Addressing Product Safety for the Bleeding Disorders Community

Multistakeholder summit aims to improve education and communication

The National Hemophilia Foundation (NHF) and the Hemophilia Federation of America (HFA) convened a Safety Summit on January 29-30, 2020, in Washington, DC, 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, pharmaceutical manufacturing companies, home healthcare companies, clinicians, patient advocacy organizations and people affected by bleeding disorders.

The summit was convened in response to a number of recalls and product safety notifications issued in 2019. As trusted resources for unbiased information about treatments and products, NHF and HFA put on the forum to discuss how to improve education on current drug safety standards and communications to 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.”

The summit walked participants through an in-depth explanation of the regulatory requirements for drug development, then segued into a panel discussion on the methods of safety reporting, including the responsibilities of each stakeholder.

Attendees also broke into working groups 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 has a part to play.”

NHF and HFA released a report summarizing the findings of the summit in April 2020.

“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,” Rice says. “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.”