Prof. Gavin GiovannoniBarts and The London School of Medicine and Dentistry

… dealing with increasing economic constraints

Disclosures

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

Micro-economic perspective

Rx=prescription drugs; OTC=over-the-counter.Berg J et al. Eur J Health Econ. 2006;7(suppl 2):S75-S85.

Total mean annual cost per patient €53,601

Healthcare costs are linked to disability

Informal Care (Disposable Income) (9.2%)

Ambulatory Care (5.6%)

Tests (0.4%)

Disease-Modifying Drugs (10.6%)

Other RX & OTC Drugs (1.6%)

Investments (2.0%)

Long-Term Sick Leave andEarly Retirement (30.0%)

Short-Term Absence(2.0%)

Services(28.5%)

Inpatient Care (10.2%)

Swedish Inflation Rate

Rx=prescription drugs; OTC=over-the-counter.Berg J et al. Eur J Health Econ. 2006;7(suppl 2):S75-S85.

Total mean annual cost per patient €53,601 = €69,000

Healthcare costs are linked to disability

Informal Care (Disposable Income) (9.2%)

Ambulatory Care (5.6%)

Tests (0.4%)

Disease-Modifying Drugs (10.6%)

Other RX & OTC Drugs (1.6%)

Investments (2.0%)

Long-Term Sick Leave andEarly Retirement (30.0%)

Short-Term Absence(2.0%)

Services(28.5%)

Inpatient Care (10.2%)

Rx=prescription drugs; OTC=over-the-counter.Berg J et al. Eur J Health Econ. 2006;7(suppl 2):S75-S85.

Total mean annual cost per patient €53,601 = €69,000

Healthcare costs are linked to disability

Informal Care (Disposable Income) (9.2%)

Ambulatory Care (5.6%)

Tests (0.4%)

Disease-Modifying Drugs (10.6%)

Other RX & OTC Drugs (1.6%)

Investments (2.0%)

Long-Term Sick Leave andEarly Retirement (30.0%)

Short-Term Absence(2.0%)

Services(28.5%)

Inpatient Care (10.2%)

Treatment should begin as early as possible after diagnosis of MS

Available at: www.msbrainhealth.org

IFNβ-1b a long term follow up: Time from study randomization to death

IFNβ, interferon beta.Goodin DS et al. Neurology. 2012;78:1315–22.

At risk:IFNβ-1b 250 µgPlacebo

124123

124120

121117

118109

10488

HR=0.532 (95% CI: 0.314–0.902)46.8% reduction in hazard ratio Log rank, P=0.0173

IFNB-1b 250 µg

Placebo

Early treatment with IFNβ-1b: Associated with 46.8% reduction in the hazard rate for mortality-NNT 8

IFNβ-1b 250μgPlacebo

Delayed Diagnosis

Kobelt G et al. Mult Scler. 2017;23:1123–36.

A

Utility: EQ-5D UK Value Set

N = 15,429

EQ-5D, EuroQol 5-dimensions questionnaire. Kobelt G et al. Mult Scler. 2017;23:1123–36.

Mean annual cost per patient

PPP, purchasing power parity.Kobelt G et al. Mult Scler. 2017;23:1123–36.

N = 16,808

Mean annual cost/patient

DMT, disease modifying therapy.Kobelt G et al. Mult Scler. 2017;23:1123–36.

N = 16,808

22,800€

57,800€

37,100€

Employment

Kobelt G et al. Mult Scler. 2017;23:1123–36.

Proportion of patients below retirement age (N = 13,391) employed or self-employed (N = 6,769)

Disease symptoms

Kobelt G et al. Mult Scler. 2017;23:1123–36.

MS was associated with fatigue in 95% of respondents, cognitive difficulties in 71%, effect on productivity while at work for 79%

Dependence on others: Informal care use

Kobelt G et al. Mult Scler. 2017;23:1123–36.

N = 7,176

Stroke or brain attack:‘Time really is brain’

Passive Activewww.msbrainhealth.org

International policy initiative

Rx=prescription drugs; OTC=over-the-counter.Berg J et al. Eur J Health Econ. 2006;7(suppl 2):S75-S85.

Informal Care (Disposable Income) (9.2%)

Ambulatory Care (5.6%)

Disease-Modifying Drugs (10.6%)

Other RX & OTC Drugs (1.6%)Tests (0.4%)

Investments (2.0%)

Long-Term Sick Leave andEarly Retirement (30.0%)

Short-Term Absence(2.0%)

Services(28.5%)

Inpatient Care (10.2%)

Healthcare costs are linked to disability

Total mean annual cost per patient €53,601 = €69,000

EmailSMS

PrivateePortal

LettersBlog Apps

GroupePortal

Clinic

GroupClinics

Tele-phone

Skype

Apps

Healthcare System / Service Innovation

Rx=prescription drugs; OTC=over-the-counter.Berg J et al. Eur J Health Econ. 2006;7(suppl 2):S75-S85.

Total mean annual cost per patient €53,601

Informal Care (Disposable Income) (9.2%)

Ambulatory Care (5.6%)

Disease-Modifying Drugs (10.6%)

Other RX & OTC Drugs (1.6%)Tests (0.4%)

Investments (2.0%)

Long-Term Sick Leave andEarly Retirement (30.0%)

Short-Term Absence(2.0%)

Services(28.5%)

Inpatient Care (10.2%)

Healthcare costs are linked to disability

Patent Expiration

1. Fingolimod2. Teriflunomide3. Oral cladribine

4. Biologicalsa. Rituximabb. Natalizumab

Globally there is unequal access to healthcare and DMTs

Micro-economic perspective

Thoughts & Issues to consider for a Humanitarian Aid Project

Slide courtesy Tim McCormick (Biogen)

Areas to address

Set-Up• Who decides on the merit of which countries would receive the aid?

