Related Experiment Video
Updated: Jun 25, 2026
05:10
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
What Drives Public Preference for Rare Drugs Coverage in China? Insights From a Multi-Center Discrete Choice
Ya'nan Wu1,2,3,4, Jingdan Chen1,2,3,4, Jiachen Shao1,2,3,4
1Department of Social Medicine and Health Management, Cheeloo College of Medicine, School of Public Health, Shandong University, Jinan, China.
Summary
Public preferences for orphan drug reimbursement in China show health gains are key, but equity and affordability also matter. Policymakers should balance these values for fair rare disease resource allocation.
Area of Science:
- Health Economics
- Public Health Policy
- Rare Disease Research
Background:
- Conventional cost-effectiveness frameworks struggle with orphan drug reimbursement due to broader societal values.
- Investigating public preferences is crucial for equitable access to rare disease treatments in China.
Purpose of the Study:
- To explore public preferences for including orphan drugs in China's Basic Medical Insurance (BMI).
- To understand societal values influencing reimbursement decisions for rare disease treatments.
Main Methods:
- A multi-center discrete choice experiment (DCE) involving 622 participants across four Chinese regions.
- Analysis using mixed logit and latent class models to estimate relative importance, willingness to pay (WTP), and preference heterogeneity.
- Inclusion of public input in DCE design to ensure clarity and relevance.
Main Results:
- Health gains were the most significant factor, followed by BMI financing increases and untreated quality of life.
- The public supported orphan drug reimbursement when treatments offered substantial health gains and lacked alternatives.
- Annual willingness to pay (WTP) was ¥1.225 per Quality-Adjusted Life Year (QALY) gain.
- Two distinct preference groups were identified: 'life-saving' and 'pragmatist'.
Conclusions:
- Public preferences for orphan drugs balance health gains with equity, disease severity, and affordability.
- Integrating multidimensional social values is essential for legitimate and fair resource allocation for rare diseases.
- Policymakers must consider these diverse public values in reimbursement decisions.
Related Concept Videos
01:15Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions
PK–PD modeling has significantly influenced FDA regulatory decisions, particularly drug approval, dosage optimization, and labeling. These models integrate pharmacokinetics (PK) and pharmacodynamics (PD) to predict drug behavior and effects, aiding in optimizing dosing regimens and enhancing the probability of clinical trial success.One notable example is Nesiritide (Natrecor®), a recombinant human brain natriuretic peptide for treating acute decompensated congestive heart failure (CHF).
01:02Prescription, Nonprescription and Orphan Drugs
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.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
01:20Bioequivalence Experimental Study Designs: Completely Randomized and Randomized Block Designs
Bioequivalence experimental study designs are crucial methodologies used in evaluating and comparing the bioavailability of different drug products. These designs are categorized into various types: completely randomized, randomized block, repeated measures, cross and carry-over, and Latin square designs.Completely randomized designs involve randomly allocating treatments to all subjects participating in the experiment. This allocation is achieved by assigning unique random numbers to subjects...
01:01Dosage Regimens: Partial Pharmacokinetic Parameters
It is not uncommon for complete drug pharmacokinetic profiles to remain elusive in pharmacokinetics. This necessitates certain educated assumptions by pharmacokineticists to determine appropriate dosage regimens without comprehensive pharmacokinetic data from animal or human studies. One prevalent assumption is setting the bioavailability factor, denoted as F, to 1 or 100%. This assumption caters to the scenario where a drug doesn't achieve full systemic absorption, resulting in the patient...
01:25Drug Regulation
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...