronchodilators; inhaled and oral steroids; and combination and additive therapies in children?

2. What is the most effective way of managing asthma with other health problems?

3. What are the key components of successful "Self Management" for a person with asthma?

4. What is the most effective strategy to educate people with asthma and health professionals about managing the adverse effects of drug therapies?

5. What is the most effective way of managing asthma triggers?

6. What is the role of complementary therapies in asthma management?

7. What are the benefits of breathing exercises as a form of physical therapy for asthma?

8. What type of patient (children and adults) and health professional education is most effective in gaining asthma control?

9. What is the most effective way to manage consultations and asthma control in adolescence and young people?

10. Psychological interventions for adults with asthma?

1. Does the timing and intensity of therapies (e.g. physical, occupational and speech and language therapy, ‘early intervention’, providing information etc.) alter the effectiveness of therapies for infants and young children with neurodisability, including those without specific diagnosis? What is the appropriate age of onset / strategies / dosage / direction of therapy interventions?

2. To improve communication for children and young people with neurodisability: (a) what is the best way to select the most appropriate communication strategies? And (b) how to encourage staff/carers to use these strategies to enable communication?

3. Are child-centred strategies to improve children’s (i.e. peers) attitudes towards disability (e.g. buddy or Circle of Friends etc) effective to improve inclusion and participation within educational, social and community settings?

4. Does appropriate provision of wheelchairs to enable independent mobility for very young children improve their self-efficacy?

5. Are counselling/psychological strategies (e.g. talking therapies) effective to promote the mental health of children and young people with neurodisability?

6. What is the (long term) comparative safety and effectiveness of medical and surgical spasticity management techniques (Botulinum neurotoxin A (BoNT-A), Selective Dorsal Rhizotomy (SDR), Intrathecal Baclofen (ITB), orally administered medicines) in children and young people with neurodisability?

7. Does a structured training programme, medicines and/or surgery speed up the achievement of continence (either/or faecal or urinary) for children and young people with neurodisability?

8. What strategies are effective to improve engagement in physical activity (to improve fitness, reduce obesity etc.) for children and young people with neurodisability?

9. Which school characteristics (e.g. policies, attitudes of staff etc.) are most effective to promote inclusion of children and young people with neurodisability in education and after-school clubs?

10. What is the long term safety, effectiveness and sustainability of behavioural strategies and/or drugs (e.g. melatonin) to manage sleep disturbance in children and young people with neurodisability (outcomes include time to onset, duration, and reducing impact on family)?

Other research questions considered at the priority setting workshop in ranked order

11. What is the safety and effectiveness of drugs compared to talking therapies (e.g. Cognitive Behavioural Therapy) to treat anxiety in children and young people with Autistic Spectrum Disorders?

12. Are any types of physical therapy (e.g. Bobath, Neuro-Developmental Therapy, conductive education, hydro, constraint, strength-training etc) more or less effective to promote motor functioning in children and young people with neurodisability (e.g. cerebral palsy, acquired brain injury)?

13. Are sensory processing/integration therapeutic programmes effective in improving behaviour and/or increasing play/participation for children and young people with neurodisability?

14. Are behavioural and sensory interventions (e.g. early intensive behavioural intervention, EarlyBird, encouraging socialisation with peers etc.) effective in managing symptoms of Autistic Spectrum Disorder?

15. Are postural management programmes (using standing frames and sleep systems etc) effective and cost effective to prevent deformity (hip and/or spine) and improve function in children and young people with neurodisability? What is the incidence of adverse effects e.g. pain, sleep problems etc.?

16. Are child-focused strategies (e.g. one-to-one or group social and skills training) effective to improve confidence, self-esteem and promote participation in recreation and leisure activities for children and young people with neurodisability?

17. Do cross-sector interventions (e.g. key workers, named contacts to promote integrated health, social care, education) improve school attendance, reduce admissions, and parents’ satisfaction and experience of care for children and young people with neurodisability?

18. Does promoting public positive attitudes towards disability improve participation in recreation and leisure activities for children and young people with neurodisability?

19. Are dietary modifications/restrictions (e.g. gluten, casein, dairy, meat etc.) effective in managing symptoms of Autistic Spectrum Disorder?

20. Are oro-motor treatment strategies (e.g. oral motor exercises, sensory stimulation, sensorimotor activities etc.) effective to improve eating and drinking or speech for children and young people with neurodisability? Are there identifiable subgroups that benefit more from the strategies?

21. Are any designs of orthoses (e.g. Lycra, kinesiotaping, plastic etc.) more or less effective to promote functioning and prevent deformity for children and young people with neurodisability?

22. Are interventions to improve consistency of approach between health and education agencies (e.g. keyworkers) effective to improve behavioural problems in children with Autistic Spectrum Disorder (ASD)?

23. What is the long term safety and effectiveness of drugs used in seizure management, especially in terms of adverse effects on learning, psychosis, anxiety, anger and rage?

24. Does using instrumented gait analysis improve decision-making about treatments compared to clinical assessment alone for children and young people with cerebral palsy?

25. Do massage-based therapies improve functioning and wellbeing for children and young people with neurodisability?

1. What types of psychological intervention (individual therapy, community or school based) and at what time (from diagnosis to adulthood) are most helpful for patients with a cleft of the lip and/or palate and their families?

