Pharmaceutical Medicine最新文献

The global pharmaceutical landscape is undergoing a profound shift with the rise of biosimilars-biologic medical products that are highly similar to original biologics but manufactured by different companies. Biological therapies encompass a range of therapies, including hormones such as insulin and growth hormone, vaccines, erythropoietin (EPO), monoclonal antibodies (mAbs), antibody-drug conjugates (ADCs), and bispecific antibodies (BsAbs), etc. As patents for many blockbuster biologic drugs expire, biosimilars provide an opportunity to improve patient access to life-saving treatments while potentially lowering healthcare costs, especially in regions with high unmet medical needs. However, the development and integration of biosimilars into mainstream healthcare comes with various challenges, including regulatory approval processes, ensuring quality, potency, efficacy, immunogenicity, safety, and overcoming market resistance. In this article, we will explore the development of biosimilars in-depth, their approval pathways, benefits, and the hurdles in achieving widespread adoption.

The medicines development workforce has grown to be a multidisciplinary, interprofessional global body of broadly qualified graduate professionals working with and around the biopharmaceutical industry. To satisfy their demands of fitness for work, they must meet the changes and challenges of a rapidly developing technological and competitive field. This has generated a need for a harmonised, mutually recognised vocational programme of competencies and capabilities, based on an academic foundation, which for the adult learners in medicines development goes above and beyond their original medical and scientific specialisms. This review proposes that professional competencies and capabilities developed in pharmaceutical medicine, a specialty concerned with the discovery, development, evaluation, registration, monitoring, and commercialisation of medicines and medical products, provides a comprehensive solution for the educational needs in medicines development. To justify this proposition, it summarises the 60-year evolution of education and training in pharmaceutical medicine. It demonstrates how expanding the pharmaceutical medicine syllabus has aligned it with almost every aspect of the development process of modern medicines. This perspective proposes a vision for the future where pharmaceutical medicine professional competencies and capabilities fill the educational void for the medicines development workforce. This is suggested to pave the career path that supports the professional identity of physicians, scientists, and other professionals working to develop medicines.

Pharmaceutical medicine professionals have to face many ethical problems during the entire life span of new medicines extending from animal studies to broad clinical practice. The primary aim of the general ethical principles governing research conducted in humans is to diminish the physical and psychological burdens of the participants in human drug studies but overlooks many additional social and ethical problems faced by medicine developers. These arise mainly at the interface connecting the profit-oriented pharmaceutical industry and the healthcare-centered medical profession cooperating in medicines development. In 2002, the International Federation of Associations of Pharmaceutical Physicians and Pharmaceutical Medicine developed the International Code of Ethical Conduct for Pharmaceutical Physicians for providing ethical advice for their members to manage the frequently competitive goals characteristic for their specialty. The ethical framework compiled by the International Federation of Associations of Pharmaceutical Physicians and Pharmaceutical Medicine serves its members by presenting morally acceptable or inacceptable behaviors in frequently encountered controversies arising from competing industrial and healthcare interests in medicines development. The authors selected this format to encourage reflection and debate for finding optimal moral conclusions in specific issues. Many recent examples of serious scientific-ethical misconduct, such as the oxycodone tragedy, the recommendations of unproven useless occasionally dangerous therapies during the coronavirus disease 2019 pandemic, and the withdrawal of many papers containing non-reproducible results, contributed to the increasing loss of trust by the public in science including pharmaceutical medicine. We are convinced that the ethical guidance developed by the International Federation of Associations of Pharmaceutical Physicians and Pharmaceutical Medicine will encourage its members to reflect intensively on optimal ethical behavior in drug development for strengthening the trust of society in innovative new medicines. Finally, considering the increasingly active participation of non-medically trained scientists in producing and applying complex biological medicines, distant monitoring methods coupled together with artificial intelligence technology in innovative clinical trials, the Ethics Working Group recommended already in 2017 measures to optimize their smooth cooperation and underlined their joint ethical responsibilities in guarding the safety and human dignity of trial participants.

