News & Perspectives

HFA Welcomes Our Summer 2022 Interns to Washington, DC

June 23, 2022

HFA is proud to welcome our 2022 Policy & Government Affairs Interns to Washington, DC for the summer! Brian and David will spend the next 10 weeks working in our DC offices, learning about all we do here at HFA. Over the summer, we will share blogs from both interns as they share their experiences. […]

On the Horizon 2022: Your Questions Answered

June 14, 2022

During Symposium 2022, we hosted our annual On the Horizon sessions. Several attendees asked great questions, so we assembled the questions and answers for you here.聽For more information on these topics, please visit the Current, New, and Emerging Therapies courses in HFA鈥檚 Learning Central鈥檚 University section. Enjoy! Question Answer Isn鈥檛 NovoSeven used for acquired hemophilia? […]

FDA Accepts CSL Behring鈥檚 Biologics License Application for Etranacogene Dezaparvovec for Priority Review 聽

June 2, 2022

HFA received the following announcement from CSL Behring. See full press release. FDA Accepts CSL Behring鈥檚 Biologics License Application for Etranacogene Dezaparvovec for Priority Review 聽 聽If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with hemophilia B This milestone underscores CSL Behring鈥檚 promise to develop and deliver a truly […]

FDA Grants Breakthrough Therapy Designation for Hemophilia A

June 2, 2022

HFA received the following press release from Sanofi. FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA Designation is based on XTEND-1 Phase 3 study data demonstrating a clinically meaningful prevention of bleeds and superiority in prevention […]

BioMarin Issues Clinical Program Update

June 2, 2022

HFA received this clinical program update from BioMarin. BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community BioMarin is providing an update to the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in […]

Bayer Discontinues Kogenate庐 FS in the United States

June 2, 2022

HFA received the following update from Bayer. For more than 25 years, Bayer has remained committed to supporting patients around the world by keeping a constant pulse on their evolving needs as well as those of their caregivers and healthcare providers (HCPs). Recognizing the growing shift in patient use toward more recent products, such as […]

Washington Wire: May 2022

May 31, 2022

Federal courts tackle questions surrounding copay accumulator adjusters While HFA and allies continue advocating for sound laws and regulations regarding copay assistance, we are also monitoring a couple of noteworthy (if weedy!) court cases relating to copay accumulator adjusters (CAAPs) and copay maximizers. On May 4, drug manufacturer Johnson & Johnson filed suit in federal […]

Pfizer Reports First Quarter 2002 Update of Gene Therapy Trial

May 3, 2022

Read the original press release here: During a recent quarterly stakeholder report, Pfizer announced that in March 2022 the US Food and Drug Administration lifted a clinical hold that had been placed on the Phase 3 AFFINE study in November 2021 following the observance of Factor VIII levels greater than 150% in some study […]

Washington Wire: April 2022

April 29, 2022

The so-called 鈥渇amily glitch鈥 is one of the persistent deficiencies in the Affordable Care Act. Under the ACA, people with access to employer-sponsored health coverage only qualify for subsidies to help them buy Marketplace health insurance if premiums for their employer plan are unaffordable (i.e., above 9.83% of household income). Affordability, however, is measured against […]

Hemophilia Federation of America Offers $1000 Grants to Learn New Skills and Help Land Your Dream Job!

April 29, 2022

2022 application is now open. Could you use $1000 to gain a skill or certification to help you get a job? Are you an adult (age 18 – 64) with a bleeding disorder? You may be eligible for an HFA Job Readiness Grant! From bookkeeping to blogging, Scrum Master to Salesforce Administrator, coder to Certified […]

Career Conversations: Job Readiness Recipients of 2021

April 10, 2022

Introducing our Job Readiness Grant Recipients Have you ever faced a barrier to employment because of your bleeding disorder or financial issues? Meet the recipients of HFA’s 2021 Job Readiness Grants. To learn more, visit our Job Readiness grants page. Ahmed Tareq Al Badri,聽Six Sigma Green Belt Certification I want to thank you for giving […]

Mental Health Resources

April 8, 2022

Save This Page With growing instances of mental health emergencies, it is important to know the warning signs of a mental health crisis in ourselves and our loved ones. This helpful handout provides valuable information on spotting the signs and what to say (and not say) when someone needs immediate help. Quick Links to Mental […]

Washington Wire: March 2022

March 31, 2022

Government health agencies, while continuing to monitor COVID-19 trends, have begun planning for the end of the public health emergency. As previously reported, the U.S. Department of Health and Human Services has for two years kept in place an official determination that COVID-19 constitutes a national public health emergency. While the PHE declaration remains in […]

State of the States: Winter 2022

March 15, 2022

Progress slows on state legislation to prohibit harmful copay accumulators After unprecedented success over the past two sessions, legislation to protect people who rely on copay assistance has struggled to gain traction in state legislatures so far this year. A dozen states (and Puerto Rico) already prohibit harmful 鈥渃opay accumulator adjusters鈥 鈥 an insurer strategy […]

Freeline Announces First Patient Dosed in Hemophilia B Trial

March 11, 2022

Read original press release here. LONDON, March 09, 2022 (GLOBE NEWSWIRE) — Freeline TherapeuticsHoldings plc announced that the first patient was dosed in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for the treatment of hemophilia B, a debilitating genetic bleeding disorder caused by a deficiency in the clotting factor IX protein. FLT180a uses […]

Sanofi and SOBI Announce Results of XTEND-1 Phase 3 Study

March 11, 2022

Read the original press pelease here. Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in hemophilia A, demonstrating superiority to prior factor prophylaxis treatment Once-weekly efanesoctocog alfa met primary endpoint in phase 3 study, resulting in a clinically meaningful prevention of bleeding episodes (bleed protection) In the key secondary endpoint, efanesoctocog alfa […]

