The World Health Organization (WHO) has updated its Essential Medicines List to prioritize modern, safer therapies for hemophilia and von Willebrand Disease, advancing global access and aligning policy with current standards of care.
The World Health Organization (WHO) has updated its Essential Medicines List to prioritize modern, safer therapies for hemophilia and von Willebrand Disease, advancing global access and aligning policy with current standards of care.
Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
A new study confirms the Hemophilia Device Handling and Preference Assessment (HDHPA) as the first validated tool to capture patient and caregiver perspectives on treatment device preferences.
A new study confirms the Hemophilia Device Handling and Preference Assessment (HDHPA) as the first validated tool to capture patient and caregiver perspectives on treatment device preferences.
New real-world evidence shows that people with hemophilia A who experience bleeding or joint complications face significantly higher health care utilization and comorbidity burden.
New real-world evidence shows that people with hemophilia A who experience bleeding or joint complications face significantly higher health care utilization and comorbidity burden.
Novo Nordisk has announced the US Food and Drug Administration (FDA) approval of Alhemo (concizumab-mtci) for use as a once-daily subcutaneous prophylactic treatment in people 12 years and older with hemophilia A or B (HA/HB) without...
Novo Nordisk has announced the US Food and Drug Administration (FDA) approval of Alhemo (concizumab-mtci) for use as a once-daily subcutaneous prophylactic treatment in people 12 years and older with hemophilia A or B (HA/HB) without...
Five-year data from BioMarin’s phase 3 trial reveals Roctavian’s long-term efficacy, safety, and quality-of-life benefits for adults with severe hemophilia A.
Five-year data from BioMarin’s phase 3 trial reveals Roctavian’s long-term efficacy, safety, and quality-of-life benefits for adults with severe hemophilia A.
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A new post hoc analysis from the PERSEPT 1 trial offers compelling evidence supporting the use of eptacog beta for managing acute pain during bleeding episodes (BEs) in patients with hemophilia A or B with inhibitors (PwHABI). The data...
A new post hoc analysis from the PERSEPT 1 trial offers compelling evidence supporting the use of eptacog beta for managing acute pain during bleeding episodes (BEs) in patients with hemophilia A or B with inhibitors (PwHABI). The data...
Pfizer’s hympavzi achieved a 93% reduction in bleeding episodes in a phase 3 trial for patients with hemophilia A and B with inhibitors, offering a promising once-weekly alternative to on-demand therapies.
Pfizer’s hympavzi achieved a 93% reduction in bleeding episodes in a phase 3 trial for patients with hemophilia A and B with inhibitors, offering a promising once-weekly alternative to on-demand therapies.
A real-world study shows that emicizumab offers safe and effective outpatient bleeding prophylaxis for patients with acquired hemophilia A, even amid complex comorbidities.
A real-world study shows that emicizumab offers safe and effective outpatient bleeding prophylaxis for patients with acquired hemophilia A, even amid complex comorbidities.
The Senate has done it. After weeks of behind-the-scenes wrangling and last-minute amendments, lawmakers have passed a sprawling tax-and-spending megabill that makes good on a long-standing Republican goal: making the 2017 Trump tax cuts...
The Senate has done it. After weeks of behind-the-scenes wrangling and last-minute amendments, lawmakers have passed a sprawling tax-and-spending megabill that makes good on a long-standing Republican goal: making the 2017 Trump tax cuts...
In Washington, the most consequential policies are often buried in the fine print. One such detail in the Senate Finance Committee’s latest tax and spending proposal would gradually cap provider taxes—a core funding tool states use to pull in...
In Washington, the most consequential policies are often buried in the fine print. One such detail in the Senate Finance Committee’s latest tax and spending proposal would gradually cap provider taxes—a core funding tool states use to pull in...
Over the past few weeks, I’ve been following the progress of the One Big Beautiful Bill Act (OBBBA), which started in the House with sweeping tax reforms and some eye-catching health care proposals. Now, the Senate has released its (highly...
Over the past few weeks, I’ve been following the progress of the One Big Beautiful Bill Act (OBBBA), which started in the House with sweeping tax reforms and some eye-catching health care proposals. Now, the Senate has released its (highly...
With the One Big Beautiful Bill Act (OBBBA) still in the hands of the Senate, much of the national conversation has centered on what this legislation would do to federal coverage programs. But regardless of how the federal process unfolds,...