Pharmaceutical Medicine (PM) was defined by its founding pharmaceutical physicians as a "medical/scientific discipline concerned with the discovery, development, evaluation, registration, monitoring, and medical aspects of the commercialization of medicines, for the benefit and the health of the community." Conceived as a medical specialty, the discipline encompassed several health-related professions and occupations, such as medicine, pharmacy, clinical pharmacology, drug safety and pharmacovigilance, pharmaceutical sciences, biology, health economics and others. Historically, non-medically qualified medicines development scientists from health-related disciplines have gradually grown into the medical roles within pharmaceutical organizations, regulatory agencies, and contract research organizations and today physicians and non-physicians lead drug development groups. Medicines Development Sciences (MDS) has been accepted as an alternative name. The practice of the disciplines involved in PM/MDS has evolved from an occupation to be accepted as a distinct profession, by meeting well established criteria. Despite thousands of health professionals participating today in medicines development, their awareness of the existence of this discipline, and its evolution from occupation to profession is low. The lack of professional identity and a clear career path have been identified as key factors limiting the advancement of this profession. This position paper aims to consolidate PM/MDS as an advanced profession in health sciences and propose a career path that is envisioned and championed by educational standards setting organizations like the International Federation of Association of Pharmaceutical Physicians and Pharmaceutical Medicine (IFAPP), PharmaTrain, and Global Medicines Development Professionals (GMDP) Academy with education and training as pillars for its achievement.

Medicines development has dramatically transformed in the preceding decades. It has evolved from a task undertaken by a small team to a complex series of activities, involving several functions and qualified professionals, across multiple, interrelated, scientific disciplines, worldwide. Conceptualized as a medical specialty, concerned with the research, development, and monitoring of medicines, and spearheaded largely by pharmaceutical physicians, the discipline has extended to embrace non-medically qualified scientists progressively taking on traditional roles within the medicines development ambit. There is expanded engagement of professional backgrounds. Each individual function contributes unique expertise and skills, but they all share a sense of identity, a collective commitment, and a common goal of improving human health and well-being, through innovative treatments. At the same time, milestone advancements in the research and development environment, in healthcare delivery, the regulatory ethos, data sharing with greater transparency, the exponential digitalization, and the emphasis on patient outcomes have imposed greater accountability in systems and processes, and across all global stakeholders. Increasingly, this demands a joint purpose, fostering continuous learning, and engendering a professional identity within the medicines development community, a coinage gaining incremental affirmation as equivalent to pharmaceutical medicine. The aligned set of related competencies and capabilities across the multi-professional teams involved, the sense of identifying concurrently across different, albeit complementary, communities of practice, the obligation of ongoing specialized knowledge training, the mandated enterprise-wide codes of ethics and moral conduct, and the intertwined career paths within the larger business organizational construct of academic institutions, regulatory agencies, and the biopharmaceutical industry-all profess to meet the criteria, and merit medicines development/pharmaceutical medicine be acknowledged, regarded, and recognized as a distinct profession of stature, in its own standing. The concept of pharmaceutical medicine has been floated since 1957, and has received considerable attention in scientific literature over the years. Yet, the discipline struggles to be universally understood and appreciated. This perspective scrutinizes related engendering influences and hindering elements, while advocating the furtherance of medicines development from a traditional occupation, emerging as a vocation, and transforming into a mature profession, with the vision and purpose of advancing better medicines for improved health outcomes for people, globally.

Background: Understanding parameters such as cost and duration, which reflect the scale of clinical trials, is essential for government agencies to formulate pharmaceutical policies and for pharmaceutical companies to strategically allocate resources. As most studies on clinical trial costs and durations have primarily focused on pharmaceutical companies developing drugs in the USA, understanding actual trial costs and durations for Japanese pharmaceutical companies engaged in global development or development within their own countries remains challenging.

Objectives: This study aimed to clarify the actual phase-specific costs and durations of clinical trials conducted by leading Japanese pharmaceutical companies and to identify the factors influencing these parameters.

Methods: We conducted a questionnaire-based survey among 22 pharmaceutical companies headquartered in Japan. Respondents from each company retrospectively identified clinical trials for new drug applications or for expanding approved disease indications, conducted between 2012 and 2022, providing cost and duration data of each clinical trial. We calculated summary statistics for costs and durations by phase and conducted an exploratory analysis of the variables influencing these parameters.

Results: In total, 631 projects with clinical trials conducted by these companies were analyzed. The median costs in the analysis population were 859 million Japanese yen (JPY) for Phase 1, 1,739 million JPY for Phase 2, and 4,183 million JPY for Phase 3. For reference, the median annual average exchange rates during the study period were 109 JPY per US dollar (USD) and 130 JPY per euro (EUR). The median durations were 20 months for Phase 1, 29 months for Phase 2, and 31 months for Phase 3. For projects targeting approval only in Japan, the median Phase 3 cost was 3,050 million JPY, while for projects targeting approval involving the USA or Europe, it was 8,083 million JPY.

Conclusions: This study quantifies the costs and durations incurred by Japanese pharmaceutical companies in conducting clinical trials, revealing the high costs associated with projects targeting approval involving the USA or Europe. The findings highlight the substantial investments required by the Japanese pharmaceutical industry to bring new drugs to the global market.