Washington Wire February 2022

February 28, 2022

Court strikes down portions of surprise medical billing rule, but patient protections remain in effect. Last month HFA reported on new federal patient protections against 鈥渟urprise鈥 medical billing that went into effect on January 1, 2022. Under the new law and regulations, health care providers can no longer bill patients for amounts above their regular […]

Mild Matters: The Challenges of Managing Mild Bleeding Disorders

February 24, 2022

(Because Milds Matter Too!) When: Tuesday, March 8 from 7:00 鈥 8:00 pm EST This webinar is for patients, caregivers, and providers and includes all mild bleeding disorders such as vWD, hemophilia, platelet disorders, rare bleeding disorders, and those who consider themselves carriers or symptomatic carriers. Mild bleeding disorders are sometimes regarded as less serious, […]

BioMarin provides update to ongoing Phase 3 hemophilia A gene therapy study

February 4, 2022

An update for the hemophilia community from BioMarin, regarding the ongoing Phase 3 BioMarin hemophilia A gene therapy study, and a serious adverse event deemed, by the Independent Data Safety Monitoring Committee, unrelated to the investigational gene therapy from the phase 1/2 study. View the original Clinical Development Program Update BioMarin鈥檚 investigational gene therapy for […]

Women Power

February 4, 2022

Traditionally, women often wait years for a proper bleeding disorders diagnosis. But more awareness, advocacy and treatments are improving things now and for future generations.聽 By Risa Kerslake, Freelance Writer Kristin Voyles didn鈥檛 know growing up that she had von Willebrand disease (VWD). Looking back though, it made sense. She bruised easily and had heavy […]

The Legacy of HTCs

February 4, 2022

Hemophilia treatment centers have been around for almost 50 years. Learn about their history, successes, and new challenges.聽 By Rebecca A. Clay聽 At 58, Michael Birmingham, of Tacoma, Washington, is old enough to remember what life was like for kids with bleeding disorders before the advent of hemophilia treatment centers (HTCs). Diagnosed with severe hemophilia […]

Career Dreams

February 4, 2022

New HFA Job Readiness Grants help community members reach their career goals and improve their lives. By Allison Harrison, MSW, Associate Director of Services聽 Have you ever felt stuck in your career with low pay, limited opportunities that fit your needs or lack of advancement potential? You鈥檙e not alone.聽 鈥淏etween my physical health and the […]

Fore the Love of the Game

February 4, 2022

Hogan Lust has become a teenage standout in one-arm golf association tournaments By Melanie Padgett Powers, Managing Editor Hogan Lust, age 16, of Tucson, Arizona, has been playing golf since he was a toddler. His parents, Daniel and Corby, are golfers too, so it was only natural their oldest child would play the sport.聽 鈥淗e […]

FDA Approves Prophylactic Treatment with VONVENDI庐 [von Willebrand Factor (Recombinant)] for Adult Patients Living with Severe Type 3 von Willebrand Disease (VWD)

February 2, 2022

Original Source: Takeda Press Release January 31, 2022 VONVENDI [von Willebrand Factor (Recombinant)] is the First and Only Treatment Approved for Routine Prophylaxis to Reduce the Frequency of Bleeding Episodes in Adults Living with Severe Type 3 VWD Receiving On-Demand Therapy VWD is the Most Common Inherited Bleeding Disorder, Affecting up to Three Million (or […]

Celebrating Black History Month

January 31, 2022

As our nation celebrates Black History Month, the Hemophilia Federation of America would like to recognize the contributions of Black Americans within the bleeding disorders community. This month, we will amplify the stories of Black community members and hope you鈥檒l celebrate them with us. We recognize our society still has many challenges to address, but […]

Washington Wire: January 2022

January 31, 2022

Readers of the Washington Wire will have noticed that HFA publishes a lot of posts about 鈥渃opay accumulator adjuster programs鈥 (CAAPs). This is a weedy but important topic! CAAPs are a strategy used by insurers to redirect the value of manufacturer copay assistance 鈥 which is supposed to help patients afford their share of their […]

Lessons Learned

January 31, 2022

Members of the bleeding disorders community gathered virtually in September for the HFA Young Adult Advocacy Summit. Here, two first-time participants share their stories and explain why they wanted to get more involved with advocacy efforts.聽 Rodr铆guez-Rivera,聽Bayam贸n, Puerto Rico In September, I attended the Hemophilia Federation of America (HFA) virtual Young Adult Advocacy Summit. I […]

6 Lessons to Prepare for College from One Family鈥檚 Ordeal

January 31, 2022

by Melanie Padgett Powers, Managing Editor When Josh Morton rolled his right ankle shooting hoops at college, he thought factor and RICE鈥攔est, ice, compression, elevation鈥攚ould heal it. But his pain and swelling quickly grew, so he called his mom, who told him to head to the emergency room (ER). What happened next showed Josh and […]

Adoption as an Option

January 31, 2022

Several bleeding disorders families share their adoption experiences and advice. 聽 By Holly Leber Simmons, freelance writer Natalie Andrews remembers the first time she saw a picture of her son, Samuel, in 2016. 鈥淗e looked terrible. He had black eyes and it looked like someone had punched him,鈥 she said. 鈥淗is joints were badly swollen; […]

Aging with Resilience

January 31, 2022

We welcome the first generation of men with hemophilia living into older age鈥攁nd explore the medical issues that come with it. By Heather Boerner, freelance writer When Dan Liedl, who has severe hemophilia A, wakes up in his Morgantown, West Virginia, home every morning, he feels fine. It鈥檚 only when he starts to move that […]

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