Clinical Pharmacology - Regulation, Introduction and Monitoring of Medicines Flashcards
Who is going to be interested in your healthcare/prescribing?
- Public
- MPs (if you are a democracy)
- Industry
- Doctors/Pharmacists
- Media
You are dictator-for-life!
How are you going to Structure your health Care system?
what questions do you need to think about?
Who is going to be interested in your healthcare/ prescribing?
Who will you allow to prescribe?
Can your prescribers prescribe whatever they want to prescribe?
Drug companies have hiked _____ exorbitantly over the past several years
prices
Generic drugs should be cheaper once patent ends
Mylan pharmaceuticals: 548% increase in price of EpiPen
Isuprel and Nitropress: Valeant Pharmaceuticals increased costs of:
- Isuprel: $180 to $1,472 a vial, a 718 percent increase.
- Nitropress ($215 a vial to $881) a 300% increase
Valeant: bought ‘old’ drugs and increased their prices precipitously
how has spending on presciption drugs changed?
Number of drugs being prescribed for older people or more conditions is increased
what is the price difference of drugs like between he US and UK?
Same drug in us cost much more than it would in Europe and uk
Another drug showing the difference in price in the UK and Switzerland compared to the US
Pharmaceutical advertising - Direct to consumer advertising - what is it?
the pahrma companies advertising directly to comsumers
very expensive
Only 2 countries you can advertise direct to the consumers in the US and New Zealand
Pharma direct to consumer advertising - what are the advantages?
Empowers patients
Disease education
Prompts patients to seek more timely medical advice and support
May result in earlier diagnosis and treatment
May improve adherence
Pharma direct to consumer advertising - what are the disadvantages?
Consumers think it increases prices (Consumers think it increases price as the 5.4 billion dollars has to be paid somewhere)
Reduces authority of the doctor (as patient more empowered)
Inflates demand for new/expensive drugs even if not appropriate
Off patent use (eg surgical glue)
Advertising to doctors:
Things are now very ________ regulated in the UK in terms of the amount of advertising and promotion that companies can do to your _______ professionals
tightly
medical
what are the statistics for prescribing in scotland? (2011-2012)
Total spend on the NHS is up to around 13 billion which is the same as the deficit
With all new drugs being considered for use in the general population you need to know what?
- Does it work?
- What dose is therapeutic?
- What dose is toxic?
- Is it safe?
- Is it necessary?
Who regulates drugs/devices in the UK?
Commission on Human Medicines (CHM)
Formerly Committee for Safety of Medicines (CSM)
what do Commission on Human Medicines (CHM) do?
Advises Ministers on matters relating to human medicinal products
Advises Licensing Authority (LA)
Considers representations by an applicant or MA (Marketing authorisation) holder
Promotes collection and investigation of information relating to adverse Drug Reactions to human medicines.
CMH sits within a bigger organisation called the MHRA
Medicines & Healthcare products Regulatory Agency (MHRA) –Executive Agency of DoH
what is their role?
Post-marketing surveillance – ADRs and incidents
Assessment & Authorisation of medicinal products for sale in UK
Devices
Quality control
Internet sales & counterfeiting (drugs coming to the UK that are made elsewhere)
Clinical Trials regulation
Statutory controls
Promotion of safe use
Manage British Pharmacopoeia & Clinical Practice Research Database
They will ask for a report of the first prescriptions of the drug to see how its been
Why is pharmacovigilance needed?
Rarer the side effects may not be identified in the RCT
So you do need post marketing surveillance of a drug to see how it is doing once it has came into the world
how do you report an ADR?
yellow card scheme
Where have all the yellow card reports came from?
Need to be looked at to see if they are truly related to the drug or if they are part of a wider sickness profile of the patient
how does the yellow card shceme work?
How do you know when a signal is significant?
•Another role is monitoring drug ___________, ________ of the drug
Careful regulation of where did the drug ____ ____, what is it, and does it do what it _____ on the tin
production
standard
come from
says
what is the European Medicines Agency? and what is their role?
European equivalent of FDA (food & drug administration in the US)
Ensure quality, safety and efficacy of drug
Marketing Authorisation (MA) or License Valid for whole of EU
Committee for Medicinal Products for Human Use (CHMP):
- Quality
- Safety
- Efficacy
Once drugs are on the ______, companies can ______ whatever the market will ____; prices for cancer therapies now routinely top $100,000 a year.
market
charge
bear
So who decides whether a drug can be used?
(In the US, insurance companies)
Scottish Medicines Consortium (SMC)
what is the role of the Scottish Medicines Consortium (SMC)?