2. What are the educational, employment and personal (eg relationships) outcomes for individuals with a cleft of the lip and/or palate during childhood, adolescence and in the long term?

3. What is the best protocol for primary repair of both the lip and palate, including technique/timing and sequence?
4. In individuals with a cleft of the lip and/or palate when is the most effective age to begin speech therapy?

5. What is the best treatment for otitis media with effusion (glue ear) in individuals with a cleft of the lip and/or palate?

6. Can stem cells be used to improve palate repair (both primary and secondary)?

7. What interventions would enhance the educational outcomes for children with a cleft of
the lip and/or palate?

8. What is the impact of having a baby born with a cleft of the lip and/or palate on maternal/child attachment?

9. What is the best way to manage infants with a cleft of the lip and/or palate undergoing primary surgery before, during and after hospital Eg. Fluids, pain control, antibiotics, probiotics, arm splints, feeding practices?

10. What are the genetic and environmental causes of clefts of the lip and/or palate?

11. How can we improve the diagnosis of cleft palate (without cleft lip)?

12. What is the best way to prevent tooth decay in children with a cleft of the lip and/or palate?

1. What are the most effective components of care that keep a person with dementia as independent as they can be at all stages of the disease in all care settings?

2. How can the best ways to care for people with dementia, including results from research findings, be effectively disseminated and implemented into care practice?

3. What is the impact of an early diagnosis of dementia and how can primary care support a more effective route to diagnosis?

4. What non-pharmacological and/or pharmacological (drug) interventions are most effective for managing challenging behaviour in people with dementia?

5. What is the best way to care for people with dementia in a hospital setting when they have acute health care needs?

6. What are the most effective ways to encourage people with dementia to eat, drink and maintain nutritional intake?

7. What are the most effective ways of supporting carers of people with dementia living at home?

8. What is the best way to care for people with advanced dementia (with or without other illnesses) at the end of life?

9. When is the optimal time to move a person with dementia into a care home setting and how can the standard of care be improved?

10. What are the most effective design features for producing dementia friendly environments at both the housing and neighbourhood levels?

1. What is the best way to enhance communication between health care professionals and patients and to maximize patient participation in decision-making with regards to the advantages and disadvantages of different forms of dialysis, and access to test results to facilitate self-management?

2. How do different dialysis modalities compare in terms of their impact on quality of life, mortality and patient acceptability, and are there specific patient factors that make one modality better for some patients with kidney failure than others?

3. What are the causes and effective treatment(s) of, and ways to prevent, itching in dialysis patients?

4. What is the best strategy to increase kidney transplantation; including access to transplantation, increasing the efficiency of the recipient workup, and increasing the availability of donor kidneys?

5. What is the psychological and social impact of kidney failure on patients, their family, and other caregivers, and can this be reduced?

6. What are the best ways to promote heart health in dialysis patients, including management of blood pressure?

7. For people with kidney failure, what is the impact of each of the dietary restrictions (sodium, potassium, phosphate) separately, and when taken in combination, on important outcomes including quality of life?

8. What are the best ways to manage symptoms in people on or nearing dialysis including poor energy, nausea, cramping, and restless legs?

9. What are the causes and effective treatment(s) of depression in dialysis patients?
The uncertainties were expressed nearly equally by patients and health professionals. There were concerns about the emotional effects of dialysis on the patient; how to manage mood changes and depression, and what may be responsible for depression – i.e. the side effects of medications or other treatments, or kidney disease itself.

10. What is the best vascular access (among both new and existing types of access) for people on hemodialysis?

1. What is the optimal process for GP education and training for improved diagnosis and management of balance disorders?

2. What is the best way of training health professionals in the management of balance disorders?

3. What is the most effective treatment for Vestibular migraine?

4. What are the best interventions to improve balance/minimise symptoms in daily activities such as supermarkets, escalators etc.?

5. Is any specific surgical intervention effective in Ménière's disease and what procedure is best?

6. Are there any effective interventions for the ear pressure symptoms in Ménière's disease?

7. What is the optimum pharmacological strategy for the management of patients with Ménière's disease? In particular, what are the effects of betahistine (including long term effects)?

8. Is it helpful in preventing the severity, frequency and progression of attacks of Ménière's disease to adopt a specific diet, or restrict salt, caffeine or fluid intake?

9. Are the home-based exercises given to patients with balance disorders effective?

10. Are stress management techniques helpful in patients with balance disorders?

For more information on the Balance Partnership, click here.

Shared Priorities:

What is the best and safest way of using topical steroids for eczema: frequency of application, potency, length of time, alternating with other topical treatments, and age limits for treatment?

What is the long term safety of applying steroids to the skin for eczema?

What role might food allergy testing play in treating eczema?

Which emollient is the most effective and safe in treating eczema?

Patient and Carer Priorities:

What is the best psychological treatment for itching/scratching in eczema?

Which is the best way for people with eczema to wash: frequency of washing, water temperature, bath versus shower?

What are the best and safest natural products to apply to the skin for eczema?

How much does avoidance of irritants and allergens help people with eczema?

What is the role of diet in treating eczema: exclusion diets and nutritional supplements?

Healthcare Professional Priorities:

Which is more effective in the management of eczema: education programmes, GP care, nurse-led care, dermatologist-led care or multi-disciplinary care?

Which is safer and more effective for treating eczema: steroids or calcineurin inhibitors?

