FDA and NIH Announce Collaboration With C-Path to Advance Treatment of Rare Neurodegenerative Conditions
The US Food and Drug Administration (FDA) and the National Institute of Health (NIH) have announced they are partnering with the Critical Path Institute (C-Path) to promote research into rare neurodegenerative conditions. The agencies will work with the non-profit organization to advance individualized therapeutic development and collect a central repository of research and patient data for rare neurological disorders.
Webinar: Overcoming Patient Recruitment Challenges in Rare Disease Trials
Nearly 450 million people live with rare diseases globally. This diverse population needs treatment options, but clinical trials are made [...]
Endpoints Webinars: How the Current Investment Landscape is Stifling Rare Disease Development – September 15th
Despite the pressing need for appropriate treatments, the investment landscape is not amenable to therapeutic development. In an Endpoints webinar hosted on September 15th, speakers will discuss the current state of rare disease drug development funding and how the industry can foster innovation in spite of these difficulties.
Webinar: Overcoming Patient Recruitment Challenges in Rare Disease Trials – September 28th
Patient recruitment is one of the most difficult parts of any clinical trial, but it’s even tougher when studying therapeutics for rare diseases. In an upcoming webinar taking place on September 28th, speakers will discuss the unique challenges of patient recruitment in the field, how to speed up patient recruitment for rare diseases, and the lived experience of someone living with a rare disease.
World Orphan Drug Congress Europe 2022 – 14-17 November 2022
Taking place in Spain this year, the World Orphan Drug Congress, the leading conference for orphan drugs and rare diseases worldwide, takes place in Sitges, Spain, from 17-14 November. The conference will bring together professionals from healthcare, research, regulatory agencies, payer organizations, and other key decision-makers to one place to connect and share ideas.
EFPIA-EURORDIS Publishes Joint Statement on Patient Access to Rare Disease Medicines
The European Federation of Pharmaceutical Industries and a Associations has issued a joint statement with EURODIS Rare Diseases Europe on [...]
CDER Director Requests Accelerated Approval Reforms and Expresses Worries over Recent Court Decisions
Patrizia Cavazzoni aired her concerns about recent court cases and made requests regarding accelerated approval reform in a recent hearing [...]
