Patient Advocacy Summit 2019

November 6, 2019 8:00 AM - 4:30 PM

Marriott Boston Cambridge, 50 Broadway, Cambridge, Massachusetts 02142 USA

Add to Calendar 11/6/2019 8:00:00 AM 11/6/2019 4:30:00 PM Patient Advocacy Summit 2019 Marriott Boston Cambridge, 50 Broadway, Cambridge, Massachusetts 02142 USA

MassBio's Patient Advocacy Summit brings together the patient advocacy community to showcase best practices and cutting-edge examples of how life sciences companies can more fully incorporate the patient voice into the work they do—not just approaching regulatory applications or at commercialization, but throughout the drug development cycle.

This year we'll explore competing views on what value therapies bring to various stakeholders, what a cure is worth to patients and society, how to improve the clinical trial experience for patients, and more. We will also have a frank discussion about patient access in the era of transformative therapies, and you’ll hear directly from patients on what this means to them.

The program includes panel discussions, case study presentations(spotlighting industry/patient partnerships), an inspirational keynote address, as well as a networking lunch and cocktail reception.

See sponsorship opportunities here, or contact Laura Rudberg.

Founder & President of Texas Rare Alliance & Advisory Committee Member, Texas Newborn Screening
Khrystal K. Davis Founder & President of Texas Rare Alliance & Texas Newborn Screening Advisory Committee Member. Khrystal joined the rare disease community in 2011 when her newborn son, Hunter, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, the leading genetic cause of mortality in children under the age of two. In May of 2016, Khrystal advocated alongside the FAST Movement (Families for the Acceleration of Spinal Muscular Atrophy Treatments) in a meeting with top FDA representatives for access to Spinraza, an SMA treatment in clinical trials at the time. Together with other FAST members, she asked the FDA to stop placebo trials, provide a means of access for the weakest SMA patients, accelerate the approval, and approve the treatment for all SMA patients regardless of age or type of SMA. In an interim look completed on August 1, 2016, the FDA found the treatment met trial objectives. An Expanded Access Program for SMA Type 1 patients commenced August 12, 2016, and the FDA approved Spinraza for children and adults with SMA on December 23, 2016. Khrystal is committed to improving health outcomes in those with rare diseases through improved access to rare disease diagnoses and treatments. She advocates for whole genome sequencing of newborns, the expansion of newborn screening programs, insurance policies that conform to FDA labels for orphan drugs, and clinical trials and real-world evidence that include endpoints and data points that are meaningful to patients and caregivers. She is a proponent of patient-driven access to rare disease treatments. Khrystal holds a Juris Doctorate from Stetson University College of Law and is certified in Clinical Trial Design and Interpretation by Johns Hopkins University and Drug Discovery by University of California San Diego through the Coursera program.

Brought to you by