How effective are interventions to reduce skin infections in the management of eczema?

Which should be applied first when treating eczema, emollients or topical steroids?

What is the best and safest way of using drugs that suppress the immune system when treating eczema?

1. What is the most effective and safe group of oral treatments in treating Hidradenitis Suppurativa (HS)? (e.g. antibiotics, hormonal treatments, retinoids, immunosuppressants, metformin, steroids)

2. What is the best management of an acute flare?

3. What is the impact of HS and the treatments on people with HS? (physical, psychological, financial, social, quality of life)

4. How effective are biologics (etanercept, adalimumab, infliximab, ustekinumab) in treating HS

5. Does early diagnosis and aggressive treatment influence the course of HS?

6. What is the best surgical procedure to perform in treating HS e.g. incision & drainage, local excision, wide excision?

7. Which factors are useful in determining the prognosis (disease progression) of HS?

8. What is the best method of wound care after surgery or for active disease? (e.g. skin grafts, secondary intention, dressings)

9. To what extent is HS caused by genetic factors?

10. What is the best management of pain associated with HS?

1. What are the most important patient and clinical outcomes in hip and knee replacement surgery, for people with OA, and what is the best way to measure them?

2. What is the optimal timing for hip and knee replacement surgery, in people with OA, for best post-operative outcomes?

3. In people with OA, what are the pre-operative predictors of post-operative success (and risk factors of poor outcomes)?

4. What (health service) pre-operative, intra-operative, and post-operative factors can be modified to influence outcome following hip and knee replacement?

5. What is the best pain control regime pre-operatively, peri-operatively and immediately post-operatively for hip and knee joint replacement surgery for people with OA?

6. What are the best techniques to control longer term chronic pain and improve long term function following hip and knee replacement?

7. What are the long-term outcomes of non surgical treatments compared with operative treatment for patients with advanced knee/hip OA?

8. What is the most effective pre and post operative patient education support and advice for improving outcomes and satisfaction for people with OA following hip/ knee replacement?

9. What is an ideal postoperative follow up period and the best long term care model for people with OA that have had hip/knee replacement?

10. What is the best way to protect patients from the risk of thrombotic (blood clots, bleeding) events associated with hip/knee replacement?

1. How can patients who may benefit from intensive care be identified early and admitted to the ITU at the right time?

2. How can patients and their families be best supported as they start living at home again (e.g. health and social care services, ICU support groups, long term follow-up)?

3. What is the best way to identify patients with, or at risk of delirium or agitation – how should the immediate and long term effects of delirium or agitation be monitored and managed?


What is the best way to prevent, diagnose and treat hospital acquired infection (e.g. ventilator associated pneumonia, blood stream infections related to the use of invasive lines)?

When should physical rehabilitation start and what rehabilitation methods during and after critical illness achieve the best outcomes for patients?

How can we enhance patient comfort during Intensive Care (i.e. minimise pain, discomfort, agitation and anxiety) and does this improve patient outcome?

How can the physical consequences of critical illness (such as muscle wasting, weakness, nerve damage) be prevented and what is the best way to support recovery from these after intensive care?

What psychological support should be provided for patients in Intensive Care?

How can we predict who will benefit from intensive care before admission and during treatment in the ICU?

What is the best way of ensuring new knowledge and the latest defined standards are introduced into clinical practice in a timely and effective way?

How can we use the experiences of patients and families to improve intensive care?

What is the best way of preventing damage to the lungs of patients receiving respiratory support (ventilation)?

• What is the best treatment for children and adults presenting with a) early Lyme disease without neurological involvement and not including erythema migrans and b) late Lyme disease of any manifestation? To include consideration of drug(s), dose, duration.

What key questions (clinical and epidemiological) should be considered to help make a diagnosis of Lyme disease in children and adults in the UK and would a weighting table be useful?

How effective are the current UK tests in detecting infections due to the genospecies and strains of B burgdorferi sl in the UK and which single test and what combination of tests performs best in diagnosing or ruling out active Lyme disease. Should stage of the disease and patient age be taken into account when interpreting these tests?

What are the outcomes of cases where long term treatment has been used?

What is the optimal course of action if symptoms relapse after a treatment course is finished?

What is the optimal course of action if symptoms persist after initial treatment: should antibiotic treatment be continued until all symptoms have resolved or should a different dose or different antibiotic be used and what is the course of action if treatment appears to fail completely?

Are continuing symptoms following conventional recommended treatment due to continued infection, or an immune response or other process?

How common is relapse and treatment failure and is it related to disease stage, gender, co-infections or any other factor?

Are there long-term consequences if treatment is delayed?

Can Lyme be transmitted via other means: person to person sexually, transplacentally or by breast feeding; through organ donation; through blood transfusion?

1. Does boosting the immune system (using new agents such as PD-1 or PD-L1) improve response and survival rates for mesothelioma patients?

2. Can individualised chemotherapy be given to mesothelioma patients based on predictive factors (e.g. the subtype of mesothelioma (epithelioid, sarcomatoid, or mixed), or thymidine synthase inhibitor status (the protein that drugs like pemetrexed act against), etc)?

3. What is the best way to monitor patients with diffuse pleural thickening and a negative biopsy who are considered to have a high risk of developing mesothelioma (e.g. repeat biopsies, imaging, etc)?

