探討參與早期臨床試驗之受試者其試驗參與決策過程與經驗感受

Abstract

【背景】 癌症是國人常見的重要死亡原因之一，並且也是診斷後對生活品質衝擊高的疾病之一，因此在追求更好更新的抗癌治療過程，以及在促進人類生醫發展的過程中，有賴臨床試驗確立療效與安全性，其中第一期臨床試驗往往是最早與最關鍵的第一步，但新藥的效益與風險存在許多不確定性，而治療往往又刻不容緩，多重因素考量下，癌症病人如何在短時間內做出是否參加早期臨床試驗的決策，並在試驗進行過程中，權衡利益與風險，或是否符合當初預期，都尚未有足夠的文獻陳述。本研究目的為藉由訪談瞭解本土文化因素對於參與早期臨床試驗癌症患者的決策過程、經驗感受的影響歷程，並分析此經驗可幫助醫療人員瞭解如何提供適當的護理照顧，同時對於其他有相同處境者，對此也能有更深入的瞭解。 【方法】 本研究採用混和性研究設計，於某醫學中心早期臨床試驗病房與門診，以立意取樣方式透過醫師轉介，邀約個案參加本研究。量性資料收集透過個案的問卷填答，後以描述性及推論性統計分析結果；質性資料則透過一對一訪談後採詮釋學觀點理解個案經驗，質性資料之收集與分析同步進行，訪談逐字稿輸入後反覆分析及持續比較，並依循Colaizzi（1978）所建議的質性研究七個步驟進行，輔以運用質性分析軟體NVivo 12萃取臨床試驗決策過程與影響因素，以及參加試驗之經驗感受與關注之議題。 【結果】 本研究共完成32位個案問卷資料收集，其中24位個案同意接受深度訪談，每人分別完成1至2次一對一訪談。問卷結果發現參加臨床試驗的決策時間點有超過一半以上的病人自認是「立刻決定」(n=18位，56.2%)；家人對於個案參加早期臨床試驗的支持程度多為支持及非常支持，分別有15位及12位(46.9%及37.5%)；病人做決策時影響最大的角色人物為試驗相關醫師(n=13，40.6%)；在參加試驗決策過程自己與他人的決策影響分數分配，以總分為10分的分配尺度下，選擇「自己與他人各佔五分」最多(n=10，31.2%)；在參與早期臨床試驗的原因中，病人認定最主要原因是「希望有療效或可以把病治好(死馬當活馬醫)」(n=15，46.9%)，其次原因為聽從「醫師建議」(n=8，25%)；一半的病人(n=16，50 %)表達再次決定參與早期臨床試驗的可能性為「有可能」。質性訪談病人對參加早期臨床試驗的看法有「未知」、「希望、機會、有信心」、「害怕、實驗、白老鼠」及「促進醫學正向發展」，呈現又期待又怕受傷害的矛盾感受；決策過程的支持，普遍提到正向支持力量包含「家人」、「醫師」及「自身信念」與「宗教靈性力量」等；決策過程主要考量的因素偏重「治療效果」，包含「希望把病治好、死馬當活馬醫」、「沒有更好的治療、最後的選項」、「試試看是一個機會」，病人做決策時也願意相信他人建議，包括「家人決定」、「相信醫療人員建議」，參與早期臨床試驗的其他考量因素還有「經濟考量」；而參加早期臨床試驗的經驗感受亦是融合多種感受的複雜情緒存在，包含正向、中立及負向感受均有人提及，普遍經常被提及的經驗有「感受到疾病獲得控制」、「感受到有希望及心理舒緩踏實」、「感受到副作用少且生活品質好」以及「感受到試驗藥物伴隨副作用」。 【結論】 參與早期臨床試驗的決策過程有賴於資訊的尋求，建構相關認知及看法，同時輔以他人的支持，如「家人」及「醫師」，或由信念與靈性的力量，包含「自身信念」以及「宗教靈性力量」等，並且更新對於臨床試驗的瞭解，排除相關負面干擾後，考量目前疾病治療上困境，又希望可以治癒疾病，衡量「治療效果」，並且不斷藉由「醫療專業人員」，包含臨床研究護理師，的說明與建議，病人自己與家人仍需互相扶持及持續鞏固信心，是病人同意參加早期臨床試驗的複雜決策歷程。 「早期」臨床試驗的安全性及副作用雖是重要考量標準，但因癌症治療的進展仍未有完全有效的治癒方式，當癌症患者在面臨已無常規治療可選擇，卻又感受到迫切需要抗癌藥物來抗癌時，因此，癌症患者在臨床試驗的選擇上，會期待有治療效果及盡快投予用藥作為衡量標準，這也是臨床研究護理師需要多理解病人的決策矛盾及參與試驗的信念，特別是參加早期(第一期)臨床試驗者，須被清楚說明早期試驗的設計理念與相關照護。

