CDER Rare Diseases Public Workshop: Strategies, Tools and Best Practices for Effective Advocacy in Rare Diseases Drug Development

The Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) is sponsoring a public workshop on rare disease advocacy Monday, October 30, 2017. This public workshop builds upon previous CDER patient advocacy public workshops and is primarily for the rare disease community members to help them effectively understand FDA's needs for enhance drug development. This workshop will include case studies demonstrating the beneficial overlap of effective advocacy techniques and FDA regulations in rare disease drug development.


There is no registration fee to attend the public workshop. Early registration is recommended because seating is limited. Registration will be on a first-come, first-served basis. There will be no onsite registration. Persons interested in attending this workshop must register before September 20, 2017 at Eventbrite url. Those without internet access should contact Francis Kalush to register.


The workshop will be held at the FDA White Oak Campus, 10903 New Hampshire Avenue Bldg. 31, Room 1503 (Great Room) Silver Spring, Maryland 20993. Public workshop participants (non-FDA employees) should enter through Building 1 of the White Oak Campus where routine security check procedures will be performed. If special accommodations are necessary due to a disability, please contact Francis Kalush at least 7 days in advance.


Francis Kalush Ph.D., Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD20993, 240-796-5429.


A transcript of the workshop will be available for review at the FDA Freedom of Information Act and Dockets Public Reading Room in room 1061 of 5630 Fishers Lane, Rockville, MD20852, and on the Internet at Transcripts will also be available in either hard copy or on CD-ROM after submission of a Freedom of Information request.



John McInnes, CMS director of the Division of Outpatient Care, is leaving the agency June 30 after seven years to return to Arnold Porter. McInnes said from the beginning his plan was to work at the agency for five to 10 years to develop policies for packaging outpatient services for Medicare reimbursement, and he has accomplished that goal.

McInnes had several jobs at the agency, and the Hospital Outpatient Prospective Payment System was his primary responsibility.

Packaging is a critical feature of the outpatient pay system. Within each Ambulatory Payment Classification, which is the primary unit of payment within the prospective pay system, reimbursement for dependent, ancillary, supportive and adjunctive items and services are packaged with the primary independent service. -- John Wilkerson (