Principal remit is to make decisions on the cost effectiveness of new/existing pharmaceutical products in respect of their use in NHS Scotland
They will all get together and discuss and look at the evidence for the specific drug and decide if it should be available for the general population in Scotland
who makes up the Scottish Medicines Consortium (SMC)?
Representation from all Area Drugs and Therapeutics Committees:
- Pharmacists
- Health economists
- Pharma representatives
- Lay representatives
- Physicians
- NHS management
whata re the good things about the SMC?
comprehensive assessment (of new drugs)
rapid response
uniformity within Scotland
minimise “post code prescribing”
education
what are the threats of the SMC?
pharmaceutical freedom
funding
what do SMC do with submissions?
3 month assessment process at time of launch
Submission by manufacturer
Health economic analysis
Cost per QALY (quality adjusted life years) central
Attempt to be fair and reasonable
Problem with very expensive drugs – Political overtones
what are the 3 different outcomes from the SMC?
- Approved for use
- Approved for restricted use
- Not recommended
why may a drug by not recommened by the SMC?
- Clinical effectiveness not convincing
- Cost-effectiveness not demonstrated
- No submission by manufacturer
- Phamasuticals are allowed to:
- Resubmission
- Appeal
- Individual Patient Treatment Requests (IPTRs) – if a particular consultant wants a drug for a patient
Cycstic fibrosis:
Life-threatening, Childhood onset, Caused by mutation in cystic fibrosis transmembrane regulator (CFTR)
G551D-CFTR occurs in ~12% cases in Scotland
Ivacaftor selectively potentiates CFTR in those with G551D mutation
Two key studies to date - Aged 6-11 years and 12+
Both showed 10% improvement in FEV1
Some improvement in respiratory symptoms and weight
Not clear how/if this translates into survival
Substantial extrapolation
“Not recommended”
Weakness of economic case
Balance of cost/benefit unfavourable
veyr expensive
“Not recommended” advice from SMC
Huge publicity in National Press
Politically unacceptable
Scottish Government overruled SMC by setting up an “Rare Conditions Medicines Fund” (RCMF) to facilitate provision of ivacaftor to CF patients with G551D-CFTR mutation (£25M)
Review of SMC processes
Enhancing access to new medicines:
what is an end of life medicine?
“A medicine used to treat a condition at a stage
that usually leads to death within 3 years with
currently available treatments.”
Enhancing access to new medicines:
what are orphan and ulta-orphan medicines?
Orphan medicines - “A medicine with EMA designated orphan status (ie conditions affecting fewer than 2,500 people in a population of 5 million) or a medicine used to treat an equivalent size of population irrespective of whether It has designated orphan status.”
Ultra-orphan medicines - “A medicine used to treat a condition with a prevalence of 1 in 50,000 or less (or around 100 people in Scotland.”
what are the challanges of enhancing access to new medicine
Company engagement – expected to improve
Current QALY “threshold” may already be over generous
(£13,000 per QALY may be more appropriate)
Incentive to propose PAS may be reduced
Net budget impact in year 1 may be £70M
Equivalent changes in England & Wales may have inflated drugs budget by an estimated 7.5%
Area Drugs & Therapeutics Committees - what is their role?
Response to SMC appraisal
“Approved for use” - Expected to approve for local use, unless equally effective alternative available locally. (“Me too” scenario). Local preference taken into account. Cost may be a consideration
“Not recommended” - Default position is not to approve for local use, IPTR may apply
(individual patient treatment requests)
Why local Formularies?
- Local ownership of decisions
- (but much common ground in Scotland)
- familiarity with limited range
- effectiveness
- value
- primary/secondary care issues
- new technology - intranet/websites, hyperlinks to guidelines
- electronic prescribing - GPASS, EMIS, Vision etc
what is the role of ADTCs?
- development of regional formularies
- implementation of SMC advice
- implementation of NICE/QIS appraisals
- implementation of SIGN guidelines
- dealing with drugs not on SMC agenda
- dealing with unlicensed products
- rationing new and expensive drugs
- regional “shared care/interface” issues
- education & communication
- prescribing errors
- “out of line” prescribers
- antimicrobial policies
- Patient Group Directives
disinvestment - Recommend removal from Grampian Joint Formulary
why may this happen?
[a] evidence for superior efficacy/safety/kinetics now available for alternative product
[b] evidence from major outcome studies now favour an alternative product
[c] evidence of superior cost/benefit now available for an alternative product
[d] evidence that this product is of limited clinical effectiveness
