PROCUREMENT & DISTRIBUTION INTEREST

GROUP

Autumn Symposium 2007

http://www.pdig.org.uk/

Pharma produce innovative products

Patients can’t access them!!

Is this true?

Professor Liz Kay, Clinical Director Medicines Management and Pharmacy Services

Leeds Teaching Hospitals NHS Trust

Overview

• Key stages in transition from trial to licence

• Compassionate use – what is this?

• Access to new medicines

• National systems delays

• Risk sharing

• What next?

Transition from trial to licence

• Key issue – separate ‘clinical trial’ and ‘unlicensed use’

• What is a clinical trial?

Research study, human volunteers, specific health question to answer

Phase I 20 to 80 people

Phase II 100 to 300 people

Phase III 100 to 1000s of people

Phase IV post licence

Clinical trials and LTH

• LTH Clinical trial policy

• REC – protect participants

• R+D check process

• Medicines Management and Pharmacy

• ‘Authorisation to proceed’ step = letter from Chief Pharmacist.

• All medicines managed by Pharmacy

LTH MMPS Authorisation stepWhat is this?

• Documentation in place

• Local risk assessment and procedures

• Operationally feasible

• Products manufactured in accordance GMP

• Written agreement about responsibilities with investigator

• Payment

New Product Review

Product Request Clinical Trial R&D Process

Outside Trial

Patient?

PbR

Unlicensed Risk Assessment

DTC

PbRRisk

AssessmentDTC

Patient?Licensed

Unlicensed

JANE 'LET DOWN OVER CANCER DRUG' 05/10/2007 14:17 (Daily Express +0) Mike Tomlinson, from Leeds, hit out after revealing that his wife had to travel to Nottingham to receive the cancer drug Lapatinib.

• Mr Tomlinson detailed how she battled to get access to Lapatinib. He said her medical team in Leeds decided the drug was her best option. However, Leeds Teaching Hospitals NHS Trust had taken the decision not to participate in a GlaxoSmithKline-sponsored access study of the treatment.

Medicines without marketing authorisation

• LTH Trust policy

• Management of policy via DTC

• Assessment of risk

• Patient consent

• Code of business conduct

• Involvement of commissioners

Unlicensed medicines policy

Element of assurance Required about the product

Licensed product (MA)

Used for licensed use

Licensed product Unlicensed use

Unlicensed product (imported or manufactured)

Responsibility for assessment

QUALITY Assured by licence

Assured by licence

No automatic assurance

Pharmacist/Purchaser

SAFETY Summary of Product

Characteristics (SPC)

No automatic assurance

No automatic assurance

Clinician/Pharmacist

EFFICACY Summary of Product

Characteristics (SPC)

No automatic assurance

No automatic assurance

Clinician/Pharmacist

Unlicensed medicines policy risk matrix

The Risk Matrix

Therapeutic Pharmaceutical

Evidence base “good” for efficacy, safety/toxicity, use, and patient status: Low Risk potential

Evidence base “moderate” for efficacy, safety/toxicity, use, and patient status: Moderate risk potential

Evidence base “poor” for efficacy, safety/toxicity, use, and patient status: High/Unknown Risk potential

Quality can be assured: Low Risk Potential

Green Yellow Red

Quality cannot be assured: High Risk Potential

Red Red Red

Relative Risk (green) LOW

Relative risk (yellow)

MODERATE

Relative risk (red) HIGH

Unlicensed medicines policy8.3 The table below summarises the minimum action required once an unlicensed medicines use has been assessed and categorised into one of the three

relative risk categories:-

Action Assessment Low relative risk potential Moderate relative risk potential High or Unknown relative risk potential

How is the new product reviewed prior to introduction to LTHT?

Requests for any new product are reviewed by LTHT D&T Committee.

Review of all unlicensed uses by LTH D&TC required. Unlicensed medicines submission required.

Review of all unlicensed uses by LTH D&TC.

What consent arrangements need to be established?

