Opinion: Orphan drugs - a rare dilemma

Published: 9-Feb-2015

Medicines for rare disease are a paradox, in that something rare and sought after is valuable, but only worth as much as someone is prepared to pay

It is an unavoidable reality that something rare will command a higher price than something that is readily available, even though both may be equally valuable to those who need them.

Healthcare providers face an unenviable task when they have to decide whether the high cost of a drug can be balanced against extending the life of a patient by no more than a few weeks, and invariably ‘greedy’ pharmaceutical companies are criticised for putting self-interest ahead of the needs of the sick and dying.

The gap between desirability and affordability is not an issue that will go away; on the contrary, it looks set to widen. As biologicals secure an increasing proportion of the market, the high cost of entry – in terms not only of capital expenditure but also of technologies and science skills – means that when the patent expires originators are likely to be faced with a gentle slope rather than the cliff that has overshadowed the small molecule market for the last decade.

The picture is further complicated by the growth in orphan drugs. Although there are numerous incentives to develop therapeutics to address unmet medical needs, the uncomfortable truth remains that to develop a product for which there is a very small market requires at least the same R&D effort as a mass-market product and the price must therefore be correspondingly higher to achieve any sort of return.

The weight of public opinion may in the past have persuaded governments to rethink their decision and allow healthcare providers to prescribe expensive medicines, but that public opinion will carry much less weight when the disease in question is virtually unknown and the patient population only a few thousand. Similarly, there will be less pressure on pharma companies to bring the price of these medicines down to what are deemed affordable levels, and the likely result will be stalemate.

Pharma has made major adjustments to the way it operates; perhaps the way the world pays for its healthcare should also be reviewed to take account of changes in technologies.

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