INFLUENCING DRUG DEVELOPMENT
In no other drug development arena has the patient been so influential. Patients, advocates and advocacy groups are impacting regulation and driving orphan drug development. Government, Payers and Industry alike are seeking collaborations with patients to improve clinical trials, drug development and access. The role of the patient is changing once more. Rare Disease Advocacy World will kick off with industry-led investigation into the impact of patient-industry collaboration.
ADVOCACY IN ACTION
After a general look at how patients are influencing drug development, Parent Project Muscular Dystrophy (PPMS), PTC Therapeutics and EveryLife Foundation for Rare Diseases will provide practical insight into how patient groups can impact policymaking and orphan drug development, through realworld examples.
Are initiatives such as ‘Right to Try’ the future of approval and patient access to treatment? Rare Disease Advocacy World is facilitating a multi-stakeholder examination of the latest access strategies, and how they will impact the review of new therapies for rare diseases.
NATIONAL PATIENT ORGANIZATIONS
Learn from the world’s leading advocates. Leaders from National Patient Organizations from the US, South Africa, Australia, Colombia, New Zealand, Japan, India and Europe will be discussing the opportunities for rare disease research and development in their respective regions. How has multi-stakeholder collaboration promoted research, diagnosis, treatments and service for all rare diseases in Australia? How has Japan made rare and intractable diseases an Asian health priority? How has EURORDIS unified the voice of European rare disease patients?