Abstract
Background: The debate on access to new drugs has focused on the time lag between applications for approval and granting of marketing authorisation. This delay was identified as the first barrier with respect to patient access to new drugs, encompassing the hurdles of safety, efficacy and quality. Additional barriers have since been identified. These pertain to reimbursement and pricing of approved drugs, the so-called fourth and fifth hurdles.
Methods: We reviewed 38 National Institute for Health and Clinical Excellence (NICE) guidance appraisals carried out between April 1999 and April 2005. These appraisals included 71 recently approved drugs considered to have either high clinical or cost impact. For each drug we first determined its marketing approval date by the British Medicines Healthcare Products Agency (MHRA) or European Medicines Evaluation Agency (EMEA). Secondly, we determined if each drug was approved by the US FDA for marketing and, if so, the date when it was approved. Thirdly, we considered whether and when each drug was recommended for reimbursement and use by NICE, and whether conditions of reimbursement applied. Fourthly, for the subset of FDA-approved drugs, we examined formulary placement, cost sharing and conditions of reimbursement on three-tier formularies used by seven leading US third-party payers serving Medicare beneficiaries. Fifthly, we reviewed each NICE recommendation to determine if cost-effectiveness data were referred to either in the appraisal documentation or in the final recommendation. Sixthly, we asked a spokesperson from each US payer whether cost-effectiveness assessments or rebates played a role in determining formulary placement of drugs in our sample, and whether there was a lag between marketing approval and reimbursement for any of the covered drugs.
Results: Of the 71 drugs contained in 38 NICE guidance appraisals, the US FDA approved 64. On average, the subset of 64 drugs received marketing authorisation in the US prior to the UK. On average, US plans covered 87% of the 64 drugs, the same percentage of drugs recommended for NHS reimbursement and use. Cost sharing in the US was significantly higher than in the UK, with wider variation across plans. On average, drugs covered in the US had fewer conditions of reimbursement (15%) than the percentage of drugs given conditions by NICE (46%). US plans were quicker to decide to reimburse drugs following marketing approval than NICE.
Conclusions: The US provides faster, more flexible access to most, but not all, of the UK-approved pharmaceuticals in our sample. However, US patients have higher cost sharing than the UK and coverage is less evenly spread across the population. From a policy perspective, our study findings confirm the need to bolster the NICE fast-track initiative to decrease the amount of time it takes to appraise certain new pharmaceuticals. Also, the study findings point to the need in the US for careful monitoring of plan compliance with regulations pertaining to the Medicare drug benefit, particularly with respect to formulary restrictions and limits on cost sharing.
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Notes
1Note that there is often a delay between licensing and the actual market launch of a drug. For simplicity, we ignore this delay.
2Step therapy indicates that a drug is a second- or third-line option, or that a drug is recommended as an alternative for patients who are intolerant, have contraindications, are unable to comply or have an unsatisfactory response to existing treatment options.
3Prior authorisation is a procedure that requires obtaining permission from the payer to prescribe a medication prior to prescribing it.
4For our analysis we used SAS version 9.1.
References
PDUFA, FDA and stakeholders public meeting [online]. Available from URL: http://www.fda.gov/ohrms/dockets/05n0410/05n-0410-tr0001-vol2.pdf [Accessed 2006 Mar 24]
Towse A. The efficient use of pharmaceuticals: does Europe have lessons for the Medicare drug benefit. Health Aff (Millwood) 2003; 22(3): 42–5
Civitas. Are the NHS reforms working? NHS: is the extra money working? 2003 [online]. Available from URL: http:// www.civitas.org.uk/nhs/nhsMoney.php [Accessed 2005 Apr 2]
Laschober MA, Kitchman M, Neuman P, et al. Trends in Medicare supplemental insurance and prescription drug coverage, 1996–1999. Health Aff Web Exclusive 2002 Feb 27; 2002 [online]. Available from URL: http://content.healthaffairs.org/Cgi/reprint/hlthaff.w2.127vl?.maxtoshow = &HITS = 10&hits = 10& [Accessed 2005 Feb 27]
Medicare prescription drug benefit. Kaiser Family Foundation. March, 2005 [online]. Available from URL: http://www.kff.org/medicare/upload/The-Medicare-Prescription-Drug-Benefit-Fact-Sheetpdf [Accessed 2005 Apr 2]
General principles: FDA-EMEA parallel scientific advice meetings [online]. Available from URL: http://www.emea.eu.int/pdfs/general/direct/internationalcoop/emea-fdascientificad-vice.pdf [Accessed 2006 May 11]
Dickson M, J Hurst, S Jacobzone. Survey of pharmacoeconomic assessment activity in eleven countries. OECD working paper [online]. Available from URL: http://www.oecd.org/dataoecd/27/25/2955828.pdf [Accessed 2006 May 10]
Barbour V. Imatinib for chronic myeloid leukaemia: a NICE mess. Lancet 2001 Nov 3; 358(9292): 1478
UK insurers ‘pay only for NICE-backed drugs’. Pharma Marketletter 2005 Oct 17; 32(41): 11
Towse A, Pritchard C. National Institute for Clinical Excellence (NICE): is economic appraisal working? Pharmacoeconomics 2002; 20 Suppl. 3: 95–105
