There had been increased and strong public interests in rare diseases and orphan drugs as well as the issue of
compulsory licencing for expensive medications in Malaysia in the mass-media and social media. We reviewed
the issues of orphan drugs and the challenges faced in many countries in developing appropriate health financial
modelling as well as getting accurate data on rare diseases. We also reviewed the old off-patent medications
and the developments on how policy-makers can intervene to make expensive treatment affordable and
sustainable for patients and the country.