The number of drugs picking up a designation from regulators — first treatments or speciality drugs — across the European Union, the US, and Japan have risen by 117 percent in 10 years.
This is mostly due to the rising numbers of new disease areas and increasing population, demanding the life sciences industry to perform faster.
However, would we be up to speed with drug research, development and innovation without the initiatives and funding of programmes for niche disease areas?
Organisations such as the International Rare Diseases Research Consortium (IRDiRC) have achieved their 2020 objectives, three years ahead of plan by delivering 222 new therapies for rare diseases in which each drug was either orphan designated or received marketing approval for the treatment of a rare disease.
Another programme such as the Innovative Medicines Initiative (IMI), Europe’s largest public-private initiative has been setup between the EU and the European Federation of Pharmaceutical Industries and Associations.
The IMI, backed by an estimated €5bn is involved in supporting the development of therapies in niche disease areas such as Alzheimer’s disease and Antimicrobial resistance.
Programmes like IRDiRC and the IMI are major drivers for increased collaboration between the public and private sectors to exchange data and change the way science is conducted, shared and applied within rare disease research.
By building bridges between drug developers and raising the bar for rare disease research, the industry will work fast to keep up with emerging disease areas and will facilitate the translation of science into clinical application.
Designations awarded by regulatory authorities are of major interest to drug developers due to incentives at stake and with the support of programmes such as IRDiRC and the IMI, we will continue to see increasing number of designations rewarded to niche drugs.