Summary
First‐line treatments for classical Hodgkin lymphoma (
HL
) include
ABVD
(adriamycin, bleomycin, vinblastine, dacarbazine) and
BEACOPP
escalated
(escalated dose bleomycin, etoposide, adriamycin, cyclophosphamide, vincristine, procarbazine, prednisone). To further improve overall outcomes, positron emission tomography‐driven strategies and
ABVD
or
BEACOPP
variants incorporating the antibody‐drug conjugate brentuximab vedotin (
BV
) or anti‐
PD
1 antibodies are under investigation in advanced‐stage patients. The present study aimed to elicit preferences for attributes associated with
ABVD
,
BEACOPP
escalated
and
BV
‐
AVD
(
BV
, adriamycin, vinblastine and dacarbazine) among patients and physicians. Cross‐sectional online discrete choice experiments were administered to
HL
patients (
n
= 381) and haematologists/oncologists (
n
= 357) in France, Germany and the United Kingdom. Included attributes were progression‐free survival (
PFS
), overall survival (
OS
), and the risk of neuropathy, lung damage, infertility and hospitalisation due to adverse events. Whereas 5‐year
PFS
and
OS
were the most important treatment attributes to patients, the relative importance of each attribute and preference weights for each level varied among physicians according to the description of the hypothetical patient for whom treatment was recommended.
PFS
and
OS
most strongly influenced physicians’ recommendations when considering young female patients who did not want children or young male patients. Infertility was more important to physicians’ treatment decision than
PFS
when considering young women with unknown fertility preferences, whereas hospitalisations due to adverse events played the largest role in treatment decisions for older patients.
This study identified challenges and motivators influencing advanced NSCLC patients' decisions to accept or refuse therapy, as well as patient and disease characteristics associated with physician's treatment recommendations for MT.
Background: Hemophilia A is a factor VIII deficiency, associated with spontaneous, recurrent bleeding episodes. This may lead to comorbidities such as arthropathy and joint replacement, which contribute to morbidity and increased health care expenditure. Recombinant factor VIII Fc fusion protein (rFVIIIFc), a prolonged half-life factor therapy, requires fewer infusions, resulting in reduced treatment burden. Objective: Use a budget impact analysis to assess the potential economic impact of introducing rFVIIIFc to a formulary from the perspective of a private payer in the United States. Methods: The budget impact model was developed to estimate the potential economic impact of adding rFVIIIFc to a private payer formulary across a 2-year time period. The eligible patient population consisted of inhibitor-free adults with severe hemophilia A, receiving recombinant-based episodic or prophylaxis treatment regimens. Patients were assumed to switch from conventional recombinant factor treatment to rFVIIIFc. Only medication costs were included in the model. Results: The introduction of rFVIIIFc is estimated to have a budget impact of 1.4% ($0.12 per member per month) across 2 years for a private payer population of 1,000,000 (estimated 19.7 individuals receiving treatment for hemophilia A). The introduction of rFVIIIFc is estimated to prevent 124 bleeds across 2 years at a cost of $1891 per bleed avoided. Conclusions: Hemophilia A is a rare disease with a low prevalence; therefore, the overall cost to society of introducing rFVIIIFc is small. Considerations for comprehensively assessing the budget impact of introducing rFVIIIFc should include episodic and prophylaxis regimens, bleed avoidance, and annual factor consumption required under alternative scenarios.
As therapies are developed for rare disorders, challenges of early diagnosis become particularly relevant. This article focuses on clinical recognition of mucopolysaccharidoses (MPS), a group of rare genetic diseases related to abnormalities in lysosomal function. As quality of outcomes with current therapies is impacted by timing of intervention, minimizing time to diagnosis is critical. The objective of this study was to characterize how, when, and to whom patients with MPS first present and develop tools to stimulate earlier recognition of MPS. A tripartite approach was used, including a systematic literature review yielding 194 studies, an online physician survey completed by 209 physicians who described
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