Quality of life as an endpoint

Quality of life (QoL) assessment adds useful information to the efficacy and safety data obtained in clinical studies. It contributes to the evaluation of different treatments and identifies patients who might benefit from supportive interventions. QoL data can be used to inform policy and resource allocation, reveal benefits to patients despite objective toxicity, and be of prognostic value. Evaluation of patient’s QoL might help to determine the suitable moment to start specific palliative interventions.

Clinical trial endpoint selection

Commonly used endpoints are based on survival, tumor response, and symptom assessment. Historically, overall survival (OS) has been viewed as the most effective measure as it addresses the biology of the tumor and the natural history of disease. PFS possesses significance because it assesses tumor shrinkage and stabilization of disease.

Phase II trials

Phase II studies provide initial assessment of efficacy in a more homogeneous patient population. Screening out of ineffective drugs is an objective here, as is the identification of promising new agents for future evaluation. The activity of a compound is assessed in a given tumor type, which allows for selection of tumor types for further study. Also, safety (types and incidence of AEs) is further defined in a specific patient population/ disease setting.

Phase I trials

Phase I trials consist of the dose escalation portion and the cohort expansion portion. The dose escalation part is dedicated to assessment of PK, safety and MTD. This segment usually encompasses only very few patients for each of several dose levels. In the expansion phase, a greater number of patients is recruited into one or two selected dose levels with the purpose of decreasing confidence intervals.

Preface – Part II

Due to the innovations in the field of oncological treatment that have profoundly changed our perception of modern cancer therapy, the design of clinical trials had to be modified appropriately with the purpose of allowing for the assessment of the distinct effects of new drugs. This means that long-standing paradigms had to be abandoned, and new principles took their place.

臨床試験の倫理的考慮および治験責任医師の責任

倫理は、良好な行為を支持する倫理上の原理として最も良いものとみなされ得る。一方、これが定義される方法は広い解釈がなされ、社会規範および宗教などの要因に依存する。ミレトスのタレス(紀元前624年~紀元前546年)は同時代の人に他人の行為を非難することを回避するように勧めた、そしてこの概念に類似した表現は道徳哲学中のあらゆる範囲にあふれている。医学では、無危害原則の概念(すなわち、患者を傷つけない)が医師の行為の柱として確立されている。

バイオマーカーに基づく臨床試験:試験のデザインおよび規制上の要件

分子診断の分野では最近の開発により、かなりの生物学的な違いが特定の解剖学的サブタイプの中でさえ存在していることから、癌自体としての存在はないことが明白になった。ゲノム特性はより良好に腫瘍のサブタイプを定義することに寄与するだけでなく、これらのサブタイプを標的とする機会も提供している。このため、これは有効性と認容性に関して、従来の治療を上回る治療的なアプローチの開発につながり得る。

序文 – Medical Education Clinical Trials Part I

バイオマーカーおよび腫瘍試験に関連したこの特別号では、国際的な専門家が自らの成功結果のデザイン、運営および実施を臨床診療に関連付ける最も重要な要素についての考え方を提供しています。 Silvia Novello博士と私自身は、癌の基礎をなす生物学に関連したバイオマーカーの同定および使用に基づいて、患者の臨床ケアにおける重要な改善を達成する際の新たな楽観主義がどのように存在してきたかを見直しています。一方、そのようなバイオマ ーカーの同定および使用には、それらが有効かつ役立つことを確認するための一連の手順に従うことが必要です。

Ethical considerations in clinical trials, and responsibilities of a principal investigator

Ethics can best be thought of as the moral principles that underpin good behavior. However, how this is defined is open to wide interpretation, and depends on factors such as societal norms and religion. Thales of Miletus (624 BC – 546 BC) advised his contemporaries to avoid doing what they would blame others for doing, and similar expressions of this concept abound throughout a range of moral philosophies. In medicine, the concept of non-maleficence (i.e., not harming the patient) is established as a pillar of physician behavior.

Biomarker-based clinical trials: study design and regulatory requirements

Due to the recent developments in the field of molecular diagnosis, it has become evident that there is no such entity as cancer per se, as there are considerable biological differences even within a particular anatomical subtype. Genomic characteristics do not only help to better define tumor subtypes, but frequently they provide the opportunity to target these subtypes as well.

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