Expanded Access to Advanced Cell Therapies
Guest Author: Frances Verter, PhD, Parents’ Guide to Cord Blood Foundation Thank you to Ax-S Pharma for the invitation to submit a contribution to your blog. My name is Frances Verter, and I am a founder of two organizations in the cell therapy space: the 501c3 non-profit Parent’s Guide to Cord Blood Foundation, and […]
Becoming Board Certified in Medical Affairs (BCMAS): The new standard in Pharma
Anyone working in medical affairs understands that it’s a highly specialized area. And often, medical affairs executives get so engrossed in their day to day activities that it’s challenging to gain a deep understanding of how other departments and functions operate, let alone train another colleague on these functions. But as medical affairs’ strategic value […]
FDA’s Project Facilitate fielded 161 compassionate use requests in first three months
Project Facilitate, an FDA call center that guides physicians through the process of obtaining expanded access — or compassionate use — to investigational cancer therapies, handled about 161 requests from its launch on June 3 to Aug. 31, an agency spokesperson told BioCentury.
Peter J. Pitts: Executive action could snuff medical research
Price controls are the wrong approach; they will stunt drug innovation and restrict patient access to new cures. Reason: Drug development is risky.
Expediting Access to Rare Disease Therapies in Europe
Globally there are approximately 7,000 known rare diseases. About 4,000 – 5,000 of those have no available therapies . Consequently, there are many patients with a rare disease who are waiting for a new therapy to become available.
Biosimilar Approvals And The BPCIA: Too Soon To Give Up
The Biologics Price Competition and Innovation Act (BPCIA), part of the 2010 Affordable Care Act, sought to drive down prices for biologics, much as the 1984 Hatch-Waxman Act did for small-molecule drugs. By allowing manufacturers…
Three (BIG) things you didn’t know about Expanded Access to new medicines
If you’re a drug company developing novel treatment for a serious unsolved disease, you have already been asked: “What’s your strategy for pre-market access?” If you’re a health care provider, health system, or disease organization serving patients with life-threatening conditions, you’ve been asked something similar. Do you have a good answer? The patients you serve […]
Why I Launched the Expanded Access Summit
I am not an event planner. In my early life as an investment manager and, more recently as a life science entrepreneur, I doubted the value of most “can’t miss” conferences. The energy and enthusiasm, the inspirational speeches, the provocative on-stage remarks – great for morale. But how often did they truly impact the landscape their attendees […]
Your updates for the global forum on pre market medicines access
The Expanded Access Summit is about the smart integration of Expanded Access and Named Patient programs into the modern drug development process. The annual leadership event was launched in 2017 to establish (and renew) best practices for big pharma, small (pre-revenue) pharma, health care providers, and public and private research networks. We invite ALL stakeholders […]
Announcement of Date and Location Change!
In response to the tremendous interest our topic is getting from media, elected officials, and policy organizations, we are excited to announce the following changes to the second annual Expanded Access Summit, the national forum on pre-approval treatment access: Broader audience and speaker faculty, to include more HHS officials, academia, and national policy institutes. New […]