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"Patient-centricity" leads a new direction in drug discovery: global progress and trend outlook

 Last Update: 2022-11-15
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In recent years, with the increase in the complexity of drug research and development and the intensification of target accumulation, more and more developers have realized that limiting their R&D focus to the data performance of a certain target can no longer adapt to the drastically changing market demand.
Developers should focus on diseases and patients, and pay attention to the role of patients in the whole process of drug development, in order to ensure that drugs can accurately respond to real clinical needs
.
Patients' participation in drug research and development not only helps to improve patient compliance and R&D success rate, but even helps to achieve better market share after the product is launched, realizes a win-win situation for patients, developers, and pharmaceutical enterprises, and comprehensively promotes the development of
the pharmaceutical innovation industry at the R&D end, supply side and use end.
Concepts such as "patient-centered" and "encouraging patients to participate in the whole cycle of drug development" are increasingly valued
by innovative drug developers.
In Europe and the United States and other leading countries in pharmaceutical innovation, a complete set of guidance and principle documents have long been formed and gradually implemented
.
Since the beginning of this year, China's drug regulatory authorities have also followed this trend and intensively issued a series of documents, and the era of "patient-centered" drug research and development in China has begun
.

First, first: international experience of patients involved in drug development

First: International experience of patient involvement in drug discovery

FDA: Guidelines for Patient-Centered Drug Development

In 2012, the U.
S.
Food and Drug Administration (FDA) spearheaded the patient-focused program drug development，PFDD）
。 The 21st Century Cures Act, enacted in 2016 With the support of the Act), PFDD became a priority
for the FDA.
Currently, FDA is developing four patient-centered approach guidance documents1 that cover the development process from data acquisition, protocol determination, process
advancement, and outcome assessment.
According to the progress, the first and second documents have been finalized, and the third draft for comments has also been released
.

Patient-centered drug development programs

Guideline 1 Methods for collecting accurate and representative patient experience data from the intended patient population

Guide 1

Guideline 2 identifies important methods for patient guidelines to staff and other stakeholders

Guide 2

Guideline 3 Identify what is important to patients and select, develop or modify appropriate clinical outcome assessment data methods

Guide 3

Guideline 4 How to integrate clinical outcome assessment into the endpoints of regulatory decision-making

Guide 4

In addition, the FDA released a "Patient-Centered Drug Development Roadmap.
"

Patient-centric drug discovery roadmap

Figure 1 FDA's pathway to patient-centered drug discoveryFigure 2

Through four guidelines and roadmaps for patient-centered drug discovery, the FDA identified a complete set of approaches
that correspond to the drug discovery process, from selecting patients, communicating symptoms and preferences, selecting appropriate evaluation methods, and gradually and eventually incorporating patient data into regulatory decisions.
FDA recommends that sponsors implement early and seek FDA's input throughout the medical product development process to ensure that clinical outcome assessment data is appropriate for the intended use environment
.

ICH: E8(R1) guidelines include patient participation in drug discovery

In 2017, ICH launched ICH The comprehensive update of the E8 guiding principles has added patient participation in drug development in E8(R1), and proposed the need to listen to patients' opinions and obtain views
from patients in the process of drug development.
ICH E8(R1) completed STEP 4 and passed 3
in October 2021.

ICH consultation with patients or patient organizations, including caregivers, is of great value to drug development, but it is noted that current methods for identifying, collecting, and analyzing patient-related data are not standard or uniform, and that a range of problems
arise when incorporating patient feedback into drug development or regulatory decision-making processes.
Hence ICH E8(R1) makes this clear:

During the study design phase, all actors involved in the study should inform the study quality creation process and encourage researchers to communicate with regulators as early as possible, especially when
new key elements of a study may affect quality.
In the important links of determining key quality factors, such as selecting key endpoint indicators, determining the time point of visits, and drafting informed consent forms, more attention needs to be paid to patient opinions to ensure the smooth implementation of the trial.

