Horizon Scanning Why, how and what lies ahead?

1. Horizon ScanningWhy, how and what lies ahead? Produced to support the Prescribing Outlook series October 2012 Helen Davis, North West Medicines Information Centre

2. 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

3. 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: • treatments likely to become available to the NHS that may have significant implications for • clinical practice • service design • finance • potential disinvestments

4. Why horizon scan for medicines? Informs and primes providers and commissioners to proactively implement management strategies • 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

5. A woman with advanced kidney cancer and six months left to live says she is missing out on a potentially life-saving drug…….

6. Prescribing data (England) 2010: • NHS medicines expenditure £12.9 billion. • Hospital use accounted for 31.7% NHS Information Centre: Hospital Prescribing England

7. 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

8. Other factors that impact on management of drug budgets International markets Commissioning Value based pricing Innovation Health Wealth and MHRA consultation Personalised healthcare Patent expiries Orphan drugs/ indications Patent expiries Cancer drug fund Payment by Results (PbR) tariff HTAs (NICE, SMC, AWMSG) Patient access schemes Homecare

9. 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

10. UK Horizon scanning organisations Medicines and Prescribing centre Health & Social Care Northern Ireland

11. How does the UKMi horizon scanning process work? Filtration and selection Systematic early identification (horizon scanning) Information retrieval Prioritisation Dissemination Assessment

12. UKMi Horizon scanning products Prescribing Outlook www.nelm.nhs.uk Password restricted to NHS

13. 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

14. 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

15. Factors influencing impact Drug specific • 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)?

16. 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?

17. Factors UKMi use for prioritisation • 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.

18. 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

19. 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)

20. NDO content • 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

21. Other publications relevant to medicines budget planning • 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)

22. 2012/13Key pressures due to new medicines or licence extensions

23. 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 Neurology/rheumatology Alemtuzumab, laquinimod, dimethyl fumarate, teriflunomide – multiple sclerosis Tofacitinib – Rheumatoid arthritis Strontium ranelate - Osteoarthritis Respiratory Ivacaftor - cystic fibrosis Aclidinium, glycopyrrolate - COPD Pirfenidone - pulmonary fibrosis

24. Key new medicines due 2012/13 Chemotherapy Vismodegib - basal cell carcinoma. Pertuzumab- breast cancer Crizotinib - NSCLC Regorafenib -colorectal cancer Abiraterone, sipuleucel-T- prostate cancer Pazopanib, vintafolide– ovarian cancer Pixantrone - NHL • Miscellaneous • Insulin degludec -diabetes. • Ruxolitinib - myelofibrosis • Nalmefene - alcohol dependence • Eltrombopag – thrombocytopenia associated with hepatitis C.

25. UKMi Prescribing Outlook content 2010 Status of featured drugs April 2012

26. 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

27. What happens when managed entry is not planned?

28. 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 3,4-diaminopyridine base; cost about £1,000/year/patient • Amifampridine is phosphate salt formulation of 3,4-diaminopyridine; cost about £44,000/year/patient • Significant cost and supply pressures resulted

29. 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

30. 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

31. 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

32. What can clinical pharmacists do? • Understand NHS funding mechanisms and service issues • Highlight key developments to horizon scanners

33. Thank you Any questions?