The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) convened a Safety Summit in Washington, DC, on January 29-30th, 2020, to discuss monitoring, educating and communicating issues around bleeding disorders product safety.
The summit brought together stakeholders from the Centers for Disease Control and Prevention (CDC), pharmaceutical manufacturing companies, home healthcare companies, clinicians, patient advocacy organizations, and individuals affected by bleeding disorders.
The summit was convened in response to a number of recalls and product safety notifications that were issued in 2019. The two organizations, who are trusted resources for unbiased information about treatments and products, wanted to have forum to discuss how to improve education around current drug safety standards and communications to the people who use those therapies.
“We have seen a huge leap forward in terms of new therapies in the past decade, and the years ahead promise to bring even more innovations in treatment,” said Michelle Rice, chief external affairs officer for NHF. “However, it’s more important than ever that our patients have access to clear, understandable information about any safety issues that might arise with these new treatments.”
Participants began with an in-depth explanation of the regulatory requirements for drug development, which segued into a panel discussion on the methods of safety reporting, including the responsibilities of each stakeholder.
Attendees also broke into working groups during the summit to discuss a case study of a notification or concern with the goal of discovering where gaps in communication typically arise. The participants also discussed gene and novel therapies, and how to improve education for both patients and clinicians on the emerging technologies.
“Making sure our patients with bleeding disorders have safe therapies isn’t the responsibility of just one group,” said Sonji Wilkes, senior director of policy and advocacy for HFA. “Everyone, from the government to the pharmaceutical companies to organizations like ours to the patients themselves have a part to play.”
NHF and HFA will create a preliminary report summarizing the findings of the summit and make it available to the bleeding disorders community for comment before presenting the finalized paper at HFA’s Symposium in April.
“Nobody knows better than people with bleeding disorders the need for everyone involved around the manufacturing and distribution of drugs to be honest and transparent about safety,” said Rice. “We now have the blueprint for procedures that will give our patients better protection and understanding as we go into a new era of treatment and care.”
