Shanlian Hu, MD, MSc, Professor of Health Economics, School of Public Health, Fudan University, Shanghai, China

ChatGPT has been widely tested and used in the global technology community in the past several months. The objective of this article is to use the Openai.com website asking about several questions. In the meantime, the author is also doing a content analysis to explore the application of artificial intelligence in pharmacoeconomic research and compares its advantages and disadvantages.

Question 1. The author first asked ChatGPT “What is the current state of international pharmacoeconomic research and applications?”

The answer is that pharmacoeconomics is a sub-discipline of health economics that evaluates the cost-effectiveness and value of pharmaceutical interventions. Here are some recent advancements in research and application of pharmacoeconomics from 2020-2022.

1. Use of real-world evidence: There has been an increasing interest in using real-world evidence (RWE) to support pharmacoeconomic evaluations. RWE refers to data collected from routine clinical practice, such as electronic health records and claims data. This approach can provide more robust data on the effectiveness and cost-effectiveness of drugs in real-world settings.
2. Cost-effectiveness of COVID-19 interventions: With the global pandemic, there has been a lot of research on cost-effectiveness of interventions, such as vaccines, therapies and diagnostic tests. This research has highlighted to show importance of considering the broader economic impact of the pandemic and the potential savings form preventing future outbreaks.
3. Value-based pricing: Value-based pricing is an approach to drug pricing that considers the value of a drug provides in terms of health outcomes. There has been a lot of interest in this approach in recent years, and several countries, including the United Kingdom and Germany, have implemented value-based pricing schemes.
4. Precision medicine: Precision medicine is an approach to healthcare that tailors treatment to an individual’s genetic makeup, lifestyle and environment. There has been a lot of research on the cost-effectiveness of precision medicine, and how it can improve patient outcomes while reducing healthcare costs.
5. Digital health interventions: Digital health interventions, such as mobile health Apps and telemedicine, are becoming more prevalent in healthcare. There has been a lot of research on the cost-effectiveness of these interventions, and how they can improve patient outcomes while reducing costs.
6. Drug pricing transparency: There has been increasing scrutiny of drug pricing in recent years, with many countries introducing measures to increase transparency in drug pricing. The includes requirements for drug manufacturers to disclose the cost of research and development, and the actual cost of producing a drug. Overall, the field of pharmacoeconomics continues to evolve and expand with new research and applications emerging every year.

The author generally agreed with the analytical framework provided by ChatGPT, but the contents need to be expanded. The author further conducted a literature retrieve from the Web of Science Database, the quantity of international pharmacoeconomics and Health Technology Assessment Literature from 2020 to 2022. The results show that the number of reports is relatively stable in the past three years. The number of HTA reports ranged from 7,000 to 9,000 per year, higher than the number of pharmacoeconomic reports, which is only less than 200 per year. The top five countries with the most reported literature were the United States, China, the United Kingdom, Italy, and Spain.

The authors also conducted a content analysis of 630 articles published in the ISPOR Journal Value in Health from 2020 to 2022 to further understand the research field of international pharmacoeconomic research. There are 13 categories, including: (1) comparative effectiveness research/HTA; (2) economic evaluation; (3) health policy analysis; (4) methodology; (5) patient reported outcomes; (6) preference-based assessment; (7) policy perspective; (8) systematic literature review. There are editorial sections, themed sections, ISPOR reports, brief reports, and commentary. The top five most frequently reported research areas are journal themed sections, economic evaluations, systematic literature reviews, preference-based evaluations and methodologies.

Question 2. As the method of preference-based evaluation is a new area that has been widely used in pharmacoeconomic research in recent years. The author asked ChatGPT “What is a preference-based assessment?”

