The G-BA had a hard time defining implementation steps for the legal requirement granting orphan drugs a positive additional benefit, as long as the value of sales at Statutory Health Insurance expense stays below €50 million per 12 months. After two unconsidered IQWiG assessments, the G-BA finally adjusted its procedure. For the third orphan drug undergoing AMNOG, IQWiG was only commissioned for the number of eligible patients and the costs of treatment. The G-BA decided to consider for its decision on additional benefit the advice of its impartial Office instead.
It took several years to get the specific G-BA orphan drug benefit assessment established in Germany. Today I still feel honoured experiencing this incredible journey with ups and downs. However, I left the G-BA with apprehension.
I follow changes regarding orphan drugs with mixed feelings. Will the legislation further cut incentives for orphan drugs? Will the G-BA devalue the benefit assessment of orphan drugs in Germany? Will IQWiG take the power back in the name of its General Methods?
Not to mention the EU joint clinical assessment. Can one worry too much? Hopefully biopharma will still find the courage to invest in research and development for good medicines that will reach patients.
• You can access Rare Disease Day’s official website here.
Meriem Bouslouk-Marx’s support for Rare Disease Day 2023