Pharmacoeconomics and Pharmaceutical Economics Commons^™

Approvals, Utilization, And Cost Of Biologics For The Treatment Of Rheumatoid Arthritis, Roya Hosseini

Pharmaceutical Sciences (PhD) Dissertations

Introduction: RA treatment has been revolutionized by the development and approval of biologic disease-modifying antirheumatic drugs (bDMARDs). However, the approval status, utilization, and high cost of bDMARDs pose challenges in RA management.

Objectives: This dissertation evaluated the association between the utilization of bDMARDs and the sociodemographic characteristics of the RA patient population. It also identified the FDA approval status of bDMARDs and assessed the annual cost of bDMARDs used in RA treatment, evaluated the factors explaining annual cost at market entry.

Methods: In the retrospective analysis of Medical Expenditure Panel Survey (MEPS) data from 2010-2021, the population includes RA patients …

An Economic And Regulatory Analysis Of Gene Therapy Products Authorized By The Us Food And Drug Administration And The European Medicines Agency, Buthainah Ghanem

Pharmaceutical Sciences (PhD) Dissertations

Background: Gene therapy has recently emerged as an alternative for preventing and treating disease. Concerns have been expressed about the clinical value and the high cost of gene therapies.

Objective: This study assessed the authorization process of gene therapy products by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), evaluated the post-marketing safety signals reported to the FDA Adverse Event Reporting System (FAERS), and assessed the cost-effectiveness analysis of three gene therapies axicabtagene ciloleucel(axi-cel), lisocabtagene maraleucel (liso-cel), and tisagenlecleucel (tisa-cel).

Research Design: For aim 1; data was collected using regulatory information from the FDA and the …

Authorizations, Prices, And Labeling Differences Of Therapeutic Biologics Marketed In The United States And Saudi Arabia, Turki Altowairgi

Pharmaceutical Sciences (PhD) Dissertations

BACKGROUND: Since 1982, Saudi Arabia (SA) and the United States (US) have seen an increase in therapeutic biologics authorization. However, each country has regulatory regulations governing the approval of these cutting-edge drugs. The high cost of biologics financially burdens healthcare systems and delays patient access to these treatments. Sometimes a company selling the same medications in various countries will have conflicting information on drug labels.

OBJECTIVES: This study compared the characteristics of therapeutic biologics authorization by the Saudi Food and Drug Authority (SFDA) and Food and Drug Administration (FDA) as of December 31, 2021. The study also compared the US …

An Analysis Of Trends Of Generic Drugs And Biologic Prices In The U.S., Mohammed Jamjoom

Pharmaceutical Sciences (PhD) Dissertations

Introduction: Drug prices are a significant problem for the US healthcare system. Generic and biosimilar market competition reduces drug and biologic prices, results in substantial savings, and improves patient access and outcomes. As the costs of healthcare and prescription drugs continue to rise, the discussion about drug prices has taken a prominent place at the forefront of national discourse. This study assessed impacting trends in prices of generic, biosimilar, and reference products in the US. We also evaluated the effect of the Affordable Care Act (ACA) of 2010 on biologic prices.

Methods: We extracted a list of the new generic …

An Exploration Of Ambulatory Visits And Medications Prescribed For Patients Diagnosed With Lupus Erythematosus In The United States, Salena Marie Preciado

Pharmaceutical Sciences (PhD) Dissertations

This dissertation sought to understand the management and treatment of patients diagnosed with lupus erythematosus. To accomplish the objectives of this research, three studies were conducted. First, a systematic literature review was done to assess the current landscape of available clinical guidelines for disease treatment. This research then used the National Ambulatory Medical Care Survey data from 2006-2016 to explore real-world trends in medications prescribed, comorbidities and chronic conditions associated with lupus. Data is weighted to be nationally representative of the U.S. The third study explored sociological influences or nonclinical factors on the prescribing of medications for patients with lupus. …

Orphan Drugs: Designations, Approvals, And Prices In The Us, Hana Althobaiti

Pharmaceutical Sciences (PhD) Dissertations

Introduction. Rare disease drug approvals have accelerated significantly in recent years. Even with these advances, patients continue to face challenges in receiving treatment, with most rare diseases lacking any approved treatments. Moreover, even when a treatment is available, it does not always reach the patients who benefit from it.

Objectives. This dissertation assessed the characteristics of orphan drug designations and approvals and the factors associated with the time lag between orphan designation and approval in the US from 1983 to 2021. It also evaluated the treatment cost of orphan and non-orphan drugs and factors explaining the treatment cost of new …