– E.g. in MS… MSIF, EMSP, ECTRIMS, PACTRIMS?• Start with a pilot country(ies)? • Will such a program delay the local government setting up

infrastructure and a pathway to make X drugs for X disease available?

Slide courtesy Tim McCormick (Biogen)

Areas to address

• Logistics– How does the product get to Clinics?

• Logistics providers such as Direct Relief?– Adequate storage conditions in transit & at Clinics? E.g. 2-8゜ C– How to ensure product is NOT diverted into commercial channels?

Slide courtesy Tim McCormick (Biogen)

Areas to address

• Regulatory/Pharmacovigilance– What is required if the product has NOT been approved by the

destination country?• Waiver from Government?

– Clinics need to follow standard PV requirements– If product requires training, does the clinic have the

capacity/skill? If not how will training be conducted?

Slide courtesy Tim McCormick (Biogen)

Areas to address

Other• How long is an acceptable time period for a Company to commit?

– 5yrs, 10yrs, Indefinitely? • What volume?• How to engage all stakeholders in a Therapeutic Area?

Slide courtesy Tim McCormick (Biogen)

Epstein Bar Virus

Genetics

Vitamin D

Smoking

Risks

Adverse events

DifferentialDiagnosis

At riskRIS CIS Minimal

impairment

Moderateimpairment

Severeimpairment

Terminal Phase

MRI

EvokedPotentials

Lumbar puncture

BloodTests

DiagnosticCriteria

Cognition

Depression

Fatigue

Bladder

Bowel

Sexual dysfunction Tremor

PainSwallowing

SpasticityFalls

Balance problems Insomnia

Restless legsFertility

Clinical trials

Gait

Pressuresores

Oscillopsia

Emotionallability

Seizures

Gastrostomy

Rehab

Suprapubiccatheter Intrathecal

baclofen

Physio-therapy

Speech therapy

OccupationalTherapy

Functional neurosurgery

Colostomy

Tendonotomy

Studying

EmploymentRelationships

Travel

Vaccination

Anxiety

Driving

Nurse specialists

Family counselling

Relapses

1st line2nd line

Maintenance Escalation Induction

MonitoringDisease-free

Disease progression

DMTs

Side Effects

Advanced Directive

Exercise

Diet

AlternativeMedicine

PregnancyBreastFeeding

Research

Insurance

Visual loss

PalliativeCare

Assistedsuicide

Socialservices

Legalaid

Genetic counselling

PreventionDiagnosis

DMTSymptomatic

Therapist

Terminal

Counselling

Intrathecalphenol

Fractures

Movement disorders

Osteopaenia

Brain atrophy

Hearing loss

Tinnitus

PhotophobiaHiccoughs

DVLA

Neuroprotection

PsychosisDepersonaliation

BrainHealth

CognitiveReserve

Sudden death

SuicideOCD

NarcolepsyApnoea

Carers

Respite

HospiceRespite

Dignitas

Advanced Directive

Rhiztomy

Wheelchair

Walking aids

Blood/Organdonation

Brain donation

Exercise therapyNABs

Autoimmunity

Infections

Outcome measures

WebResources

Pathogenesis

Doublevision

What isMS?

NEDAT2T

OCTNeurofilaments

JCV statusPharma

Anaesthesia

Holistic approach to MS

www.ms-res.org

Unplanned hospital admissions (UPHA)

Main causes of UPHA

1. UTI2. Pneumonia3. Constipation (faecal impaction)4. Relapses5. Falls and fractures6. Pressure sores

Unplanned hospital admissions

Unplanned hospital admissions (UPHA)

Hackney: 2 MS clinical nurse specialistsTower Hamlets: 2 MS clinical nurse specialists

UPHA = 13.9/100,000/year

(£36,551/100,000/year)

Newham: 0 MS clinical nurse specialists

UPHA = 24.4/100,000/year

(£64,261/100,000/year)

Macro-economic perspective

Universities

CROs Legal

Regulatory

ManufacturingR&D

Lobbyists

Marketing

CME

ConsultanciesPolitics

Tax

Profits

Pensions, etc.

CEO bonuses, etc.

Pharma Gravy Train

2002

RISK SHARING SCHEME (RSS):COST EFFECTIVENESS PROVISION OF DMTS FOR PEOPLE

WITH MS

Slide courtesy of Jackie Palace

Off-label prescribing

MS treatments in Sweden

Courtesy of Jan Lycke

The Haves

The Have Nots

Charles Booth's Inquiry into the Life and Labour of the People in London(between 1886 and 1903)

Inequality

https://en.wikipedia.org/wiki/Gini_coefficient#/media/File:Gini_since_WWII.svg

The future

Rise of the Robots

MS Prevention

Conclusions1. 3+ years

a. Off-patentb. Off-labelc. Service/Systems innovationd. Technology

2. 10+ yearsa. Creative destructionb. MS prevention

3. Short-term a. Pharma innovation vs. healthcare economics (micro and macro)b. Need to balance micro-economic (personal) and macro-economic (societal) prioritiesc. Pharmaceuticals are an important driver of economic development

4. Humanitarian access schemesa. Complex b. Not easy to set-upc. WHO essential medicines list