The Rarer Cancers Forum was disheartened to read these views and hopes this is not a common view in the NHS.
The forum welcomes the EU legislation on orphan medicines implemented in 2000, which has encouraged companies to research and develop treatments for rare conditions including cancers (which represent nearly 50 per cent of all cancers).
Orphan medicines are designated as such only if they are for severe, life-threatening or debilitating rare conditions. These medicines have to be supported by high standards of clinical and safety evidence, often-involving large prospective randomised trials, and in many cases they must provide evidence of cost-effectiveness.
In many of these cases, there are challenges in relation to comparators as no treatment was previously available. In addition to this, there is sometimes a requirement from regulators for companies to continue to collect information post-marketing. Yet another difficulty.
For conditions such as rarer cancers, we believe commissioning decisions for orphan medicines should not be made at an individual PCT level but agreed collectively at regional levels, such as by the recently formed specialist commissioning groups, due to the difficulties in planning for patient numbers presenting in small areas over time. Sensible planning would give patients access to the treatments they deserve irrespective of where they live and encourage the pharmaceutical industry to continue investing in developing medicines for the benefit of patients with such conditions.
Penny Wilson-Webb is chief executive of the Rarer Cancers Forum.
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