Ahead of Rare Disease Day on Saturday, new figures released by the Irish Pharmaceutical Healthcare Association (IPHA), the industry body for the originator biopharmaceutical industry, show that, last year, patients waited an average of almost 1,000 days from EMA approval for access to new medicines for rare diseases.
Some of these diseases included spinal muscular atrophy, acute lymphoblastic leukaemia, neuroblastoma and metastatic merckel cell carcinoma.
This year, a further 13 medicines have been waiting almost an average of 1,000 days from EMA approval to be reimbursed and made available to patients. These medicines have completed the full pharmacoeconomic assessment and some have received reimbursement approval – but funding has not been made available by the Health Service Executive.
Rare conditions impacted include acute lymphoblastic leukaemia, short bowel syndrome and acute myeloid leukaemia.
In Europe, 30 million people live with a rare disease, or one in 17. Some 300,000 people in Ireland are affected by rare conditions during their lifetime.
The Orphan Medicinal Products Regulation, an important EU intellectual property law, has fostered investment and innovation in Europe, leading to a greater understanding of rare diseases and to new treatments. Since it came into effect in 2000, the number of orphan medicines increased from eight to more than 160 now.
“We need to ensure a stable and predictable regulatory environment that continues to support and inspire further research into new treatments for rare diseases,” said Oliver O’Connor, the Chief Executive of IPHA and a member of the Board of EFPIA, the industry body for European biopharmaceutical innovators.
“The potential of new treatments can only be realised if patients have access to them. In Ireland, access to new medicines remains a major blockage in the health services. We must improve how we manage the introduction of new medicines, including for rare diseases, in the health services.
“That’s the focus of the new Agreement between industry and the State: fixing the funding and access problem so that patients get the best treatments when they need them.
“The new Programme for Government, whatever parties agree it, should adopt an explicit policy on how to allocate funding to new medicines, relying on both the State and industry contributions,” said Mr O’Connor.
Rare diseases are among the hardest medical conditions to treat. They cause difficulties with daily activities, motor and sensorial functions and social interactions. There are between 7,000 and 8,000 recognised rare diseases.
Notes for Editors
In 2019, the following rare conditions waited an average of almost 1,000 days to access new medicines: Spinal Muscular Atrophy; Acute Lymphoblastic Leukaemia; previously untreated Advanced Folicular Lymphoma; Neuroblastoma; Metastatic Merckel Cell Carcinoma; Leber’s Hereditary Optic Neuropathy; Phenylketonuria; and the treatment of Duchenne Muscular Dystrophy.