Transcript Horizon Scanning Why, how, what

Horizon Scanning
Why, how and what lies
ahead?
Key learning points
• Why is horizon scanning for medicines important
• How is horizon scanning undertaken
• What information is available to support the
managed entry of new drugs and its limitations
• What key therapeutic advances are expected in the
next year
What is horizon scanning?
Horizon Scanning has been defined as:
‘the systematic examination of potential threats,
opportunities and likely future developments…….'
Horizon scanning for medicines aims to identify:
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treatments likely to become available to the NHS
that may have significant implications for
– clinical practice
– service design
– finance
• potential disinvestments
Why horizon scan for medicines?
Informs and primes providers and commissioners
to proactively implement management strategies
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Anticipate pressures (financial and service delivery)
Manage budgets
Plan services - new and redesign
Identify areas for disinvestment
Manage entry into hospital/ formulary /practice etc
Identify drugs suitable for homecare
A woman with
advanced kidney
cancer and six months
left to live says she is
missing out on a
potentially life-saving
drug…….
Prescribing data (England)
2010:
• NHS medicines
expenditure £12.9
billion.
• Hospital use
accounted for 31.7%
NHS Information Centre: Hospital Prescribing England
Drivers of growth in prescribing
• New drugs for diseases where previous options were
limited e.g. rare genetic diseases
• Expanded indications (increase in eligible population)
e.g. chemotherapy drugs
• New drug regimens or maintenance treatments added
to standard therapy e.g. chemotherapy, antidiabetes
• Displacement of old drugs with new drugs at higher
cost e.g. “biologicals”, oral anticoagulants
• ‘Medicalisation’ e.g. social anxiety
• Ageing population
Other factors that impact on
management of drug budgets
Commissioning
Innovation
Health Wealth
and MHRA
consultation
HTAs (NICE,
SMC,
AWMSG)
International
markets
Value based
pricing
Personalised
healthcare
Patent
Patent
expiries
expiries
Payment by
Results
(PbR) tariff
Orphan
drugs/
indications
Patient
access
schemes
Cancer
drug fund
Homecare
Information sources used by
horizon scanners
• Specialist media for press
releases highlighting
– conference presentations
– dates for submission to
licensing authorities
– plans for development
• Specialist websites,
databases and journals
– UK PharmaScan
• Industry (contacts, websites,
annual reports)
• Licensing agencies
• Clinical trial registries
• Clinical specialists
• Other horizon scanners
UK Horizon scanning organisations
Health & Social Care
Northern Ireland
Medicines and
Prescribing centre
How does the UKMi horizon
scanning process work?
Systematic early
identification
(horizon
scanning)
Filtration and
selection
Information
retrieval
Prioritisation
Assessment
Dissemination
UKMi Horizon scanning products
Prescribing Outlook
www.nelm.nhs.uk
Password restricted to NHS
Horizon scanning challenges
• Licence extensions/ new formulations are
difficult to track and time frames for approval are
shorter
• Company acquisitions vs. in-house R&D (biotech)
• Company mergers
• Confidentiality issues
• Epidemiology or target population may be
difficult to define and quantify
Horizon scanning challenges
• Regulatory delays
• Differences in views between licensing
authorities
• Regulatory transparency differences
• Indication applied for may not be the same as
that eventually approved
• Cost is rarely known prior to launch
• Rate and extent of market uptake is difficult to
forecast
Factors influencing impact
Drug specific
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Anticipated licence?
Formulation and administration?
First in class?
Place in therapy?
Significant improvement in disease management?
Other trials ongoing? (Licence extensions are easier
to obtain and there may be off label use.)
• Cost of drug, administration and testing
• What could be its USP (unique selling point)?
Factors influencing impact
External factors
• Size of target population i.e. large population or
significant subset of large population? What is large?
• Will it change where patients are treated e.g. hospital vs.
intermediate vs. home vs. primary care?
• Local use (in ongoing clinical trials or unlicensed use)?
• Funding of services? E.g National commissioning
• Where in NICE agenda?
• Which company?
• Media/public interest?
Factors UKMi use for prioritisation
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significant improvement in disease management?
additional therapy or displacement of existing therapies?
first in class or has a major new indication?
limited other drug/non-drug alternatives?
high cost?
service implications e.g. route/ formulation/ method of
delivery
the drug or disease area is considered an NHS priority
in the EU licensing process
significant additional indications in the advanced pipeline
stage
likely to be significant media interest.
Who is involved in UKMi
prioritisation?
