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Table 4 Examples of performance-based or outcome-based models in Canada, Europe and US

From: Risk sharing arrangements for pharmaceuticals: potential considerations and recommendations for European payers

Country Examples
Canada Sandoz Canada promised to reimburse individuals, hospitals and government drug plans where patients with treatment-resistant schizophrenia discontinued clozapine within six months. This was initiated to address acquisition cost concerns versus typical anti-psychotics among the Provinces
Merck-Frost offered to reimburse provincial governments the full cost if patients prescribed finasteride subsequently required surgery for benign prostatic hyperplasia after one full year of medical therapy
Sanofi-Aventis agreed to reimburse the cost of docetaxel to provincial payers if an agreed responder level was not reached in patients with cancer due to concerns about its efficacy and costs. The programme lasted six months facilitating formulary listing
Denmark A population based 'no cure, no pay' strategy for valsartan to lower BP was initiated to enhance market share
Money back initiative for nicotine chewing gum if patients do not like the taste of any of the four flavours on offer
'No play; no pay' schemes for drugs for erectile dysfunction
Italy i) CRONOS scheme for Alzheimer drugs
Initially the acetyl cholinesterase inhibitors were 'C' classification in Italy, i.e. 100% co-payment
However, under the CRONOS scheme, companies initially provided acetyl cholinesterase inhibitors such as donepezil free of charge to specialist clinics for the first four months of treatment
The NHS subsequently covered the drug costs in responders, with patient outcomes recorded
This observational study, which demonstrated health gain in patients with mild to moderate AD, resulted in the NHS subsequently funding these drugs ('A' classification) provided patients were treated in specialist outpatients. However, there were no quality checks on the completed forms
ii) Ongoing registries to monitor prescribing and therapeutic value in practice
A number of registers have been initiated in Italy to monitor prescribing against licensed indications as well as monitor their therapeutic value in practice to guide future management and reimbursed prices
This includes a number of premium priced drugs such as cetuximab, ibritumonab tiuxetan, lenalidormibe, nelarabine, palifermin, temporfin and trastuzumumab for adjuvant use
Overall over 43,000 Italian patients have been included in the registries for new cancer medicines (up to Oct 2009)
UK - England and Wales i) Beta interferon for multiple sclerosis (MS)
NICE in its initial appraisal rejected funding for the β interferons in the treatment of MS on clinical and cost-effectiveness grounds with a calculated a cost/QALY of £42,000 to £98,000 over 20 years rising to a maximum of £780,000/QALY over 5-years
Following external pressure, the government in 2002 established a scheme with the four manufacturers whereby a cohort of approximately 10,000 patients would be followed for over 10 years with the cost of drugs reduced or refunds given if the cost/QALY over an envisaged 20-year horizon was over £35,000/QALY, i.e. fund a maximum value of £35,000/QALY or less
Patients would be followed using the Kurtzke Expanded Disability Status Scale (EDSS), which was the same outcome measure used in the trials
However, the scheme has been heavily criticised as unscientific and impractical
An initial assessment was published in 2009 reviewing patients who started treatment from May 2002 to April 2005. This highlighted important methodological issues with this study and the need for longer term follow-up before securing meaningful results
ii) Bortezomib for the treatment of first relapse of multiple myeloma
This scheme is based on a 50% reduction in serum paraprotein levels (M-protein) by the fourth cycle. The NHS will continue funding treatment in responders, with the cost/QALY reduced from £38,000/QALY to a more acceptable £20,700/QALY, with manufacturers refunding the cost of the drug if a 50% reduction was not achieved. This is usually in the form of free drug, which is seen as easier to implement
In addition, prices remain at the launch price despite up to a 60% discount in reality, which is important with the UK often used as a reference price country
However there have been concerns whether M-protein is a good surrogate for life expectancy. Alongside this, 10 to 15% of patients do not have measurable serum M-protein levels
iii) Atorvastatin for CHD prevention
The pharmaceutical company agreed to fund the health authority for wasted resources if atorvastatin failed to reduce LDL-C levels to agreed targets when patients have been properly titrated
No refunds were given in reality as all properly titrated patients reached target lipid levels helped by the recruitment of two nurses. The nurses worked with GPs and practice nurses aiding issues such as concordance, although a 20% adjustment was included in the outcome guarantee model
GP and patient participation was helped by CHD being a high priority disease area in the UK with national initiatives to improve care
However, there were problems with the scheme once generic simvastatin became available and lipid level targets were lowered in the UK
iv) Pharmaceutical Price Regulation Scheme (PPRS) proposals
Under the proposed flexible Value-Based Pricing (VBP) in the new PPRS scheme, pharmaceutical companies will be able to establish the initial launch price for their new products
Reimbursed prices will either fall or rise as new evidence of effectiveness and safety becomes available, or when new indications are added changing the overall value, following further appraisals by NICE
UK - Scotland Beta interferon for multiple sclerosis (as for England)
PPRS proposals apply to Scotland although the implementation of flexible pricing is still being discussed
US • Patients and insurers were refunded if simvastatin failed to lower LDL to target levels (up to 6 months)
• CIGNA agreed with pharmaceutical companies that they would reimburse the cost of treating a heart attack if this occurred whilst patients were being treated with lipid lowering drugs
• 'No cure, no pay' for valsartan and valsartan hydrochlorothiazide to patients and insures as part of a 'take action for healthy blood pressure' initiative. In addition a number of compliance enhancing initiatives were simultaneously launched by the company to help achieve BP target goals including the option to purchase a blood pressure monitoring device at significantly reduced costs
• Merck agreed to refund the cost of the drug for patients whose symptoms of benign prostatic hyperplasia did not improve within six months or who required surgery within two years assessed based on pre-determined criteria
Others (no particular country) • 'No baby - no pay' option for fertility treatments funded through national health services