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Disease surveillance is the systematic collection, analysis, and interpretation of health data essential to the planning, implementation, and evaluation of public health practice. This process integrates data dissemination to entities responsible for preventing and controlling disease, injury, and disability. Surveillance systems provide crucial information for action, helping public health authorities make informed decisions to manage and prevent outbreaks, ensure public safety, optimize...
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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 control governance involves the oversight and regulation of pharmaceuticals to ensure their safety and efficacy while preventing illegal drug use and trafficking. Regulatory bodies, including the US Food and Drug Administration (FDA) and the European Union's European Medicines Agency (EMA), play a central role in this process. These agencies evaluate the safety and efficacy of drugs before they can be marketed. They fund clinical trials and assess the benefits and risks associated with...
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Related Experiment Video

Updated: Jun 14, 2025

A Strategy to Identify de Novo Mutations in Common Disorders such as Autism and Schizophrenia
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Commentary: Which Principles Should Apply for a National Strategy on Rare Diseases?

Marc-AndrÉ Gagnon1

  • 1Associate Professor, School of Public Policy and Administration, Carleton University, Ottawa, ON.

Healthcare Policy = Politiques De Sante
|September 4, 2024
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Summary
This summary is machine-generated.

Federal funding for rare disease drugs should support development, not just purchasing. Outcomes-based risk-sharing agreements require robust institutional capacity for real-world evidence collection.

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Area of Science:

  • Health economics
  • Pharmaceutical policy
  • Rare disease research

Background:

  • Lexchin and Sirrs (2024) proposed principles for federal funding of expensive rare disease drugs.
  • These principles include outcomes-based risk-sharing agreements (OBRSAs) and evidence generation.

Purpose of the Study:

  • To question the scope of federal funding for rare disease drugs.
  • To explore whether funding should extend to drug development, not solely procurement.
  • To examine institutional requirements for OBRSAs to facilitate real-world evidence collection.

Main Methods:

  • Rejoinder analysis of existing principles.
  • Examination of funding mechanisms for pharmaceuticals.
  • Assessment of institutional capacity needs for evidence generation.

Main Results:

  • Federal funding for rare disease drugs may be limited if restricted to purchasing new medications.
  • Broader application of funding could encompass drug development, fostering innovation.
  • Successful implementation of OBRSAs necessitates significant institutional capabilities for collecting real-world data.

Conclusions:

  • Federal funding policies for rare disease drugs should consider supporting the entire drug lifecycle, including development.
  • Outcomes-based risk-sharing agreements require careful planning regarding the necessary infrastructure for real-world evidence gathering.