Correspondence |
Orphan drugs revisited: author's response
Sir,McCabe and colleagues1 discuss what they consider to be a range of inaccuracies in our article on the funding of drugs for exceptionally rare diseases.2 However, we may in fact be singing from a similar hymn sheet.
Where we differ from McCabe et al.13 is in offering practical solutions. Their strict efficiency stance ignores a range of other issues that appraisal committees consider. The fact that expensive, and cost-ineffective, ultra-orphan drugs have been approved by appraisal committees in some countries suggests that economic criteria alone are not sufficient for decision-making. In our section on options for policy recommendations, we list five possible mechanisms that may be considered by EU member states. We do not endorse any specific recommendation; rather we conclude that each EU country would need to develop their own policy guidelines.
McCabe et al.1 imply that we support the frequently cited arguments for special status consideration. However, this is not the case. We provide a balanced discussion of views that are commonly expressed, both in favour of and against granting special status for ultra-orphan drugs. We do not claim, for instance, that ultra-orphan drugs should be made available on the basis that no other treatment exists. Likewise, we do not propose the ‘rule of rescue’ as a basis for the special status for ultra-orphan drugs.
With regards to methodological issues concerning evidence on effectiveness, we highlight that there are difficulties in recruiting adequate sample sizes for clinical trials. We go on to suggest how improvements in post-marketing studies, and development of national or international registries that allow long-term follow-up on safety and effectiveness would go part way to address these issues. McCabe et al.1 state that the opportunity to collect information on the natural history of a disease is significantly reduced once a disease-modifying therapy becomes available. They reiterate the need for such registries in reference to our sections on risk sharing schemes, and the use of clinical and pharmacogenetic criteria to target potential responders. Without a doubt, data on the natural history of any disease, and rare diseases in particular, are enormously valuable. However, one needs to consider how practical and ethical this may be, once a treatment has been granted a licence. How rare does a disease have to be before the uncertainty around natural disease progression is adequately reduced?
The section on equity weighting, a way of incorporating social preferences into the QALY framework, uses a hypothetical scenario where society would value health gains in patients with rare diseases higher than in patients with more prevalent conditions. More research is required to establish whether this is the case.4
We support McCabe et al.1 in their discussion on pharmaceutical pricing. In Belgium, Finland, France and Spain, the authorities link the acceptable price to the therapeutic value of the drug.5 Health economic studies and classifications of therapeutic value are used as tools when assessing reasonableness of price. The case for integrating pricing and reimbursement has also been put forward in the UK.6
In conclusion, the decision to finance expensive treatments for rare diseases from public funds should be transparent, and based on principles of equity as well as clinical and cost-effectiveness. We share the sentiments expressed by McCabe et al.1 that the extension of the privileged regulatory provision for rare diseases to the reimbursement arena should not go unchallenged.
Centre for Economics and Policy in Health Institute of Medical and Social Care Research University of Wales Bangor
email: d.a.hughes{at}bangor.ac.uk
References
1. McCabe C, Tsuchiya A, Claxton K, Raftery J. Orphan drugs revisited. Q J Med 2006; 99:341–46.[Web of Science]
2. Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? Q J Med 2005; 98:829–36.[Web of Science]
3. McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? Br Med J 2005; 331:1016–19.
4. Hughes DA. Rationing of drugs for rare diseases. Pharmacoeconomics 2006; in press.
5. Martikainen J, Kivi I, Linnosmaa I. European prices of newly launched reimbursable pharmaceuticals-a pilot study. Health Policy 2005; 74:235–46.[Medline]
6. Walley T, Earl-Slater A, Haycox A, Bagust A. An integrated national pharmaceutical policy for the United Kingdom? Br Med J 2000; 321:1523–6.
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