At the study design stage

At the operational practice stage, feasibility plans should fully consider the needs of the patient population, including regional differences in medical practice and patient population, and promote data retention and patient follow-up in a timely manner to ensure that these key factors inform study design to improve study quality
.

In the operational practice phase

EMA: Through the "Patient Preference" framework, the European Medicines Agency (EMA) published its Regulatory Science Strategy 2025 in March 2020, which highlights the need to strengthen patient relevance in evidence generation and explicitly include data on patient participation in clinical trials into regulatory decision-making
.
EMA will work with the Council for Medicinal Products for Human Use (CHMP) to improve patient data collection, and will also work with Health Technology Assessment (HTA) agencies to identify a mutually beneficial approach to the systematic use
of quality-of-life patient-reported outcomes (PROs) in drug development.

EMA: through the "Patient Preference" framework

In June, the EMA approved the Innovative Medicines Initiative.
IMI) Patient Preferences in Benefit–Risk Assessments during the Medical Product Lifecycle, PREFER) framework
.
Designed to assess when and how to incorporate patient preferences for benefits and risk into drug decisions4, the framework describes how drug and device sponsors can use patient preference studies to gauge patient views and gradually incorporate them into
regulatory decision-making processes.

The PREFERENCE recommendation consists of four main steps: stakeholder interviews, classification and assessment of preferred methods, identification and evaluation of psychological and educational characterization methods, and forward-looking case studies
.
The document is presented in eight subsections that determine how patient preference studies should be designed, executed, and used in decision-making
.

Table PREFERENCE for evaluation and use of patient preferences in the development of medicines5

2.
Follow-up: Relevant guiding principles that China is developing

Follow-up: Relevant guidelines are being developed in our country

In July this year, the Center for Drug Evaluation (CDE) of the State Medical Products Administration issued the Guidelines for General Consideration of Organizing Patient Participation in Drug R&D (Draft for Comments), which for the first time clarified patient participation in the whole life cycle of drug R&D for registration purposes and encouraged other clinical studies to refer to
them.

Figure 2 CDE "Guidelines for General Considerations for Organizing Patients to Participate in Drug R&D (Draft for Comments)"

In August, CDE issued three guidelines (draft for comments) related to patient-centered clinical trials in one day, providing corresponding guidance on patient-centered drug research and development in terms of regulation, marking the beginning
of the era of "patient-centric" drug research and development in China.

III.
Discussion: The key issue of Chinese patients' participation in drug research and development

Discussion: Key issues for patients to participate in drug research and development in China

Complete, true and standardized clinical data for patients to participate in drug research and development must be subject to strict data management
.
The authenticity, completeness and standardization of clinical trial data are the basis for the correct evaluation of the effectiveness and safety of research drugs, and the doubts of international institutions including the FDA on China's clinical data have also become a major bottleneck
restricting China's innovative drugs to go global.
In the process of clinical trials in which patients participate, there are still a large number of data or samples collected outside the research center, and there are still doubts
about whether their authenticity, completeness and standardization are ready for audit.
At the same time, the issue of data compliance involving patient privacy is also worth paying attention to, because relevant measures such as patient participation have not yet taken shape in the process of ethical review, and it is difficult to ensure that patients' opinions can be adopted and implemented, and the risk of
privacy leakage is avoided.

Complete, true and normative clinical data

How to incorporate patient opinions in the scientific supervision process of ICH Management Committee Chairman Theresa MULLIN HAS STATED THAT "PATIENT NEEDS ARE THE FOCUS OF THE ICH'S GUIDING PRINCIPLES, AND IN THE FUTURE IT WILL FOCUS ON DRAFTING DATA-DRIVEN GUIDELINES TO BETTER IDENTIFY POTENTIAL RISKS AND PROTECT PATIENT HEALTH"6
.
The inclusion of relevant data of patient participation in scientific supervision requires the mutual promotion and cooperation of a set of relevant guiding principles, increasing the interoperability between guidelines, forming a complete set of scientific supervision system, and constantly enriching and improving
in the process of practice.

