david baker ms research day
TRANSCRIPT
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