The answer is an assessment that focuses on personal preferences and values, which are increasingly prevalent in our society, is a more appropriate way to assess a person's skills and abilities in terms of their needs and goals. Including personal strengths and weaknesses, overall goals, and interests. This allows us to identify areas where individuals may need additional support or guidance. By understanding their preferences and values, it provides the necessary resources and support to help them achieve their goals. From the analysis of the literatures, the author found that most contents of the reports were concentrated as follows: (1) attitude preference toward health risk; (2) family members and doctors' preference toward treatment; (3) heart disease patients' preference toward the value-based EQ-5D-5L scale; (4) different reasons for different age groups to evaluate the value of the EQ-5D scale; (5) health insurance payers' preference toward real-world evidence; (6) preference toward disease-prioritizing interventions; (7) health resource utilization of hospital patients; (8) population, residents and family members' preference for long-term care, etc.. Wu Jing et al. report a preference-based health utility scale CHROME has been developed in China as an example.

From the perspective of pharmacoeconomic research on COVID-19 infection, there are mainly systemic reviews and reports on the diagnosis, prevention and treatment of COVID-19, evaluation of the efficacy of public and social-distance interventions, cohort studies of the efficacy of therapies, such as cost-effectiveness analysis of Remdesivir and Paxlovid, safety and immunogenicity of Covid-19 vaccines, changes in the loss of QALY and quality-of-life utility index due to the deaths of COVID-19 infection, promotion to increase COVID-19 vaccination rates, public policy preferences, the real-world impact of drug transfer from hospital to community pharmacies, etc.

Question 3. The authors asked ChatGPT “What is the international experience with Orphan Drug Price Regulation?”.

Policy research on orphan drugs for rare diseases is an important topic in recent years. From a policy perspective, there are six areas: (1) definitions of rare and orphan drugs; (2) licensing, prioritization, and rapid approval; (3) safety and efficacy requirements; (4) price controls; and (5) encouraging market availability, payer subsidies/compensation/co-payments, financial support for cross-border services, compassionate drug use, tax exemption, free return of profits; (6) incentives for research and development, patent protection, market exclusivity, national plans and strategies. With the development of medical technology, the number of drugs to treat some rare diseases is increasing. because of the high price of drugs, many patients cannot afford them, so countries and regions began to take measures to control the price of orphan drugs.

Firstly, the European health organization (WHO) proposed the concept of “orphan drug price control”, which includes price control measures for specific drugs of certain diseases and allowing governments to adopt subsidies and technical agreements to lower the price of drugs for rare diseases.

Secondly, the government has also taken a number of steps to regulate orphan drug prices, including Federal and state drug price control laws and drug price subsidies from the Food and Drug Administration. In addition, the United States regulates orphan drug prices through measures such as promoting drug research and development and improving drug distribution, drug quality management and drug technology agreements. China has also enacted a series of laws and regulations to ensure that the price of medicines is reasonable.

The author summarized the international experience of orphan drug price control in recent years. For example, Germany has proposed a budget cap of €50 million for each active ingredient of orphan drug since 2016. If the budget is exceeded compared with the control drug should be evaluated within three months, the standards of cost-benefit and reimbursement should be adjusted after one year. In 2017, the UK set its rare-disease drug budget impact at 20 million GBP over three years. In the UK, where rare disease drugs are designed as high specific technology (HST), the criteria for ICER values can be raised to 100,000/QALY, and up to 300,000/QALY in life-cycle assessment. The Drug Economic Commission of the French Ministry of Health negotiates the price of orphan drugs with pharmaceutical companies, setting the price according to clinical added value, and referring to the sales volume, application, price and cost data from Ireland, Italy, Portugal, Spain and the European Union. Belgium promotes managed entry agreements (MEA). Austria sets maximum retail prices. Even Pakistan waives orphan drug pricing regulations. These are just some examples of different approaches.

Conclusion: The focus of pharmacoeconomics research and application is related to the political system, economic status, health care system and patients' needs in different countries. The aim is to improve the health of the population based on values and outcomes through health technology assessment. In recent years, the research and application of international pharmacoeconomics has developed rapidly. This paper confirms that ChatGDP can provide a framework of thought for the needed research topic. The content of the answer depends on the amount of information already retrieved in the database and the quality of the original data collected. If the need for more specific content in detail also requires the wisdom and judgment of researchers. At least under the present condition, the intelligence of researchers is much better than that of artificial intelligence.

The original article can be accessed here: https://mp.weixin.qq.com/s/D-m2RrvRj5yV5DCQkzB1Mw