WITH reference to Azrul Mohd Khalib’s letter on “Politics of rare diseases”, (The Star, Aug 29), as a former lecturer, I know that medical students are fascinated by rare diseases even though they may not encounter these diseases again as doctors.
Both under-graduate and post-graduate examinations tend to use rare presentations of common diseases or common presentations of rare diseases for their clinical cases. But as practising clinicians, the reality would set in.
Dr Theodore Woodward, a former professor at the University of Maryland School of Medicine, Baltimore, in the late 1940s first alluded to, “When you hear hoofbeats, think of horses, not zebras,” which means, “common diseases occur commonly.” It means that a doctor should look for the expected cause first, rather than the exotic. This is practical as more patients are likely to be helped if a doctor is well-versed in common diseases which affect a lot of patients.
Rare diseases are best left in the hands of tertiary centres with personnel who have a special interest in them. Funding for research, diagnosis, treatment and management of orphan diseases should therefore be diverted to these tertiary centres and not spread too thinly.
Orphan diseases are treated by orphan drugs, which are economically not viable to produce because of their small patient pool. Research and development (R&D) in the treatment of rare diseases is likely to be costly and not worthwhile financially for the pharmaceutical industry. There is another group of orphan drugs where the cost of R&D was borne by the original parent company. These are old off-patent drugs whose patient pools are again small. But should any pharmaceutical company acquire the exclusive manufacturing and marketing rights to these old, off-patent under-utilised but nevertheless essential drugs, they would have the monopoly and patients, albeit few, will be at their mercy.
A recent example is the acquisition by Turing Pharmaceuticals of the marketing right to pyrimethamine in August 2015. Pyrimethamine is used to treat malaria and toxoplasmosis. Its patient pool includes pregnant women, HIV/AIDS patients, post-organ transplant patients on immune-suppressants and patients on chemotherapy.
Within a month of the acquisition, Turing hiked the price per tablet of pyrimethamine from US$13.50 to US$750, an increase of 5500%.
It was in the market for more than 60 years and was off-patent since the 1970s. The small patient pool, the absence of a competitor and lack of alternatives, all contributed to an effective monopoly.
For a while, Turing’s then CEO Martin Shkreli was the most hated man on social media. This “drug profiteering” by Turing was further put in perspective when a group of Sydney Grammar School students produced 3.7g of pyrimethamine in the school laboratory for a paltry sum of US$20.
Keeping the costs of off-patent drugs low despite monopoly should be a social obligation of the pharmaceutical industry.
DR CHUAH SEONG YORK