Improve Compassionate Use Programmes to ensure patients’ early access to medicines

EURORDIS-Rare Diseases Europe has published a new position on compassionate use, calling for the adoption of measures to revolutionise patients’ access to new medicines through Compassionate Use Programmes (CUP).

The length of time needed to develop a medicine varies, but on average it takes five to seven years, usually followed by the regulatory process and its legal timeframe, followed finally by pricing and reimbursement negotiations. Not all patients have the time to contemplate this relatively long process: their disease worsens, they gradually lose their body functions, and eventually die. In addition, at the same time as this inescapable deterioration is taking place, they can hear about “promising results” coming in the news, in real time. No other situation can be a source of greater despair in a patient’s life: dying and yet being aware a possible medicine is approaching the market.