With the One Big Beautiful Bill Act (OBBBA) still in the hands of the Senate, much of the national conversation has centered on what this legislation would do to federal coverage programs. But regardless of how the federal process unfolds,...
It’s been weeks of headline-grabbing debate over Medicaid work requirements, and the “One Big Beautiful Bill Act” remains in the Senate as they deliberate over several health care provisions. While the Medicaid provisions sparked most of the...
It’s been weeks of headline-grabbing debate over Medicaid work requirements, and the “One Big Beautiful Bill Act” remains in the Senate as they deliberate over several health care provisions. While the Medicaid provisions sparked most of the...
Last week, the House of Representatives narrowly passed the One Big Beautiful Bill Act (HR 1, 119th Congress) by a single vote, sending the sweeping tax and spending reconciliation package, backed by President Trump, to the Senate. With its...
Last week, the House of Representatives narrowly passed the One Big Beautiful Bill Act (HR 1, 119th Congress) by a single vote, sending the sweeping tax and spending reconciliation package, backed by President Trump, to the Senate. With its...
It’s been another eventful week in health policy—a phrase that’s quickly becoming routine. This time, House Republicans advanced a sweeping budget reconciliation bill, designed to implement elements of the March budget resolution, with a...
It’s been another eventful week in health policy—a phrase that’s quickly becoming routine. This time, House Republicans advanced a sweeping budget reconciliation bill, designed to implement elements of the March budget resolution, with a...
It’s been a busy few days for drug pricing policy. While I’d love to dig into the details of both President Trump’s latest executive order and the newest Republican bill that would cut Medicaid spending, we don’t have the time—and this isn’t...
It’s been a busy few days for drug pricing policy. While I’d love to dig into the details of both President Trump’s latest executive order and the newest Republican bill that would cut Medicaid spending, we don’t have the time—and this isn’t...
In the span of just 9 months, the landscape of administrative law has undergone a transformation so profound that pharmaceutical executives, health care providers, and patients alike are struggling to navigate the new terrain. The Supreme...
In the span of just 9 months, the landscape of administrative law has undergone a transformation so profound that pharmaceutical executives, health care providers, and patients alike are struggling to navigate the new terrain. The Supreme...
Over the past 2 weeks, I’ve examined the growing friction between the Section 232 pharmaceutical tariff investigations and the Medicare drug pricing reforms authorized by the Inflation Reduction Act (IRA). These 2 policy agendas—industrial...
Over the past 2 weeks, I’ve examined the growing friction between the Section 232 pharmaceutical tariff investigations and the Medicare drug pricing reforms authorized by the Inflation Reduction Act (IRA). These 2 policy agendas—industrial...
Featuring Yash Prajapati, consultant at ZS Associates
Featuring Yash Prajapati, consul...
Join us in exploring the challenges and opportunities in the development, approval, and integration of digital therapeutic solutions with ZS Associates consultant Yash Prajapati.
Join us in exploring the challenges and opportunities in the development, approval, and integration of digital therapeutic solutions with ZS Associates consultant Yash Prajapati.
Featuring Tejaswi Kompala, MD, head of cardiometabolic clinical strategy, Teladoc
Featuring Tejaswi Kompala, MD, h...
Delve into the rising demand for anti-obesity medications, the complexities of obesity management, and the importance of personalized approaches in improving cardiometabolic health in this insightful interview.
Delve into the rising demand for anti-obesity medications, the complexities of obesity management, and the importance of personalized approaches in improving cardiometabolic health in this insightful interview.
The FDA's new regulations on laboratory-developed tests have sparked debate over access to innovative tests and patient care in the medical field, with opponents arguing that increased oversight could hinder the development of crucial new...
The FDA's new regulations on laboratory-developed tests have sparked debate over access to innovative tests and patient care in the medical field, with opponents arguing that increased oversight could hinder the development of crucial new...
Focusing solely on relapse rates and magnetic resonance imaging (MRI) lesion activity to track disease progression in patients with early multiple sclerosis (MS) may miss indicators of subclinical disease activity, with important implications...
Focusing solely on relapse rates and magnetic resonance imaging (MRI) lesion activity to track disease progression in patients with early multiple sclerosis (MS) may miss indicators of subclinical disease activity, with important implications...
