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Be sure to check back regularly to get our latest news updates.

Supporting the Judith Graham Pool Fellowship

Investing in research that deepens our understanding of inheritable bleeding disorders and fuels new treatment options is important to NHF.

Findings such as these could begin to fill a knowledge gap as current recommendations relevant to the safe use of epidural analgesia during pregnancy in VWD patients are not well defined in the U.S.

This webinar explores the diagnosis and clinical management of patients with rare inherited bleeding and platelet disorders.

First National HTC Patient Satisfaction Survey: The Results Are In

The survey was designed to assess patient demographics, their satisfaction with the HTC’s core multidisciplinary care team, plus affiliated clinicians, services, and care processes.

COVID-19 Vaccines: What to Expect

Watch NHF President and CEO Dr Len Valentino discuss the many unanswered questions regarding a COVID 19 vaccine and what it would mean for the world.

The My Life, Our Future Research Repository includes the largest bank of genomic and phenotypic data about people with hemophilia.

The First National Patient Satisfaction Survey of U.S. HTCs

This undertaking represents the first-of-its kind, a nationally uniform and comprehensive patient satisfaction survey of HTC clinicians, services and care processes.

A joint statement of the World Federation of Hemophilia (WFH), European Haemophilia Consortium (EHC) and National Hemophilia Foundation (NHF).

COVID-19 Vaccines: What to Expect

We all want an end to this global pandemic, and an effective vaccine is a critical part of finding that path. However, there are many unanswered questions on the effectiveness of the vaccine, how it will be distributed, and who will have access to it.

COVID-19 Vaccines: What to Expect

Join us on Friday, November 6th at 4:00pm EDT to discuss COVID-19 Vaccines: What to Expect, with NHF President and CEO Leonard A. Valentino, MD.

With Patient Perspectives in Mind, ICER Evaluates Hemophilia Therapies

On October 30th, 2020, the Institute for Clinical and Economic Review (ICER) held a public meeting to discuss its evidence report on therapies for hemophilia A. ICER is an independent and non-partisan research organization that evaluates the clinical and economic value of prescription drugs, medical tests, and other healthcare and healthcare delivery innovations. For this report, ICER reviewed emicizumab (Hemlibra, produced by Genentech) and valoctocogene roxaparvovec (Roctavian, produced by Biomarin), a gene therapy currently in phase 3 clinical trials.

ICER’s review individually compared emicizumab and valoctocogene roxaparvovec to prophylaxis with clotting factor. ICER’s analysis found that over a lifetime compared to prophylaxis with factor replacement, emicizumab reduces cost and produces equal or substantial net health benefits for people with hemophilia A. In their assessment of valoctocogene roxaparvovec, ICER used a placeholder price of $2.5 million. At this price, when compared to prophylaxis, ICER found valoctocogene roxaparvovec would also be cost saving but given the on-going clinical trial, the evidence of health benefit was deemed promising, but inconclusive.  

In the review, ICER created an economic model of costs and clinical effectiveness based on data reported from clinical trials, related studies, and evidence for patient-important outcomes. The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) participated throughout the 9-month ICER review process providing evidence, expert testimony, and real-life patient testimony of the impact of hemophilia on daily living and the importance of ensuring access to potentially transformative therapies. Throughout, NHF and HFA have stressed the importance of including real-world, patient-important outcomes when assessing the value of emicizumab or gene therapy for those living with hemophilia. Clinical trial data focuses primarily on bleeding frequency and typically undervalues outcomes important to patients.

“Given the transformative nature of the therapies under consideration, patient important outcomes including joint pain, limitations on activities, lost time from school or work, emotional well-being, psychological stress related to anticipation of bleeding especially with activities, and of course, the cost of bleeding to the healthcare system must be considered to demonstrate benefit to patients, their families and society,” said Leonard A. Valentino, MD, president and CEO of NHF.

ICER’s review is helpful for assessing the economic and clinical value of a therapy; it should not override any treatment decisions that are made between doctors and patients, said Sonji Wilkes, senior director of policy and advocacy of HFA. “The choice of treatment for each person with hemophilia needs to be individualized, patient-centric, accessible and affordable. The full range of products – clotting factor, non-factor therapies, and eventually gene therapies – must be available for patients, working in consultation with their doctors, to develop treatment plans that best preserve their health and quality of life.”

“Today is the culmination of more than five years’ worth of work by the national and international hemophilia communities to define patient-important outcomes and collect real-world evidence to assess the true reflection of value for a new therapy: the difference it makes in the lives of those living with the disease,” commented Mark W. Skinner, hemophilia community member, member of ICER’s expert review panel and ICER board member. “We appreciate ICER’s increasing efforts to engage and include patients directly in its review process and recognize that our work is not done. Enhancing the patient voice is vital if patient advocacy groups, providers, manufacturers, payers and policymakers are to find common ground and ensure high-value therapies remain within reach of patients.”

Hemophilia experts stressed to ICER’s panel, as well as other stakeholders participating in the meeting, that all treatment options should be available to patients, without barriers due to cost, short-sighted utilization management strategies, narrow formularies or narrow provider networks. The role of the hemophilia treatment center (HTC) will also continue to be extremely important in helping patients make these complex treatment decisions, managing utilization and in providing the longitudinal follow-up required for these new treatments.  

NHF, HFA and the entire bleeding disorders community look forward to generating additional real-world data to demonstrate the value of these transformative therapies to not only better inform ICER’s economic modeling and analysis but to preserve access to these life-changing treatments for all people with bleeding disorders.

Highlights from NHF’s Virtual Insurance and Reimbursement Conference

The intensive, four-day virtual workshop provided participants with the necessary tools and resources to help the community maintain access to care.

This scholarship is named in tribute to Kevin Child, who died of AIDS in 1989, shortly before graduating from college.

Public Health Symposia Now Available on the NHF Website!

These symposia were designed to encompass critical topics with far reaching implications for our communities, including emerging and novel therapies, surveillance, and key public health-related issues relevant to affected women and girls.

86 percent of registered voters believe the government should require copay assistance to be applied to patients’ cost-sharing requirements 

Thirty three organizations, including NHF, urge elected officals to ensure all people living in the US have access to adequate and affordable health coverage.

The purpose of the study is to evaluate the efficacy and safety of this investigational gene therapy in patients with moderately severe to severe hemophilia A.

NHF’s Red Tie Soiree is a Virtual Success

On September 25, more than 300 people from across the country tuned in to this year’s virtual Red Tie Soiree.

Gene Therapy: What's New & What's Next Webinar

Watch this moderated webinar regarding the current status of gene therapies in the pipeline and other novel therapies.

HHS Announces New Coronavirus Funding for Healthcare Providers

The funding considers financial losses caused by the pandemic.

Primary care physicians, obstetrician/gynecologists, nurse/nurse practitioners, dentists, oral surgeons and other allied professionals are all encouraged to take advantage of these online activities.

CSL Behring States Stimate Will Not Be Resupplied until 2022

CSL Behring provided a statement to NHF nand HFA regarding the recall of Stimate® (desmopressin) nasal spray.

What's Next for the ACA?

How will the Trump administration's lawsuit against the ACA affect the bleeding disorders community?

The abstracts featured at this summer's BDC covered a wide spectrum of research topics.

This commentary was authored by six individuals with severe hemophilia, all of whom have worked to achieve better health outcomes for patients with bleeding disorders.