4. In mesothelioma patients, what is the best second line treatment (i.e. what chemotherapy drugs should be used if a cancer has recurred following first line chemotherapy, usually with cisplatin and pemetrexed)?

5. Which is the most effective current treatment for ascites (excessive accumulation of fluid in the abdominal cavity) (e.g. denver shunt, pleurex catheter, etc) in patients with peritoneal mesothelioma?

6. What are the relative benefits of immediate standard chemotherapy compared to a watch and wait policy for mesothelioma patients?

7. For mesothelioma patients, what is the best follow-up strategy post-treatment, to identify and treat emerging side effects and other problems?

8. In mesothelioma, is there a role for intrapleural immunostimmulants (a drug designed to stimulate an anti-cancer immune response, such as corynebacterium parvum extract) in addition to any other treatment?

9. Does an annual chest x-ray and/or CT scan and medical examination in high-risk occupations (e.g. carpenters, plumbers, electricians, shipyard workers) lead to earlier diagnosis of mesothelioma?

10. What, if any, are the benefits of pleurectomy (pleurectomy/decortication) compared to no surgery, and which mesothelioma patients might benefit?

In addition to the top 10, the workshop participants requested that the following three questions also receive a special mention for their importance:

11. Can PET-CT scans (which produce 3D images of the inside of the body) help to diagnose mesothelioma (as well as aiding the assessment of response to treatment)?

12. How can the levels of mesothelin (a protein present in mesothelioma cells that can be measured in the blood) best be incorporated in the diagnosis, response and progression of mesothelioma?

13. What is the best current treatment for breathlessness in mesothelioma patients (e.g. exercise, handheld fans, etc)?

1. Which treatments are effective to slow, stop or reverse the accumulation of disability associated with MS

2. How can MS be prevented?


3. Which treatments are effective for fatigue in people with MS?

4. How can people with MS be best supported to self-manage their condition?

5. Does early treatment with aggressive disease modifying drugs improve the prognosis for people with MS?

6. Is vitamin D supplementation an effective disease modifying treatment for MS?

7. Which treatments are effective to improve mobility for people with MS?

8. Which treatments are effective to improve cognition in people with MS?

9. Which treatments are effective for pain in people with MS?

10. Is physiotherapy effective in reducing disability in people with MS?

1. What are the best ways of providing palliative care outside of working hours to avoid crises and help patients to stay in their place of choice? This includes symptom management, counselling and advice, GP visits and 24-hour support, for patients, carers and families.

2. How can access to palliative care services be improved for everyone regardless of where they are in the UK?

3. What are the benefits of Advance Care Planning and other approaches to listening to and incorporating patients’ preferences? Who should implement this and when?

4. What information and training do carers and families need to provide the best care for their loved one who is dying, including training for giving medicines at home?

5. How can it be ensured that staff, including healthcare assistants, are adequately trained to deliver palliative care, no matter where the care is being delivered? Does increasing the number of staff increase the quality of care provided in all settings? To what extent does funding affect these issues?

6. What are the best ways to determine a person’s palliative care needs, then initiate and deliver this care for patients with non-cancer diseases (such as chronic obstructive pulmonary disease (COPD), heart failure, motor neurone disease (MND), AIDS, multiple sclerosis, Crohn’s disease, Parkinson’s disease, dementia, and stroke)?

7. What are the core palliative care services that should be provided no matter what the patients’ diagnoses are?

8. What are the benefits, and best ways, of providing care in the patient’s home and how can home care be maintained as long as possible? Does good coordination of services affect this?

9. What are the best ways to make sure there is continuity for patients at the end of life, in terms of the staff that they have contact with, and does this improve quality of palliative care? Would having a designated case coordinator improve this process?.

10. What are the best ways to assess and treat pain and discomfort in people at the end of life with communication and/or cognitive difficulties, perhaps due to motor neurone disease (MND), dementia, Parkinson’s disease, brain tumour (including glioblastoma) or head and neck cancer, for example?

1. Tackling problems with getting around safely and fear of falling emerged at the top of your priority list.

2. Feeling stressed, worried or anxious is common in Parkinson's and urgently needs further research.

3. Unwanted uncontrollable movements are a distressing side effect of Parkinson's treatment that affects many people with the condition.

4. We currently treat Parkinson's as if it's one condition, but there may be many different forms which need to be treated differently.

5. People with Parkinson's are at greater risk of dementia but at the moment we cannot identify who will go on to develop it, or prevent its onset.

6. These subtle changes in thinking, such as problems finding words and forgetfulness, can have a significant impact on quality of life.

7. Finding ways to monitor symptoms throughout the day would help people with Parkinson's know when to take their medication.

8. Sleep problems are one of the most common non-motor symptoms in Parkinson's and can have a huge impact on everyday life.

9. Problems with dexterity can make simple tasks, such as tying shoelaces and doing up buttons, impossible.
10. Many people with Parkinson's experience problems with bladder control at some stage which can really affect daily life.

1. How effective is repositioning in the prevention of pressure ulcers?

2. How effective at preventing pressure ulcers is involving patients, family and lay carers in patient care?

3. Does the education of health and social care staff on prevention lead to a reduction in the incidence of pressure ulcers and, if so, which are the most effective education programmes (at organisational and Health/Social Care level)?

4. What is the relative effectiveness of the different types of pressure relieving beds, mattresses, overlays, heel protectors and cushions (including cushions for electric and self-propelling wheelchairs) in preventing pressure ulcers?

