Drug Development, EMA, Nice & NHS England: how treatments are developed and licensed

David Baker

Why Does it take so Long to get Drugs Available? Why Don’t Neurologists Develop More Drugs? Why Don’t We have Affordable Treatments?

Phase I-in Humans Safety

Ph

ase II in

MSe

rs –Safety &

Efficacy

Phase III in MSers –Efficacy (& Safety) Regulators

Toxicology in Animals

Ph

ase

O

Pre

-clin

ical

Te

stin

g in

Ce

lls &

An

imal

s

Licensing

Idea

>$1-4 Billion Dollars

10-15Years

DRUG DEVELOPMENT PROCESS

DRUG DEVELOPMENT PROCESS

PHARMA (HAVE RESOURCE) MADE FOR PROFIT

REGULATORS & ACADEMIA (LITTLE RESOURCE) BRIGHT IDEAS

TOGETHER THEY AIM TO DELIVER TREATMENTS EFFECTIVE & SAFE

REGULATORS

MEDICINES & HEALTHCARE PRODUCTS AGENCY (MHRA) IN UK COSTS OFFSET BY 100% PHARMA FUNDING

EUROPEAN MEDICINES AGENCY (EMA) IN EUROPE COSTS OFFSET BY 80% PHARMA FUNDING)

FOOD & DRUG ADMINISTRATION (FDA) IN USA

• MARKET AUTHORISATION (LICENSE HOLDERS) FOR SUPPLY OF DRUG Doctors should prescribe Licensed Therapies if available

• PRESCRIPTION & AVAILABILITY GUIDELINES

COST EFFECTIVENESS OF HEALTH CARE: MS DRUGS ARE NOT CONSIDERED COST-EFFECTIVE-RISK SHARING SCHEME COSTS OF MS DRUGS IN UK ARE LOWER THAN ELSEWHERE, BUT RATIONED

NATIONAL INSTITUTE OF HEALTH AND CLINICAL EXCELLENCE

Quality-adjusted life-year (QALY) is a measure of disease burden including the quality and quantity of Life. The QALY is based on the number of years of life that would be added by the intervention Suggested Limit for Cost effectiveness is about £30,000/QALY

SATIVEX: £49,300/QALY in UK (Lu et al 2012) €11,214/QALY in Germany (Slof & Gras 2012) FAMPYRA: £160,000/QALY in UK

NATIONAL INSTITUTE OF HEALTH AND CLINICAL EXCELLENCE

LICENSED PRODUCT FOR CANCER NEW USE IN MS

REPURPOSING

REPURPOSING FOR RRMS –LICENCED OPTIONS AVAILABLE (AZATHIOPRINE AS GOOD AS BETA INTERFERONS) REPURPOSING FOR PPMS/SPMS-NO LICENCED OPTIONS AVAILABLE

patent

THE PHARMA WAY REPURPOSING

THE PHARMA WAY REPURPOSING THEIR OWN DRUGS

REGULATORS: BARRIERS CREATED FOR PHARMA-HARD TO ACHIEVE FOR ACADEMICS FUNDERS: FINANCING TWO PHASE III DEVELOPMENT PLANS & REGULATORS DIFFICULT GOVERNMENT: NOT CREATING INCENTIVES/STRUCTURES

Cost: Simvastatin £2.70/week Cost Trial : £2-£50,000,000 Cost Regulators: £30,000/h

THE ACADEMIC WAY REPURPOSING

SCIENCE INNOVATION: IDENTIFY TARGETS LEAD TO PATENTS & COMMERCIAL DEVELOPMENT CLINICAL INNOVATION: IDENTIFY TARGETS FOR PHARMA EXPLOTATION IDENTIFTY TARGETS FOR PHARMA REPURPOSING LEAD TO PATENTS & COMMERCIAL DEVELOPMENT IDENTIFY METHODS TO DETECT EFFICACY RAPID PHASE II DESIGN ROBUST PHASE III TRIAL DESIGN SUPPORT THE CASE FOR A CHANGE IN REGULATION

THE ACADEMIC WAY REPURPOSING

CoI: Members of team are trying to commercially develop this drug We are shareholders/consultants and may gain financially from this.

DRUG DEVELOPMENT PROCESS

2002

2005

2013 2014 SAFE IN HUMANS (n=80)-NO SAE

VSN16 MADE. INHIBITS

SPASTICITY IN ANIMALS.

NO SEDATION

OVER 1000 FOLD THERAPEUTIC

WINDOW. NO TOXICITY IN RODENTS

NO REPRODUCTIVE TOXICITY

2011

2015

2015/2016

BIG PHARMA ACQUIRE LICENSE OPTION

PHASE II READY TO STATRT IN TWO MONTHS