Related Experiment Video
Updated: Nov 29, 2025
08:59
An Intestine/Liver Microphysiological System for Drug Pharmacokinetic and Toxicological Assessment
Published on: December 3, 2020
8.3K
Payer and Implementation Challenges with Advanced Therapy Medicinal Products (ATMPs)
John Spoors1,2, Alec Miners3, John Cairns3
1London School of Hygiene and Tropical Medicine, London, UK. john.spoors@nhs.net.
Summary
Advanced therapy medicinal products (ATMPs) present implementation challenges and financial uncertainties for healthcare systems. Careful consideration of health economic principles is crucial when accelerating access to these novel one-off therapies.
Area of Science:
- Health Economics
- Pharmaceutical Policy
- Biotechnology
Background:
- Advanced therapy medicinal products (ATMPs) are rapidly entering the market, creating new challenges for healthcare systems.
- A significant gap exists between regulatory requirements and payer expectations for ATMPs.
- The limited evidence base for ATMPs increases clinical and financial uncertainties.
Purpose of the Study:
- To analyze the implementation challenges and health economic considerations of Advanced therapy medicinal products (ATMPs).
- To highlight the uncertainties associated with ATMPs at the time of evaluation.
- To discuss the potential impact of ATMPs on healthcare systems' net health gain and opportunity costs.
Main Methods:
- Review of current literature on ATMP implementation.
- Analysis of health economic principles relevant to novel therapies.
- Evaluation of uncertainties in clinical and financial aspects of ATMPs.
Main Results:
- ATMPs introduce significant clinical and financial uncertainties due to limited evidence.
- Implementation pressures can exacerbate these uncertainties, affecting the value proposition of ATMPs.
- ATMPs may reduce net health gain for healthcare systems, necessitating careful evaluation of opportunity costs.
Conclusions:
- While ATMPs hold transformative potential for clinical care, their implementation requires caution.
- Key health economic principles must be applied to manage the uncertainties associated with ATMPs.
- A balanced approach is needed to accelerate access while ensuring value and sustainability in healthcare systems.
Related Concept Videos
01:28Drug Products: Biologics, Biosimilars and Interchangeables
126
Body: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...
126
01:29Therapeutic Drug Monitoring: Affecting Factors
95
Therapeutic Drug Monitoring (TDM) is the clinical practice of measuring specific drug levels in a patient's blood or body tissues to manage and optimize therapy. TDM is crucial for drugs with narrow therapeutic windows, like warfarin and phenytoin, where incorrect doses can lead to treatment failure or severe side effects. This monitoring ensures the dosage administered is within a safe and effective range. The factors affecting therapeutic drug monitoring include:Patient-Specific Factors:a.
95
01:27Pharmaceutical Alternatives: Polymorphic Form-Related and Particle Size-Related Therapeutic Nonequivalence
81
Changes in polymorphic forms can significantly influence the bioavailability of poorly soluble drugs. Although the FDA defines pharmaceutical equivalence based on having the same active ingredient, dosage form, and route of administration, it does not automatically disqualify products with different polymorphic forms. This means two products with different polymorphs can still be deemed pharmaceutically equivalent. However, polymorphic differences can affect properties like wettability,...
81
01:26FDA Approved Drugs: Changes to Approved Drugs
89
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,...
89
01:18Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
297
Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
297
01:18Formulation and Manufacturing Process: Physical Attributes of Generic Tablets and Capsules
90
Bioequivalence in generic drugs, such as tablets and capsules, refers to their pharmaceutical equivalence to the brand-name counterparts. However, for therapeutic equivalence, manufacturers must also consider physical attributes like size, shape, and weight (FDA Guidance for Industry, December 2003). Discrepancies in these aspects could impact patient compliance and cause medication errors. For instance, swallowing difficulties, often experienced with larger tablets or capsules, can lead to...
90