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Prescription, Nonprescription and Orphan Drugs01:02

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Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
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Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
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Biologics, derived from living sources such as humans, animals, or microorganisms, represent a significant category of pharmaceuticals. These complex molecules, developed through advanced biotechnological methods or purified from natural sources, include essential medical treatments like insulin and growth hormones. The complexity of biologics arises from their large molecular structures and the intricate processes required for their production, making them distinct from conventional...
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Product specifications define the acceptable quality of a pharmaceutical product by ensuring identity, purity, potency, and strength. These specifications serve as benchmarks during development, manufacturing, and post-approval quality control. Clinically relevant specifications are particularly important because they directly relate to a drug's safety and efficacy in clinical use.Dissolution studies are critical biopharmaceutic tools that link in vitro behavior to in vivo performance. They...
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FDA Approved Drugs: Changes to Approved Drugs01:26

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Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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Saving orphan drug legislations: misconceptions and clarifications.

Hanna I Hyry1, Timothy M Cox1, Jonathan C P Roos1

  • 1a Department of Medicine , University of Cambridge , Cambridge , United Kingdom.

Expert Review of Pharmacoeconomics & Outcomes Research
|January 16, 2016
PubMed
Summary
This summary is machine-generated.

Orphan drug sales are rapidly increasing, posing sustainability challenges for patients and healthcare systems due to high costs. This analysis examines orphan drug pricing and proposes legislative solutions.

Keywords:
Gaucherlawmonopolyorphan diseaseorphan drugprice settingpricingreimbursement

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Area of Science:

  • Pharmacoeconomics
  • Health Policy
  • Drug Development

Background:

  • Orphan drug sales are experiencing significant growth, projected to reach $176 billion annually by 2020.
  • Orphan drugs constitute nearly 15% of global prescription revenue, growing at 10.5% annually, outpacing the overall market.
  • The high cost of orphan drugs, up to 19 times higher than conventional medications, creates access barriers for patients, particularly children.

Purpose of the Study:

  • To investigate the factors contributing to the rise in orphan drug designations.
  • To analyze the pricing mechanisms and regulatory frameworks for orphan drugs.
  • To propose modifications to existing legislation to ensure its long-term viability and improve drug accessibility.

Main Methods:

  • Review of market trends and sales data for orphan drugs.
  • Analysis of pricing strategies and regulatory policies governing orphan drug development and sales.
  • Examination of proposed legislative reforms and their potential impact on drug accessibility and healthcare costs.

Main Results:

  • Orphan drug market growth significantly exceeds that of the general prescription market.
  • Disparities in pricing create substantial financial burdens for patients and healthcare systems.
  • Current trends risk jeopardizing the sustainability of orphan drug legislation.

Conclusions:

  • The escalating cost and market share of orphan drugs present a critical challenge to healthcare affordability and patient access.
  • Urgent consideration of regulatory and pricing reforms is necessary to ensure the continued success and accessibility of orphan drug therapies.
  • Proposed legislative changes aim to balance innovation incentives with the need for sustainable and equitable access to these vital medications.