5. What impact do different service models have on the incidence of pressure ulcers including staffing levels, continuity of care [an on-going relationship with same staff members] and the current organisation of nursing care in hospitals?

6. What are the best service models (and are they sufficiently accessible) to ensure that patients with pressure ulcers receive the best treatment outcomes (including whether getting people with pressure ulcers and their carers more involved in their own pressure ulcer management improves ulcer healing and if so, the most effective models of engagement)?

7. For wheelchair users sitting on a pressure ulcer, how effective is bed rest in promoting pressure ulcer healing?

8. How effective are wound dressings in the promotion of pressure ulcer healing?

9. Does regular turning of patients in bed promote healing of pressure ulcers?

10. Does improving diet (eating) and hydration (drinking) promote pressure ulcer healing?

11. How effective are surgical operations to close pressure ulcers?

12. How effective are topical skin care products and skin care regimes at preventing pressure ulcers?

1. Which interventions are most effective to predict or prevent preterm birth?

2. How can infection in preterm babies be better prevented?

3. Which interventions are most effective to prevent necrotising enterocolitis in premature babies?

4. What is the best treatment for lung damage in premature babies?

5. What should be included in packages of care to support parents and families / carers when a premature baby is discharged from hospital?

6. What is the optimum milk feeding strategy and guidance (including quantity and speed of feeding and use of donor and formula milk) for the best long-term outcomes of premature babies?

7. What is the best way to judge whether a premature baby is feeling pain (for example, by their face, behaviours or brain activities)?

8. Which treatments are most effective to prevent early onset pre-eclampsia?

9. What emotional and practical support improves attachment and bonding, and does the provision of such support improve outcomes for premature babies and their families?

10. Which treatments are most effective for preterm premature rupture of membranes?

11. When is the best time to clamp the umbilical cord in preterm birth?

12. What type of support is most effective at improving breast feeding for premature babies?

13. Which interventions are most effective to treat necrotising enterocolitis in premature babies?

14. Does specialist antenatal care for women at risk of preterm birth improve outcomes for mother and baby?

15. What are the best ways to optimise the environment (such as light and noise) in order to improve outcomes for premature babies?

1. How can over-treatment for prostate cancer be prevented by indentifying and excluding the treatment of harmless tumours?

2. Is there a genetic marker for prostate cancer that would be both more sensitive and more specific than PSA serum level?

3. What can be done to delay or prevent the onset of hormone independent prostate cancer?

4. Are there any dietary measures that can prevent prostate cancer or slow its progression?

5. Does serial PSA measurement in patients with prostate cancer accurately monitor disease progression?

6. Would prostate cancer screening targeted at high risk groups, i.e. those with positive family history, and ethnic minorities with higher rates, improve the outcomes of treatment in these groups?

7. Does active surveillance work for treatment of prostate cancer?

8. Is there a vaccine that can prevent prostate cancer?

9. Do variations in GP awareness of prostate cancer affect outcomes?

10. Are there any non-intrusive diagnostic tests that will identify aggressive prostate cancers whilst not identifying harmless cancers?

11. What is the effectiveness of new treatments for prostate cancer such as High Intensity Focused Ultrasound (HIFU) and Cryotherapy?

For more information on the Prostate Cancer Partnership, click here.

1. What is the best way to treat people with schizophrenia that is unresponsive to treatment?

2. What training is needed to recognize the early signs of recurrence?

3. Should there be compulsory community outpatient treatment for people with severe mental disorders?

4. How can sexual dysfunction due to antipsychotic-drug therapy be managed?

5. What are the benefits of supported employment for people with schizophrenia in terms of quality of life, self esteem, long-term employment prospects and illness outcomes?

6. Do the adverse effects of antipsychotic drugs outweigh the benefits?

7. What are the benefits of hospital treatment compared with home care for psychotic episodes?

8. What are the clinical benefits and cost-effectiveness of monitoring the physical health of people with schizophrenia?

9. What are the clinical, social and economic outcomes — including quality of life and the methods and effects of risk monitoring — of treatment by acute day hospitals, assertive outreach teams, in-patient units, and crisis resolution and home treatment teams?

10. What interventions could reduce weight gain in schizophrenia?

For more information on the Schizophrenia Partnership, click here.

Priorities for Age Related Macular Degeneration (AMD) research

1. Can a treatment to stop dry AMD progressing and/or developing into the wet form be devised?

2. What is the cause of AMD?

3. How can AMD be prevented?

4. Are there ways of restoring sight loss for people with AMD?

5. Can the development of AMD be predicted?

6. What is the most effective way to detect and monitor the progression of early AMD?

7. What factors influence the progression of AMD?

8. Can a non-invasive therapy be developed for wet AMD?

9. Can dietary factors, nutritional supplements, complementary therapies or lifestyle changes prevent or slow the progression of AMD?

10. What are the best enablement strategies for people with AMD?

Priorities for Cataract research

1. How can cataracts be prevented from developing?

2. Can the return of cloudy or blurred vision after cataract surgery known as posteriorcapsule opacity (PCO) or secondary cataract be prevented?