Devlin N, Parkin D. Does NICE have a cost-effectiveness threshold and what other factors influence its decisions? A binary choice analysis. Health Econ 2004 May; 13(5): 437–52
Harris A, Buxton M, O’Brien B, et al. Using economic evidence in reimbursement decisions for health technologies: experience of 4 countries. Expert Rev Pharmacoeconomics Outcomes Res 2001 Jul; 1(1): 7–12
Buxton M. Implications of the appraisal function of the National Institute for Clinical Excellence (NICE). Value Health 2001 May-Jun; 4(3): 212–6
House of Commons Health Committee: NHS charges — third report of session 2005–06 [online]. Available from URL: http://www.publications.parliament.uk/pa/cm200506/cmselect/cmhealth/815/815ii.pdf [Accessed 2006 May 11]
Syrett K. A technocratic fix to the ‘legitimacy problem’? The Blair Government and health care rationing in the United Kingdom. J Health Polit Policy Law 2003; 28: 715–46
Cohen J. PBMs and a Medicare drug benefit. Food Drug Law J 2000; 55: 311–20
Kreling D. Cost control for prescription drug programs: pharmacy benefit manager (PBM) efforts, effects, and implications [online]. Available from URL: http://aspe.hhs.gov/health/reports/drug-papers/kreling-final.htm [Accessed 2006 May 5]
Academy of Managed Care Pharmacy. The AMCP format for formulary submissions. Version 2.1, April 2005. A format for submission of clinical and economic data in support of formulary consideration by health care systems in the United States [online]. Available from URL: http://www.209.35.33.5/data/resource/FormatVersion_2_lFinal_Final.pdf [Accessed 2005 May 5]
Fox DM. Evidence of evidence-based health policy: the politics of systematic reviews in coverage decisions. Health Aff (Millwood) 2005 Jan-Feb; 24(1): 114–22
Blue Cross Blue Shield technology evaluation center [online]. Available from URL: http://www.bcbs.com/tec/index.html [Accessed 2005 Apr 13]
Procedure for producing guidance documents describing Medicare’s coverage process. Fed Reg 2004 Sep 24; 69(185): 57325–6 [online]. Available from URL: http://www.cms.hhs.gov/coverage [Accessed 2005 May 19]
Drug Effectiveness Review Project. Center for Evidence Based Policy. Oregon Health & Science University [online]. Available from URL: http://www.ohsu.edu/drugeffectiveness/reports/final.cfm [Accessed 2005 Apr 12]
Drug class reviews. Strategic Healthcare Group. Pharmaceutical benefits management. U.S. Department of Veterans Affairs [online]. Available from URL: http://www.vapbm.org/pbm/reviews.htm [Accessed 2005 Apr 21]
NICE home page [online]. Available from URL: http://www.nice.org.uk [Accessed 2005 Apr 4]
Find a Medicare prescription drug plan [online]. Available from URL: http://www.medicare.gov [Accessed 2006 Feb 2]
PBM National Formulary [online]. Available from URL: http://wwwl.va.gov/health [Accessed 2006 Jan 31]
Drug coverage and pricing [online]. Available from URL: https://www.advancerx.com [Accessed 2006 Feb 3]
Hoadley J, Hargrave E, Cubanski J, et al. An in-depth examination of formularies and other features of Medicare drug plans [online]. Available from URL: http://www.kff.org/medicare/7489.cfm [Accessed 2006 May 11]
April 2005 payment allowance limits for Medicare Part B drug [online]. Available from URL: http://www.wpsic.com/medicare/provider/pdfs/0405drug_allow.pdf [Accessed 2005 May 5]
FDA home page [online]. Available from URL: http://www.fda.org [Accessed 2005 Jun 21]
Center for Medicare and Medicaid Services. Overview [online]. Available from URL: http://www.cms.hhs.gov/HisfPartB-DrugPricingFiles/01_overview.asp [Accessed 2006 Apr 10]
Niles S. Medicare market makeover. Med Ad News 2005 May; 24(5): 4, 54-6
Fox P. Prescription drug benefits: cost management issues for Medicare [online]. Available from URL: http://www.cms.hhs.gov/apps/review/03_04winter/03_04winterpg7.pdf [Accessed 2006 Apr 5]
US Medicare drug plans must expedite formulary review of new medicines in 2007. www.scripnews.com. Scrip 2006 Apr 7; 3146: 14
Sheldon T, Cullum N, Dawson D, et al. What’s the evidence that NICE guidance has been implemented? Results from a national evaluation using time series analysis, audit of patient’s notes, and interviews. BMJ 2004; 329: 999–1007
National Institute for Health and Clinical Excellence. Final appraisal determination: donezepil, galantamine, rivastigmine and memantine for the treatment of Alzheimer’s disease [online]. Available from URL: http://www.nice.org.uk [Accessed 2006 Aug 24]
US Pharmacopeia Convention guidelines for formulary development [online]. Available from URL: http://www.usp.org [Accessed 2006 May 11]
CancerBACKUP. CancerBACKUP ‘Dossier of delay’ reveals cancer patients waiting for potential life-saving cancer treatment. 2005 May 26 [online]. Available from URL: http://www.cancerbackup.org.uk/News/Press/Pressreleasesstatements/2005/84975716 [Accessed 2005 May 19]
PhRMA. What goes into the cost of prescription drugs? [online]. Available from URL: http://www.phrma.org/files/Cost_of_Prescription_Drugs.pdf [Accessed 2006 May 11]
Acknowledgements
We are grateful to Mark Goddard (Medicines and Healthcare Products Regulatory Agency) for providing us with UK marketing authorisation dates for all 71 drugs in our sample.
No sources of funding were used to assist in the preparation of this study. The authors have no conflicts of interest that are directly relevant to the content of this study.
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Cohen, J., Cairns, C., Paquette, C. et al. Comparing Patient Access to Pharmaceuticals in the UK and US. Appl Health Econ Health Policy 5, 177–187 (2006). https://doi.org/10.2165/00148365-200605030-00004
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DOI: https://doi.org/10.2165/00148365-200605030-00004