• UKMi pharmacists with extensive horizon scanning
expertise
• Primary care/ commissioning pharmacists
• Secondary care/ interface pharmacists
• Other people/organisations with horizon scanning
expertise
UKMi Horizon scanning products
New Drugs Online (NDO) database
• Accessible via NHS Evidence (limited) and UKMi (full access
for NHS staff) websites
• Contains over 1300 active monographs
• Updated daily
• In September 2012
– 356 monographs updated
– 14 evidence based evaluations added
– 2,833 registered users
• Monthly newsletter sent to registered users (NHS only)
• Reporting facility (NHS only)
NDO content
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Name (generic, company, synonym)
Indication, formulation
Pharmacology, epidemiology
Key trial data
Stage in licensing process (EU, US), anticipated UK launch
date
• Orphan status
• Links to independent evaluations e.g. NHSC, LNDG
• In NICE pipeline
Other publications relevant to
medicines budget planning
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NICE/ SMC/ AWMSG guidance
London New Drugs Group reviews
UKMi Prescribing Outlook cost calculator
UKMi New Medicines Profiles
UKMi IFR summaries
UKMi NICE bites
MPC* Evidence summaries: new medicines
Forward Look (Scotland)
Regional advisory committees e.g. NETAG, MTRAC
* Medicines Prescribing Centre (formerly NPC – National Prescribing Centre)
2012/13
Key pressures due to
new medicines or licence
extensions
Key new medicines due 2012/13
Cardiovascular system
Apixaban, rivaroxaban, dabigatran
- stroke prevention, VTE treatment
and long term prevention
Defibrotide - hepatic veno-occlusive
disease
Lomitapide, mipomersen hypercholesterolaemia
Respiratory
Ivacaftor - cystic fibrosis
Aclidinium, glycopyrrolate - COPD
Pirfenidone - pulmonary fibrosis
Neurology/rheumatology
Alemtuzumab, laquinimod,
dimethyl fumarate,
teriflunomide – multiple
sclerosis
Tofacitinib – Rheumatoid
arthritis
Strontium ranelate Osteoarthritis
Key new medicines due 2012/13
Chemotherapy
Miscellaneous
Vismodegib - basal cell
carcinoma.
Insulin degludec - diabetes.
Pertuzumab - breast cancer
Nalmefene - alcohol dependence
Crizotinib - NSCLC
Eltrombopag –
thrombocytopenia associated
with hepatitis C.
Regorafenib - colorectal cancer
Abiraterone, sipuleucel-T prostate cancer
Pazopanib, vintafolide –
ovarian cancer
Pixantrone - NHL
Ruxolitinib - myelofibrosis
UKMi Prescribing Outlook content 2010
Status of featured drugs April 2012
Withdrawn/
discontinued
10%
Filed/PIII
13%
Launched/ approved/
positive opinion
77%
Reasons for delay
• Licensing process raises questions causing
– delay
– withdrawal from licensing process
– discontinuation
• Once licensed the company may not launch in the
UK at all
• Waiting for NICE appraisal/ reimbursement
negotiations
What happens when managed
entry is not planned?
Amifampridine (Firdapse)
• Licensed Feb 2010 for Lambert-Eaton myasthenic syndrome
(LEMS) - prevalence of about 1 per 100,000
• Licensed as orphan drug under exceptional circumstances
• LEMS previously treated with unlicensed 3,4diaminopyridine base; cost about £1,000/year/patient
• Amifampridine is phosphate salt formulation of 3,4diaminopyridine; cost about £44,000/year/patient
• Significant cost and supply pressures resulted
Firdapse ..why missed?
• Ultra orphan drug (v. small no. patients in few centres)
• LEMS already treated with comparatively cheap
(although unlicensed) drug
• Lack of background ‘noise’
– Exceptional circumstances status shortened the licensing
process (more difficult to track)
– Evidence required not the same for orphans as for other drugs
– Licensing company relatively small and bought in amifampridine
just prior to submitting for a licence
– No publicity about filing for a licence
• Price could not be anticipated
Firdapse learning points
• Be aware of currently unlicensed treatments
• Be more aware of orphan drugs (database
amended)
• EMA filing data now available
• Highlights the fact horizon scanning processes
cannot be comprehensive
What can clinical pharmacists do?
• Utilise horizon scanning resources
• Be aware of developments within your
speciality
• Liaise with key clinicians and budget holders to
raise awareness and ensure new medicines and
licence extensions are planned for
What can clinical pharmacists do?
• Understand NHS funding mechanisms and
service issues
• Highlight key developments to horizon scanners
Thank you
Any questions?