3. How can cataract progression be slowed down?

4. What alternatives to treat cataracts other than cataract surgery are being developed?

5. What is the cause of cataract?

6. How can cataract surgery outcomes be improved?

7. How safe and effective is laser assisted cataract surgery?

8. Should accommodative lenses be developed for cataract surgery?

9. What is the best measure of visual disability due to cataract?

10. Can retinal detachment be prevented after cataract surgery?

11. What are the outcomes for cataract surgery among people with different levels of cognitive impairment (whatever the cause but including dementia, stroke, neurological conditions, head injuries)?

Priorities for Childhood Onset Disorders research

1. How can cerebral visual impairment be identified, prevented and treated in children?

2. How can treatment for visual pathway damage associated with pre-term birth be developed?

3. How do we improve screening and surveillance from the ante-natal period through to childhood to ensure early diagnosis of impaired vision and eye conditions?

4. Can the treatment of amblyopia be improved to produce better short and long term outcomes than are possible with current treatments?

5. How can cataract be prevented in children?

6. What are the causes of coloboma and microphthalmia/anophthalmia and how can they be prevented?

7. Can vision be corrected in later life for people with amblyopia?

8. How can retinoblastoma be identified, prevented and treated in children?

9. Can better treatments for glaucoma in children be developed?

10. Can a treatment be developed to improve vision for people with albinism?

Priorities for Corneal and External Eye Conditions research

1. Can new therapies such as gene or stem cell treatments be developed for corneal diseases?

2. What is the most effective management for dry eye and can new strategies be developed?

3. Can treatments to save eye sight from microbial keratitis be improved?

4. How can the rejection of corneal transplants be prevented?

5. Can the outcomes of corneal transplantation be improved?

6. What causes keratoconus to progress and can progression be prevented?

7. Can non-surgical therapy be developed for Fuchs' corneal dystrophy?

8. Can corneal infections be prevented in high-risk individuals such as contact lens wearers?

9. What is the cause of keratoconus and can it be prevented?

10. What is the most effective management of ocular complications associated with Stevens Johnson Syndrome?

11. Can severe ocular surface disease in children, such as blepharokeratoconjunctivitis and vernal keratoconjunctivitis be managed better?

Priorities for Glaucoma research

1. What are the most effective treatments for glaucoma and how can treatments be improved?

2. How can loss of vision be restored for people with glaucoma?

3. How can glaucoma be stopped from progressing?

4. What can be done to improve early diagnosis of sight-threatening glaucoma?

5. What causes glaucoma?

6. What is the most effective way of monitoring the progression of glaucoma?

7. How can glaucoma patients with a higher risk to progress rapidly be detected?

8. Why is glaucoma more aggressive in people of certain ethnic groups, such as those of West African origin?

9. How can glaucoma be prevented?

10. Is there a link between treatment adherence and glaucoma progression and how can adherence be improved?

Priorities for Inherited Retinal Disease research

1. Can a treatment to slow down progression or reverse sight loss in inherited retinal diseases be developed?

2. How can sight loss be prevented in an individual with inherited retinal disease?

3. Is a genetic (molecular) diagnosis possible for all inherited retinal diseases?

4. What factors affect the progression of sight loss in inherited retinal diseases?

5. What causes sight loss in inherited retinal diseases?

6. What is the most effective way to support patients with inherited retinal disease?

7. Can the diagnosis of inherited retinal diseases be refined so that individuals can be given a clearer idea about their specific condition and how it is likely to progress?

8. What is the relationship between sight loss and mental health for people with inherited retinal diseases?

9. Would having a treatment for an inherited retinal disease preclude a patient from having another treatment?

10. With regard to inherited retinal disease what is the role of pre-natal and pre-implantation diagnosis in helping parents make informed choices?

Priorities for Neuro-ophthalmology research

1. What is the underlying cause of optic nerve damage in optic neuropathies, such as anterior ischaemic optic neuropathy, Leber's hereditary optic neuropathy, optic neuritis and other optic neuropathies?

2. What are the most effective treatments and rehabilitation for optic neuropathies, e.g. Leber's hereditary optic neuropathy and anterior ischaemic optic neuropathy?

3. Can vision loss due to optic nerve diseases such as giant cell arteritis, Leber's hereditary optic neuropathy, optic neuritis and optic atrophy, be restored, for example through gene therapy and stem cell treatment?

4. What rehabilitation or treatment methods are most effective for vision loss following brain damage due to stroke, brain injury, cerebral vision impairment, tumours and dementias?

5. What is the most effective way to assess vision in patients with neurological visual impairment i.e. stroke, dementia and cerebral/cortical visual impairment?

6. Can the early stages of optic neuropathy be detected?

7. How can optic neuropathies be prevented, for example anterior ischaemic optic neuropathy, Leber's hereditary optic neuropathy, optic neuritis and other optic neuropathies?

