Research Studies

Objective:

NHF Chapters must develop, support, and sustain influential advocacy programs to protect and enhance access to health care for the bleeding disorders community.

Method:

NHF supports the development of state health care policy advocacy programs with in-kind and financial assistance. NHF’s State-Based Advocacy Coalition (SBAC) program and the standard advocacy expectations our Chapters are the pillars of a robust framework of state advocacy programs. The standard advocacy expectations provide clear expectations and identify best practices for advocacy programming. These include: an advocacy committee of staff and volunteer advocates that meets monthly, multi-year strategic planning, clear engagement with legislative and administrative policymakers, an advocacy annual budget, and more. Chapters are also expected to actively engage their network of Hemophilia Treatment Centers, and all Industry partners.

The SBAC program provides funding and in-kind support from NHF for chapters to either start an advocacy program or grow and maintain an advanced one, using the standard advocacy expectations as a guide. NHF provides extensive in-kind support for SBAC grantees, as well as assisting all other chapters with their specific advocacy needs.

Summary:

Since the inception of the SBAC program NHF has seen participation by and enthusiasm among community members in advocacy events and programming grow. Nearly 500 volunteers participated in NHF’s Washington, DC Days in recent years. Chapter run State Advocacy Days are more prevalent and well-attended. At least 1000 advocates now travel to their state Capitol each year to participate in chapter Advocacy Days. There are 15 grantees participating in the SBAC program covering 20 states. Collectively, NHF has trained more than 5,000 volunteer advocates across the country.

Increasing numbers of volunteer advocates have turned out in state capitols for several years to make their voices heard and influence health care access. In addition, NHF’s investments in public policy resources have allowed us to encourage and support state regulatory advocacy, e.g. with state Medicaid offices. These new efforts have demonstrated tangible results in the form of protecting and enhancing patients’ access to care.

The promotion and support of state chapter advocacy programs by NHF, especially among the SBAC-participating states, has advanced the public policy interests of the bleeding disorders community. Objectively, we’ve seen the increased numbers and enthusiasm of volunteer advocates. Anecdotally, there are many stories of chapters playing a key role in state health care policy,

Conclusion:

Successful state advocacy work has led to improved access to quality healthcare. Start-up and ongoing support from NHF have played a critical role in that success. The continued refinement of our Chapter standard advocacy expectations and in-kind support of NHF policy staff will continue to be key to advancing state advocacy programs uniformly around the country.

The hemophilia and thrombosis centers in Tucson and Phoenix would like to join efforts to make a meaningful contribution to our understanding of the mental health profile of our pediatric population. This knowledge could contribute to a more tailored approach when designing clinics and programming, and, by identifying mental health issues, inform the development of targeted interventions. We hope to look at the prevalence of depression and anxiety in children with bleeding disorders including hemophilia, von Willebrand disease, and other congenital coagulopathies. We expect that the existence of a chronic health condition could affect a child's psychological development. We're also aware of the critical impact of family culture so our survey will include data on the mental health of the participants’ primary caretakers as well as other significant socioeconomic markers.

Dr. Satish Nandakumar is currently a postdoctoral fellow in the laboratory of Dr. Vijay Sankaran at the Boston Children's Hospital. Previously, he did his graduate work at the St. Jude's Children's Research Hospital in Memphis, Tennessee. In his JGP Fellowship project, Dr. Nandakumar aims to develop a novel gene therapy approach for hemophilia that involves activation of the endogenous factor VIII or IX genes within hematopoietic stem cells by taking advantage of the CRISPR/Cas9 gene activation system. This work has the potential to benefit patients with mild hemophilia mutations.

Objective:

While a new generation of therapies for patients with Hemophilia A are available, it is unclear what patient characteristics, perceptions, and barriers are associated with the decision to switch FVIII replacement therapies. This study assessed patient characteristics, health history, and reasons for switching from a FVIII product with more frequent dosing (³3x infusions/week) to a product with less frequent dosing (≤2x infusions/week) from patient/caregiver and physician perspectives.

Methods:

Data collection was a mix of qualitative and quantitative procedures. The qualitative portion consisted of two online discussion forums: patients (n=17) and caregivers of patients (n=11) receiving a FVIII product dosed ³3x/week, and patients (n=22) and caregivers of patients (n=5) who switched to a product dosed ≤2x/week. The quantitative portion was a retrospective medical chart review (n=207) which captured variables (e.g., bleed rate, treatment history) 6 months pre- and 6 months post-switching to a product with less frequent dosing.

Summary:

Prominent drivers among patients for starting a FVIII product with less frequent dosing included: 1) experiencing diminished effectiveness while on a product dosed ³3x/week resulting in increased breakthrough bleeding, 2) experiencing vein access issues, and 3) beginning prophylaxis as opposed to on-demand infusions after a bleed.

Key barriers to changing included: 1) fears regarding the process of switching being complicated, time consuming, and costly, 2) perceived risks associated with switching, and, 3) possible lack of healthcare provider support.

Physicians were most likely to report that patients switched products because they sought a newer product with twice weekly dosing or less per FDA-approved dosing recommendations (35.3%), followed by patient requested the switch (30.4%), and patient sought a reduction in infusion frequency to improve adherence (27.5%).

Switching to a product with less frequent dosing was associated with improvements in patient-reported bleeding-related outcomes. Patients were more likely to self-administer the treatment post-switch (63.8%) compared with pre-switch (48.8%; p<0.001) and had fewer infusions per week post-switch (2.8 vs. 3.3; p<0.001). Patients’ annualized bleed rate was lower (5.9) post-switch compared with pre-switch (7.7; p<0.001).

Both the number of spontaneous joint bleeds and joint bleeds after trauma or injury were lower (3.2 and 2.7) post-switch (3.6 and 4.3; p=0.044 and p<0.001). The bleeding event was less likely to be classified as moderate or severe (34.5% and 5.9%) post-switch compared with pre-switch (55.0% and 10.0%; p<0.001 and p=0.049). Fewer infusions were required to resolve the bleeding event post-switch (2.6 vs. 3.2; p<0.001).

Conclusion:

A prominent reason why patients switch treatment is to improve bleeding-related outcomes. Indeed, we found that switching to a FVIII product with less frequent dosing was associated with improved patient-reported bleeding-related outcomes. These findings are critical for improving patient outcomes and support the FDA mandate to incorporate patient perspectives in the regulatory process.