This September, it will be discussed crucial topics around orphan drugs.
Far beyond the scientific studies, laboratory development of the drugs, the clinical trials, all the permissions needed from FDA, EMA and other drug regulators,before getting the drug in the market and the people needing it, get access to the drug, there is a very intricate and complex process of the drug market access.
lately, I´ve being deeply thinking about this and I´ve done research about this particular theme, reading about orphan drug regulation and access.
BMN-111 will reach this particular point when it will be ready to use by all babies and children with achondroplasia, but the pricing negotiations between countries and BioMarin for BMN-111 can drag all the process and may postpone the use of the drug for several months and sometimes, years.
And because of this, I´m confidante that this Summit will have a very relevant influence in this process.
At the official site of the Summit, you can read the topics of interest of the Orphan drug summit 2014:
• Market Access, pricing and reimbursement strategies
• Regulatory Framework in Europe, US and beyond
• International collaboration and alliances in Orphan Drugs development
• Scientific & Technological Clinical Development
• Patient Registries
Tagged: access, market, Orphan drugs summit 2014, patient