8. Can treatments be developed for visual field and ocular motility manifestations following stroke?

9. How can electronic devices improve or restore vision for people with optic neuropathies?

10. Can an alternative or new treatment be developed that will treat the sight loss caused by giant cell arteritis?

Priorities for Ocular Cancer research

1. What can be done to help ocular cancer sufferers?

2. Can gene-based targeted therapies for ocular cancers be developed?

3. How can immunotherapy be used to fight metastatic ocular melanoma?

4. What are the most effective detection and screening methods for follow-up to detect metastasis of ocular melanoma?

5. How can follow-up for ocular complications be managed in patients with ocular melanoma?

6. What is the best management of metastatic choroidal melanoma?

7. What activates choroidal melanoma metastasis in the liver after the primary melanoma has been treated?

8. Can adjuvant therapies be developed to treat ocular melanoma?

9. What are the causes of ocular cancer and how can they be prevented?

10. What is the most effective treatment for primary ocular melanoma?

Priorities for Ocular Inflammatory Disease research

1. What are the most effective treatments for ocular and orbital inflammatory diseases?

2. What causes thyroid eye disease?

3. Can the severity of ocular and orbital inflammatory disease in an individual be predicted?

4. Is it possible to prevent further occurrences of retinal damage caused by toxoplasmosis?

5. What causes birdshot retinopathy?

6. Why does disease burn out in patients with ocular and orbital inflammatory diseases?

7. Can early detection methods be developed for ocular and orbital inflammatory diseases?

8. What medications best prevent the development of eye disease in Behcets?

9. What causes scleritis?

10. Can diet or lifestyle changes prevent uveitis from developing?

Priorities for Refractive Error and Ocular Motility

1. What factors influence the development of refractive error (myopia, astigmatism, presbyopia, and long-sightedness)?

2. What is the cause of both congenital and acquired nystagmus?

3. How can the development of binocular vision in young children with squint and amblyopia be promoted, and would the same approach work in older individuals without inducing intractable diplopia?

4. Would correction of refractive error have a positive impact on early life learning and development?

5. Does early diagnosis of refractive error improve long-term prognosis and promote faster, more effective treatment?

6. What is the effect of congenital nystagmus on visual and emotional development?

7. What is the most effective treatment for exotropia and when should it be delivered?

8. How can the functional effects of surgical treatment for squint be best assessed?

9. Could the accurate testing of refractive error be made less dependent on a subjective response, i.e. the person's own response?

10. How can myopia be prevented?

Priorities for Retinal Vascular Disease research

1. What are the best methods to prevent retinopathy of prematurity?

2. How can sight loss from diabetic retinal changes be prevented and reduced?

3. What are the predictive factors for the progression to sight threatening diabetic eye disease?

4. Is there a way to improve screening of premature babies for retinopathy of prematurity?

5. Can an effective long lasting treatment for diabetic macular oedema, both ischaemic and non-ischaemic, be developed?

6. Can a retinal vein occlusion be predicted and prevented?

7. Can new non-invasive treatments be developed to slow down the progression of diabetic retinopathy?

8. What are the barriers that prevent diabetic patients having regular eye checks?

9. What rehabilitation programmes are best for the management of distorted vision from retinal diseases?

10. What is the efficacy and safety of anti-VEGF agents in the treatment of retinopathy of prematurity?

Priorities for Vitreoretinal/Ocular Trauma research

1. How can surgical techniques be improved to save sight for eyes damaged by injury?

2. How can the risk of losing sight for people with retinal detachment be reduced?

3. How can better interventions be developed that are effective in treating vitreous opacities/eye floaters?

4. What causes retinal detachment and can it be prevented?

5. Can more effective diagnostic tools be developed for assessing the vitreous and eye floaters?

6. Can a functioning prosthetic eye be developed to replace an eye damaged by injury?

7. How can epiretinal membrane/fibrosis be prevented or treated?

8. Can stem cells be used to regrow an eye or part of an eye?

9. What causes posterior vitreous detachment/vitreous syneresis?

10. Are there methods to prevent and improve the treatment of macular holes?

1. Does activity based rehabilitation, including functional electrical stimulation coupled with physical activity and hydrotherapy, improve outcomes such as muscle function and neuroplasticity after spinal cord injury?

2. Does stem cell therapy result in better outcomes after spinal cord injury and does this depend on the type of injury (e.g. acute or chronic; complete or incomplete)?

3. Does the provision of care packages in the community, including physiotherapy, after discharge from hospital improve the health and wellbeing of people living with spinal cord injury?

4. Which bladder management strategy is most effective in reducing the number of urinary tract infections and secondary complications that may result from these after spinal cord injury?

5. Does early mobilisation or a period of 4-6 weeks of physically active bed rest result in improved patient outcomes after surgical spinal column stabilisation?

6. Does discharge from a hospital to a physically enabling environment, including people and facilities which enable a person to become physically active, improve the quality of life of spinal cord injury patients?

7. Does the provision of specialist rehabilitation services, which includes multidisciplinary team planning, improve the health and wellbeing of spinal cord injury patients?

8. Do interventions including controlled fibre and fluid intake after spinal cord injury improve individuals' bowel function and quality of life?

9. What are the effects of ageing after spinal cord injury on the development of complications, including spasticity and bladder and bowel incontinence, and need for home-based support?

10. Does early diagnosis and treatment lead to improved outcomes for people with (a) cauda equina syndrome and (b) transverse myelitis (including relapses)?

1. What are the best ways to improve cognition after stroke?

2. What are the best ways of helping people come to terms with the long term consequences of stroke?

3. What are the best ways to help people recover from aphasia?

4. What are the best treatments for arm recovery and function, including visual feedback, virtual reality, bilateral training, repetitive task training, imagery/mental practice, splinting, electromechanical and robot-assisted arm training and botulinum toxin.

5. What are the best ways to treat visual problems after stroke?

6. What are the best ways to manage and/or prevent fatigue?

7. What are the best treatments to improve balance, gait and mobility, including physiotherapy, gait rehabilitation, visual and auditory feedback, electrical stimulation, different types of ankle foot orthoses and electromechanical assisted gait training?

