What does the future hold for new tracking and assessment tools in MS?

Gavin Giovannoni

Barts and The London School of Medicine and Dentistry

London, UK

Disclosures

Professor Giovannoni has received personal compensation for participating onAdvisory Boards in relation to clinical trial design, trial steering committees and dataand safety monitoring committees from: Abbvie, Bayer-Schering Healthcare, Biogen-Idec, Canbex, Eisai, Elan, Fiveprime, Genzyme, Genentech, GSK, GW Pharma,Ironwood, Merck-Serono, Novartis, Pfizer, Roche, Sanofi-Aventis, Synthon BV, Teva,UCB Pharma and Vertex Pharmaceuticals.

Regarding www.ms-res.org survey results in this presentation: please note that nopersonal identifiers were collected as part of these surveys and that by completing thesurveys participants consented for their anonymous data to be analysed andpresented by Professor Giovannoni.

Relapses

Unreported relapses

Clinical disease progression

Subclinical relapses: focal MRI activity

Focal gray and white matter lesions not detected by MRI

Brain atrophy

Spinal fluid neurofilament levels

Treat-2-Target

Clinical activity

Focal MRI activity

Hidden focal and diffuse MRI activity

Microscopic or biochemical pathology

Biomarkers

Yes, 72

No, 28

Relapse reporting

• Most common reasons for not reporting their most recent relapse to a specialist MS team were: • ‘Mild relapse so not felt necessary’ 5/28 (18%)• ‘Saw or spoke to their GP’ 4/28 (14%)

• Most common reasons for not seeking healthcare support were:• ‘Felt I could manage’/mild relapse 18/42 (43%) • ‘Nothing that they can do to help’ 8/42 (19%)

Duddy M, et al. ECTRIMS 2013. P590.

N = 101

Yes, 46

No, 54

N = 102

Patients who have everexperienced an MS relapse and

not contacted a healthcare professional

Patients reporting most recentrelapse to a specialist MS team

Relapses

Unreported relapses

Clinical disease progression

Subclinical relapses: focal MRI activity

Focal gray and white matter lesions not detected by MRI

Brain atrophy

Spinal fluid neurofilament levels

Treat-2-Target

Clinical activity

Focal MRI activity

Hidden focal and diffuse MRI activity

Microscopic or biochemical pathology

Biomarkers

Yes – I do an EDSS whenever I see a patient 14 25%

Yes – I do an EDSS annually 10 18%

Yes – I occasionally do an EDSS 20 36%

No – I never do an EDSS 3 5%

Other 9 16%Yes – I do an EDSS whenever I see a

patient [14]Yes – I do an EDSS

annually [10]

Yes – I occasionally do an EDSS [20]

No – I never do an EDSS [3]

Other [9]

Survey of UK MSologists

Schmierer K, et al. ABN 2014; Unpublished.

Clinical – In your routine MS clinical practice, do you use the EDSS?

Clinical – If you do an EDSS in your routine clinical practice, do you walk the patients to assess their walking distance?

Yes [9]

No [20]

Sometimes [22]

Other [5] Yes 9 16%

No 20 36%

Sometimes 22 39%

Other 5 9%

Validating a novel web-based method to capture disease progression outcomes in MS

P-EDSS, physician or actual EDSS.The midpoint of the diamonds is the mean difference between the two EDSS scores, the upper and lower lines within the diamonds are the 95% CI. The width of the diamond indicates the sample size, the dots the actual values. The horizontal line at 0.46 indicates the mean difference between the two scores. The graph indicates the greater variation at lower EDSS scores, with greater agreement at scores > 5. Leddy S, et al. J Neurol 2013; 260:2505–2510.

ORIGINAL COMMUNICATION

we

b-

EDSS

–P

-ED

SS s

core

P-EDSS score

–4

–3

–2

–1

0

1

2

3

4

0 1 1.5 2 2.5 3 3.5 4 4.5 5.5 6 6.5 7 8

Cli

nic

Sp

eak

www.clinicspeak.com

Unmet Needs remain from both the MSer and HCP perspectives

UNMET NEEDSHCP Perspective

UNMET NEEDSPatient Perspective

Monitoring your own disease

PROMs / PROs

Generic

Outcome-specific

MS-specific

MS outcome-specific

•EQ5D

• SF36

•Barthel Index

•Berg Balance Scale

•Ambulation Index

•MSIS-29

•MusiQoL

•MSWS-12

•MSFIS

PROMIS Domain Framework

Self-Reported

Health

Social

Health

Mental Health

Physical Health

Symptoms

Function

Affect

Behavior

Cognition

Relationships

Function

Mental Health

Affect

Behavior

Testing

General Population

Clinical Samples

Analysis

Interpretation

Refining

Qualitative Research and Item Writing

Item Bank

Cycle of Development and Validation

Physical Functioning Item Bank

Item1

Item2

Item3

Item4

Item5

Item6

Item7

Item8

Item9

Itemn

100500

• Are you able to get in and out of bed?• Are you able to stand without losing your balance for 1 minute?• Are you able to walk from one room to another?• Are you to walk a block on flat ground?• Are you able to run or jog 2 miles?• Are you able to rund 5 miles?

Questionnaire Fatigue

Relapses

Unreported relapses

Clinical disease progression

Subclinical relapses: focal MRI activity

Focal gray and white matter lesions not detected by MRI

Brain atrophy

Spinal fluid neurofilament levels

Treat-2-Target

Clinical activity

Focal MRI activity

Hidden focal and diffuse MRI activity

Microscopic or biochemical pathology

Biomarkers

Conclusions

• eMonitoring and eHealth are here to stay

• Adoption is occurring very rapidly in some sectors

• Advantages are obvious

• Improved efficiency (cost and time effective)

• Better outcomes

• Higher satisfaction

• Hurdles to adoption are not insurmountable

• Privacy and data protection

• Medico-legal issues

• eMonitoring

• I don’t know how to do it yet

• Adoption, adherence, design, etc.

• automatic, smartphone (‘the great disruptor’)