Evolving commercialisation opportunities in rare diseases

Despite high approval rates of orphan drugs by the EMA, with the pressure of rising prices we are seeing increased payer sensitivity resulting in a reduced opportunity for reimbursement in the typically targeted first wave markets. In the UK for example, 17% of orphan drugs receiving EMA approval were not selected to undergo an appraisal and can only be reimbursed subject to individual funding requests. Further to this, Germany, who is considered the EU market leader when it comes to orphan drug access, has made recent changes to its AMNOG assessment process which may result in limited patient access and an increase in time-limited decisions. Given the reduced opportunity for achieving pricing and market access in the usual first-wave markets, we have assessed the current and mid-term (5-year) commercialisation opportunity for orphan drugs in 19 common second- or later-wave markets.