Hu Shanlian: New Trends and Challenges in Global Pharmacoeconomics Research 2022

Emerging scientific frontiers and opportunities are driving the development of HTA (Health Technology Assessment) and HEOR (Health Economics and Outcomes Research), and the widespread application of real-world research methods will become a trend
.

Scientific knowledge spillovers (SKS) will promote the development of information sharing, cooperative research, and innovative technology

2022 is coming soon, let us sum up and look forward to the new trends in pharmacoeconomics research together!

0 1.
Rare diseases and gene therapy have become the forefront of research

0 1.
Rare diseases and gene therapy have become the forefront of research

In November 2021, Europe held the 2021 ISPOR Annual Conference, with "Emerging Frontiers and Opportunities: Special Populations and Technologies" as the theme of the conference, showing the new trends, new developments and new methods of pharmacoeconomics research
.

In recent years, as the world has paid more and more attention to the research and development of rare disease treatment drugs, digital medicine and personalized medicine have been widely promoted, and cell and gene therapies have emerged.

0 2.
HEOR priority research ten issues

0 2.
HEOR priority research ten issues

Recently, the Global Pharmaceutical Economics and Results Research Association conducted an expert consultation survey on the future global HEOR trend
.

The survey involved 67 ISPOR branch organizations in Asia, Africa, the Middle East, Europe, and South America.

The top 10 focuses are: ①Economic evaluation/HEOR methodology; ②Health system management, financing and reform; ③Health technology assessment; ④Real world data/evidence; ⑤Pharmaceutical prices and compensation issues; ⑥Value-based Health care; ⑦Participation of different interest groups; ⑧Digital health; ⑨Global health coverage; ⑩Precision and personalized medicine
.

0 3.
Different areas have different priorities for HTA development

0 3.
Different areas have different priorities for HTA development

It should be pointed out that the priorities of HTA development in different regions of the world are different
.

Experts in the Asia-Pacific region care about real-world data, digital medicine, and precision medicine
.

The Latin American region is concerned with value-based health care (VBHC) and global health coverage (UHC)

0 4.
HEOR research methods need to adapt to the new core

0 4.
HEOR research methods need to adapt to the new core

In the context of the COVID-9 pandemic and disruptive technological innovation (disruptive technological innovation), HEOR core methods need to adapt to real-world research as an important future direction
.

In addition, the estimation of the quality of life and health utility of children and other special populations, the discussion of ICER thresholds, the choice of reference drugs, the opportunity cost and the willingness to pay to society and individuals, the development and revision of national pharmacoeconomic guidelines, etc.
, have become The focus of future research
.

Economic evaluation and HEOR methodology will change: In addition to COVID-19-related research (including the role of HEOR in the pandemic, treatment cost-effect analysis), the research focus will be on opportunity costs and willingness to pay (social and personal), children’s The estimation of quality of life and utility weights, and the development of pharmacoeconomic evaluation guidelines
.

0 5.
Health technology assessment faces new challenges

0 5.
Health technology assessment faces new challenges

The main new challenges facing current health technology assessment are:

①How to conduct health technology assessment related to COVID-19 treatment? How to quickly evaluate in the absence of sufficient clinical or cost-effective data (especially single-arm trials)?

②How to develop a hospital-based HTA (hospital-based HTA)? How to conduct HTA on various medical technology projects?

③How to ensure the transparency of the HTA evaluation process? How to establish a database of regional HTA reports to realize resource sharing?

④How can HTA play a greater role in reducing health costs?

⑤How to carry out health technology assessment and multi-dimensional decision analysis (MDCA) of medical devices?

0 6.
Real world data/evidence faces five difficulties

0 6.
Real world data/evidence faces five difficulties

①Utilization of patient data
.

Involving electronic medical records (EMRs), gene bank data, disease registration, and use-case specific relevance and quality assessment framework (UReQA)

② Availability of data
.

③Big data utilization and machine learning methods of HTA and economic evaluation
.

④The establishment of national real-world evidence guidelines
.

⑤Use real-world evidence to serve the legislation and compensation decision-making
.

Among them, UReQA is a tool used in the United States to evaluate the quality of real-world research commercial databases, focusing on the evaluation of the quality and relevance of retrospective research data
.

The framework consists of 5 steps: pre-assessment, data standardization, determination of the study cohort, validation of patient data, and benchmark requirements

Take tumors as an example
.

The pre-assessment involves the coverage of tumor types, geography and time, as well as patient privacy protection, and whether it meets application analysis

0 7.
Five main challenges of pricing and compensation

0 7.
Five main challenges of pricing and compensation

The main challenges facing the issue of pricing and compensation include: ①Pricing of essential drugs; ②Transparency of drug prices; ③Pricing policies in the COVID-19 pandemic era (for example, India and South Africa proposed to the WTO to cancel patent protection and global cooperation for therapeutic drugs ); ④ Pricing of vaccines; ⑤ New ways to improve patient access to innovative/highly priced technologies
.

0 8.
COVID-19 brings new opportunities and new challenges

0 8.
COVID-19 brings new opportunities and new challenges

The impact of COVID-19 on health, economy, and social economy requires comprehensive research and evaluation, including comparison of value frameworks, preventive interventions, innovative vaccines, and treatment methods
.

The new opportunities and challenges it brings mainly involve the following 4 aspects:

① Speed ​​VS quality
.

Health technology compensation issues related to the diagnosis and treatment of COVID-19, the accuracy of the diagnosis and the effectiveness of the treatment
.
Evidence-based policy recommendations need to be provided as soon as possible, especially ultra-rapid reviews and rolling reviews of vaccines
.

②The impact on the economy
.
The COVID-19 epidemic has caused economic recession in various countries, such as the financial burden caused by city closures, business closures, economic stimulus policies, and materials needed to fight the epidemic
.

③ Speed ​​up the approval process of health technology, including conditional approval of diagnostic reagents, therapeutic drugs, vaccines and breakthrough technologies
.

④Vaccine pricing issues
.
The efficacy and protection rate of various vaccines against different mutant strains are worth studying
.
The research on the second and third generation COVID-19 vaccines needs to be encouraged
.
It is proposed to consider benefit-based advance market commitment (BBAMC), and determine the value-based vaccine price through HTA
.
Poor countries consider only paying a discounted low price (tail-price)
.
Through low-cost or invaluable technology transfer, protection of intellectual property rights, and encouragement of competition, we ensure that all people have fair vaccination opportunities
.