Impact case study database

We have improved care for people with early and advanced Parkinson’s disease (PD) by defining a first line drug that improves the mobility and overall health of patients with early PD, demonstrating that surgical intervention can improve mobility for patients with advanced disease and downgrading guidance on use of rehabilitation therapies in early PD. We have achieved this through: (1) changing national guidelines for PD in the UK and Canada and by (2) changing practitioner best practice. As a result of these changes in therapy, NHS treatment costs for PD patients have been reduced.

1. UK and Canadian guidelines for PD have changed resulting in improved care practices for patients

In 2017, the UK National Institute for Health and Care Excellence (NICE) guidelines for the treatment of PD were changed. The key findings [KF1, KF2, KF3] from UoB trials were instrumental in underpinning many of these changes [S1]. NICE now recommends that clinicians:

1. “Offer levodopa to people in the early stages of Parkinson’s disease whose motor symptoms impact on their quality of life.” [S1. Recommendation 26. p. 74; from KF1].

2. “Consider deep brain stimulation for people with advanced Parkinson’s disease whose symptoms are not adequately controlled by best medical therapy.” [S1. Recommendation 89. p. 215; from KF2].

3. “Consider referring people who are in the early stages of Parkinson’s disease to

4. a physiotherapist with experience of Parkinson’s disease for assessment, education and advice, including information about physical activity.” [S1. Recommendation 69. p. 161; from KF3].

5. an occupational therapist with experience of Parkinson’s disease for assessment, education and advice on motor and non-motor symptoms.” [S1. Recommendation 72. p. 168; from KF3].

Prior to 2017, NICE did not specify which therapy to use as the first line treatment for PD patients with early disease. The Guideline Development Group (GDG) stated that when reaching their decision to recommend offering levodopa (Recommendation 26), they gave particular attention to KF1 from PD MED as “it was a long-term independently-funded study conducted in the UK” [S1. p. 64]. Similarly, PD SURG [R4] contributed significantly to NICE decisions regarding surgery guidelines as it was 1 of only 4 surgical studies considered [S1. p. 200]. UoB was also asked to provide additional data from PD SURG beyond the 1-year follow up reported in R4, which were used, along with R4, to determine guidance on use of surgery for early [S1. p. 217] and advanced PD [S1. p. 201] as well as to assess the cost–utility of surgery [S1. p. 204–5], which is an important factor in governing recommendation changes. UoB’s Professor Adrian Williams and Dr Caroline Rick, who had been involved in the design and conduct of PD SURG, were also invited as ‘Expert Witnesses’ to answer questions about the trial to assist the GDG in their review of evidence for DBS and subsequent decision to recommend DBS [S1. p. 198]. The provision of individual patient data from the PD SURG trial allowed NICE to perform their own health economics analysis which found the provision of DBS favourable.

PD REHAB [R6] was also deemed “of particular importance” by the GDG, as it was “a large, recent UK-based study” [S1. p. 159]. Based on KF3, NICE changed the 2006 guidance that physiotherapy and occupational therapy should be available to people with PD (CG35), to instead advise clinicians to ‘ consider’ rather than ‘ recommend’ physiotherapy and occupational therapy and stated that if offered, therapy should be given by therapists with experience of PD.

These UoB-driven changes to the NICE guidelines have been acknowledged as life changing for PD patients, as stated by the CEO for Parkinson’s UK, the world’s largest patient-led organisation for PD. Patients believe that through the changes to NICE guidelines in response to Birmingham’s research: “the quality of care for everyone affected by Parkinson’s disease in the UK has improved.” [S2]

The 2017 NICE guidelines [S1] have also underpinned subsequent changes to guidelines in Canada [S3 Ref 103], providing downstream impact of the influence of our research [KF1–KF3] in the UK. In 2019, the Canadian Guidelines for PD were modified to recommend that:

1. “Levodopa may be used as a symptomatic treatment for people with early Parkinson disease (Grade A).” [S3. Recommendation C31; from KF1].

2. “Deep brain stimulation of the subthalamic nucleus or the globus pallidus interna is effective against motor fluctuations and dyskinesia (grade A).” [S3. Recommendation C49; from KF2].

3. “Consideration should be given to referring people who are in the early stages of Parkinson disease to:

(1) a physiotherapist with experience of the disease for assessment, education and advice, including information about physical activity (grade B).” [S3. Recommendation C56; from KF3].

(2) an occupational therapist with experience of Parkinson disease for assessment, education and advice on motor and nonmotor symptoms (grade B).” [S3. Recommendation C58; from KF3].

2. Care practices for PD patients have changed, resulting in cost savings to the NHS

Use of levodopa as first line treatment for PD patients in the UK has increased. During this REF period, the use of levodopa has increased by 11.5% from 78.5% in 2013 to 90% in 2019, whilst the combined use of dopamine agonists and MAOBIs has decreased by 12% [S4]. Based on national estimates for new incident PD patients (18,461 in 2018), this means that 2,210 more PD patients are now treated initially with levodopa each year compared to in 2013 and should experience the improved quality of life that comes with this treatment. KF1 clearly drove these changes in treatment since use of levodopa first started to increase when Clarke first disseminated the results of PD MED at congress meetings around 2011 [S5]. Subsequently, during this REF period, publication of KF1 in 2014 [R2] and its incorporation in NICE guidelines in 2017 [S1] has supported a continuing rise in use of levodopa, which has increased by 7.5% since 2014, as in the year following each landmark, the percentage of PD patients in the UK who received levodopa increased.

International clinician awareness of levodopa as the best initial treatment for PD patients has been raised, through its inclusion in 2016 in Bradley’s Neurology in Clinical Practice [S6] — the main US textbook on Neurology — and by its inclusion along with KF2 and KF3 in the Birmingham Movement Disorders Course [S7] that is held bi-annually for 200 international specialist registrars in neurology and geriatrics.

Use of Deep Brain Stimulation surgery for patients with advanced PD has increased. More than 200 patients in the UK now receive DBS each year — an increase of 44% compared with 2013/2014. This increase in DBS was driven directly by KF2 and DBS usage has doubled since publication of PD SURG in 2010. It would be expected that this change in practice would substantially improve quality of life and mobility for these patients [S8].

Taken together, these research-led changes in treatment practice have also resulted in reduced treatment costs for PD patients, resulting in savings for the NHS. This is demonstrated by a cost-effectiveness analysis of PD MED which showed that initial treatment with levodopa, instead of levodopa-sparing therapies, would save the NHS an average of £3,390 per patient over 4 years [S9]. Based on 2,120 more PD patients now receiving levodopa as initial treatment each year, this represents an annual saving of £1.8m for the NHS [S7]. Furthermore, extrapolation of the PD SURG data, combined with assumptions concerning future costs and Quality Associated Life Years, suggests that use of deep brain stimulation surgery would be cost-effective for the NHS over 5 years [S10].