Obtain consent prior to treatment from patients or carers by discussing medicine choice as in normal practice. Additional arrangements should not normally be required.

Ensure as a minimum a record is made of a consent process in the individual patients medical notes with clear documentation of issues covered.

Formal written evidence of patient consent required.

Patient information: What written patient information is to be used?

Use generic unlicensed medicines information for outpatient or discharge medicines or specialist area patient information e.g. RCPCH and Pain/Palliative Care Patient Information Leaflets (PIL).

Use generic unlicensed medicines patient information or Patient group or unlicensed use specific information prepared for the purpose.

Product specific unlicensed medicines patient information will be required and will be reviewed by D&TC.

Will the product require specific labelling to highlight it unlicensed status?

Many products in this category will be licenced products being used for unlicensed use. In addition to standard requirements all products without a UK Marketing Authorisation will be labelled with the code “ULM”.

In addition to standard requirements all products without a UK Marketing Authorisation will be labelled with the code “ULM”.

In addition to standard requirements all products without a UK Marketing Authorisation will be labelled with the code “ULM”.

What product use records are legally required and how will these be managed?

Normal records of products issued will be retained by the Pharmacy Management System (PMS) at each dispensing episode.

Pharmacy will ensure a record is maintained of Prescribing Consultant, patients name and batch number details in addition to the PMS record of issues

Pharmacy will ensure record is maintained of, Prescribing Consultant, patients names and batch number details in addition to the PMS record of issues

See handout 1

EAPsDiscourage use of investigational agents outside clinical trials

ISSUESManagement of clinical risk‘Unlicensed medicine’Lose potentially valuable dataManagement of costs and financial risksSubstantial charges (‘admin and supply’!!)Equity of access for patientsPharma ‘bypass of process’ for managing entry of new drugs

National approval systems

• NICE and SMC

• Different terms of reference – timelines, Political issues??????????

• LTH approach

• Medicines Finance Committee and use of Health Economist

• Commissioning Group for Medicines, Yorkshire & The Humber (North)

What effect the PbR tariff?

• LTH £ 32+million spend (50%) non tariff

• Non tariff NICE approved – PCT pay to agreed volumes

• Non Tariff Non NICE (NTNN), individual patient PCT approval – postcode lottery, LTH 440+ in 2007

DoH view on Risk Sharing Schemes

• Not against such schemes in principle• Any schemes should be operationally workable for the

NHS (not lead to disproportionate costs or bureaucracy). • Velcade - content on this point and for NICE to appraise

the scheme.• Responder schemes - important that response can be

measured and in the case of Velcade, NICE agreed with the company's method for measuring response.

• Cash/credit or free stock – no strong view but vital that scheme is not too burdensome on the NHS.

NICE guidance on Bortezomib

Velcade Response Scheme

Janssen Cilag: Drug costs removed for ‘non responders’

NICE: Response based on serum M protein after 4 cycles

Rebate: ‘Patients responded less than partially’

ICER of £20,700 per QALY

Additional cost to NHS to administer – not funded.

Process for rebate: Form to Fax

Replace stock, credit note, cash

Myeloma matters – impact on NHS

• Bortezomib licence NICE approval planned use

• Lenalidomide (better than thalidomide)

Licensed June 2007

No NICE approval

Planned use

Cost £50K pa

NTNN – postcode lottery

Doesn’t reflect use

More responders thanplanned

Less rebates than DoH expected

Issues for industry

• Ready to use product – minimise waste (infliximab)

• Appropriate vial size – minimise waste (infliximab)

• Packed with risk management in consideration• Realistic costs – global pricing• Free stock or BOGOF – pharmacy computer

schemes• No more rebate schemes

Where next?

• Clarity about medicines used without or outside marketing authorisation and peer support for their use outside clinical trials

• Systems to ‘fast track’ to marketing authorisation for major advances

• Systems for ‘national approval’ faster than NICE• Commissioning on wide geographical scale to

ensure equity of access for patients