How to incorporate patient opinions in the scientific supervision process

Individual patients with problems of patient screening and education and communication are often limited by problems such as lack of health knowledge and information, single way to participate in the trial process, and limited mastery of various resources, and cannot fully participate in the process of pharmaceutical research and development and better exercise their right to
speak.
Therefore, how to select patient representatives from the population, how to let the public understand the direction of clinical trials, and how to incorporate high-quality patient views into the endpoint have become core issues, which put forward more requirements
for patient education, patient organization construction, patient-family communication, and the development of related technical means.

Communication issues with patient screening and education

The FDA and EMA established the FDA-EMA Patient Engagement Group in 2016 to share best practices
for patient engagement in the drug regulatory lifecycle through strategic collaboration.
According to Pharma According to Intelligence's 2012-2017 data, the US and the European Union accounted for the highest proportion of patient-centric clinical trials, at 38% and 34%, respectively, compared to 5% in China7
.
China urgently needs to learn from the experience of pioneering countries, improve the construction of standards, promote scientific supervision and improve decision-making
.

International cooperation

4.
Prospect: new ideas, new practices, new breakthroughs

Prospect: new ideas, new practices, new breakthroughs

Patient participation in drug discovery from concept to practice requires the formulation and implementation of a series of principles and guidelines, as well as the innovation
of data collection, recording and presentation.
With the application of new technologies and means, the medical big data sharing platform, especially the digital tool platform for clinical research, is constantly being built, and patients, an important subject in clinical research, can participate in the research and development process in a better way and achieve deeper breakthroughs
in pharmaceutical innovation.

In specific practice, on the one hand, it is expected to further expand the patient-centered connotation in depth and encourage patients to participate extensively and deeply in the whole life cycle of
drug management.
For example, expand the patient population, cultivate patient tissues, and add patient voices in combination therapy, medication strategies, and companion diagnosis; On the other hand, it will expand more attempts in breadth, such as exploring the in-depth participation of patients in the research and development process of Chinese medicine
.

Patient-centered drug R&D has become an irreversible new development direction, and we look forward to further innovation in policy and practice to adapt to the latest changes in pharmaceutical R&D, dock unmet clinical needs, enhance the clinical value of pharmaceutical R&D, and realize the two-way development of
patient needs and industrial development.

Policy Research Center, China Association for the Promotion of Pharmaceutical Innovation

Author: Wu Qiong Wenqian Analyst wuqwq@phirda.
com

Review and proofreader: Zhang Zhijuan, Liu Fapeng

Resources:

1.
FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making（2022-6-29）

2.
FDA.
Roadmap to patient-focused outcome measurement in clinical trials .
(2014-06-01) .

2.
FDA.
Roadmap to patient-focused outcome measurement in clinical trials .
(2014-06-01) .
3.
ICH HARMONISED GUIDELINE GENERAL CONSIDERATIONS FOR CLINICALSTUDIESE8(R1) （2021-10-6）

3.
ICH HARMONISED GUIDELINE GENERAL CONSIDERATIONS FOR CLINICALSTUDIESE8(R1) （2021-10-6）

https://database.
ich.
org/sites/default/files/E8-R1_Guideline_Step4_2022_0204%20%281%29.
pdf

4.
PREFER Including the patient perspective （2022-4-19）

5.
PREFER Recommendations - Why, when and how to assess and use patient preferences in medical product decision-making（2022-4-19）

https://zenodo.
org/record/6491042#.
YtYHL3ZByUl

6.
"Cohesion of Patient-Centered Pharmaceutical Industry Synergy - ICH Boosts Higher Quality Development of Pharmaceutical Industry" China Pharmaceutical Food Network(2021-5-21)

http://test.
health-china.
com/c/2021-05-21/789907.
shtml

7.
The economist intelligence unit.
Patient-centric trials].
(2018-06-27)

https://druginnovation.
eiu.
com/patient-centric-trials/

8.
Xie Jing, Wang Huanling Patient-centered clinical trials: a new model for pharmaceutical research and development Journal of Union Medical College, Issue 6, 2021

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