8. How can stroke survivors and families be helped to cope with speech problems?

9. What are the best ways to improve confidence after stroke, including stroke clubs/groups, offering support, one-to-one input and re-skilling?

10. Are exercise and fitness programmes beneficial at improving function and quality of life and avoiding subsequent stroke?

For more information on the Stroke Partnership, click here

  • What management strategies are more effective than a usual model of audiological care in improving outcomes for people with tinnitus?
  • Is Cognitive Behaviour Therapy (CBT), delivered by audiology professionals, effective for people with tinnitus? Here comparisons might be with usual audiological care or CBT delivered by a psychologist.
  • What management strategies are more effective for improving tinnitus-related insomnia than a usual model of care?
  • Do any of the various available complementary therapies provide improved outcome for people with tinnitus compared with a usual model of care?
  • What type of digital hearing aid or amplification strategy provides the most effective tinnitus relief?
  • What is the optimal set of guidelines for assessing children with tinnitus?
  • How can tinnitus be effectively managed in people who are Deaf or who have a profound hearing loss?
  • Are there different types of tinnitus and can they be explained by different mechanisms in the ear or brain?
  • What is the link between tinnitus and hyperacusis (over-sensitivity to sounds)?
  • Which medications have proven to be effective in tinnitus management compared with placebo?

The following question was carefully deliberated and just fell outside of the top ten, however participants in the final meeting felt it was worthy of a special mention:

  • Does stem cell technology (e.g. for hair cell regeneration) provide an effective pipeline to develop new medical interventions for treating

Overarching research aspiration: An effective cure for type 1 diabetes

1. Is it possible to constantly and accurately monitor blood sugar levels, in people with type 1 diabetes, with a discrete device (non-invasive or invasive)

2. Is insulin pump therapy effective? (immediate v deferred pump, and comparing outcomes with multiple injections)

3. Is an artificial pancreas for type 1 diabetes (closed loop system) effective?

4. What are the characteristics of the best type 1 diabetes patient education programmes (from diagnosis to long term care) and do they improve outcomes?

5. What are the cognitive and psychological effects of living with type 1 diabetes?

6. How can awareness of and prevention of hypoglycaemia in type 1 diabetes be improved?

7. How tightly controlled do fluctuations in blood glucose levels need to be to reduce the risk of developing complications in people with type 1 diabetes?

8. Does treatment of type 1 diabetics by specialists (e.g. doctors, nurses, dieticians, podiatrists, ophthalmologists and psychologists) trained in person-centred skills provide better blood glucose control, patient satisfaction and self-confidence in management of type 1 diabetes, compared to treatment by non specialists with standard skills?

9. What makes self management successful for some people with type 1 diabetes, and not others?

10. Which insulins are safest and have the fewest long term adverse effects?

For more information about the Type 1 Diabetes Partnership, click here.

1. What are the optimal pelvic floor muscle training protocols (frequency and duration of therapy) for the treatment of different patterns of urinary incontinence?

2. Can guidance or training for general practitioners on appropriate pathways of care improve the management of patients with urinary incontinence?

3. What is best practice for the treatment of combined stress urinary incontinence and detrusor overactivity?

4. What catheter regimens are most effective in preventing urinary tract infections in patients using intermittent self-catheterisation for the management of a neurogenic bladder?

5. Which treatment is most effective for the reduction of urinary frequency and urgency?

6. Is urodynamic testing prior to surgery for urinary incontinence associated with better continence rates and quality of life than surgery indicated without such testing?

7. What is best practice for the management of stress urinary incontinence following failed tension free vaginal tape surgery?

8. What are the most effective treatments of daytime urinary incontinence in children?

9. Are disposable catheters more or less acceptable than reusable catheters in terms of effective bladder management, patient experience and urinary tract infections?

10. In women with prolapse (symptomatic or asymptomatic) and SUI, should suburethral tapes be inserted at the same time as repairing the prolapse?

For more information about the Urinary Incontinence Partnership, click here.

1. How effective are systemic immunosuppressants in treating vitiligo?

2. How much do psychological interventions help people with vitiligo?

3. Which treatment is more effective for vitiligo: light therapy or calcineurin inhibitors?

4. How effective is ultraviolet B therapy when combined with creams or ointments in treating vitiligo?

5. What role might gene therapy play in the treatment of vitiligo?

6. How effective are hormones or hormone-related substances that stimulate pigment cells (melanocyte-stimulating hormone analogues, afamelanotide) in treating vitiligo?

7. Which treatment is more effective for vitiligo: calcineurin inhibitors or steroid creams/ointments?

8. Which treatment is more effective for vitiligo: steroid creams/ointments or light therapy?

9. How effective is the addition of psychological interventions to patients using cosmetic camouflage for improving their quality of life?

10. How effective is pseudocatalase cream (combined with brief exposure to ultraviolet B) in treating vitiligo?

In addition, two treatment uncertainties were suggested as ‘ones to watch’, as these interventions were still in an early investigative stage.

11. How effective is piperine (black pepper) cream in treating vitiligo?

12. What role might stem cell therapy play in treating vitiligo?

More more information on the Vitiligo Partnership, click here.

Copyright 2015 James Lind Alliance