【Background】 Cancer is one of the most common and important causes of death in the Chinese population, and it is also one of the diseases that have a high impact on the quality of life upon diagnosis. Therefore, the pursuit of advance in new treatment regions depends on clinical trials to establish efficacy and safety. Phase I clinical trials are often the earliest and the most critical first step. Although the treatment is often urgent for advanced cancer, there are many uncertainties regarding the benefits and risks of new drugs. There is no enough information about the decisions that the cancer patients make a decision to participate in an early phase clinical trial, and the process to balance the benefits and the risks in the early phase trials. The purposes of this research are to understand the decision making process of participating an early phase clinical trial and associated factors among cancer patients. In addition, the study is also to explore the experience and feelings of cancer patients in the early phase clinical trials. 【Method】 This research adopts a mixed-method design. The quantitative data comes from self-filling questionnaires. The qualitative part adopts a hermeneutics standpoint and using an intentional, purposive sampling for the participants’ recruitments. The data collection and analysis are carried out simultaneously. Qualitative interviews are conducted individually, the interviews are recorded, and the audio files are converted to verbatim drafts. Content analysis and continuous comparisons are used to analyze qualitative data. The data analysis method is mainly based on the seven steps suggested by Colaizzi (1978) and the software NVivo-12 is used to analyze the qualitative data. 【Results】 A total of 32 participants are recruited for questionnaires, among which 24 agreed to be one-on-one interviewed in depth for once or twice. Regarding the decision making process of participating the clinical trials, more than half of the patients (n=18, 56.2%) identified themselves making the decisions at an “immediately” manner, and family members being supportive (n=15, 46.9%) and very supportive (n=12, 37.5%). The most influential persons in the process of decision making for clinical trials were trial-related physicians (n=13，40.6%). At a 10-points scale, the patients though themselves and others had almost equal weight on the decision making process (n=10, 31.2%). Among the reasons for participating in the early clinical trials, the patient determined that the main reason was 'hope for efficacy or cure (try as a last resort)' (n=15, 46.9%), followed by “listening to physician’s advice' (n=8, 25%). Half of the patients believed that it is possible they would make the same decisions to participant the clinical trials in the future (n=16, 50%). Qualitative interview data reveal that patients' views on participating in early clinical trials were 'unknown', 'hope, opportunity, confidence', 'fear, experimentation, white mice' and 'promoting the positive development of medicine', rendering contradictory feelings of anticipation and fear of harm. Support for the decision-making process generally refers to positive support forces such as 'family', 'physician' and 'self-belief' as well as 'religious spiritual power.' The main considerations in the decision-making process are the 'therapeutic effect', which includes 'hope to cure the disease', 'no better treatment, and last option' and 'try it out as an opportunity.' Patients are also willing to trust other people's suggestions when making decisions, including 'family decisions' and 'trust medical staff advice.' Other considerations involved in early clinical trials include 'economic considerations.' The experience of participating in early clinical trials is also a complex emotional presence that incorporates multiple emotions, including positive, neutral, and negative feelings. Experiences that are often mentioned include 'feeling disease under control', 'feeling hopeful and psychologically soothing', 'feeling few side effects and good quality of life' and 'feeling the side effects associated with experimental drugs'. 【Conclusion】 The decision-making process of participating in the early clinical trials relies on the search for information, the construction of relevant cognitions and opinions, and the support of others, such as family members and physicians, or the power of belief and spirituality, including 'self-belief' and ' religious spiritual power', and update the understanding of clinical trials, after eliminating the related negative interference, considering the current difficulties in the treatment of the disease, and hoping to cure the disease, measure the 'treatment effect', and continue to rely on the advice of 'medical professionals,' including clinical research nurses. Patients themselves and their families still need to support each other and continue to build confidence, which is a complex decision-making process for patients to agree to participate in early clinical trials. The safety and side effects of new drugs are the major considerations of early clinical trials. The progress of cancer treatment has not yet been fully effective cure, when cancer patients in the face of no conventional treatment options, but also feel the urgent need for anti-cancer drugs to fight cancer, cancer patients in the choice of clinical trials, will look forward to the therapeutic effect and can use the therapeutic regions as soon as possible to fight cancer. Clinical research caregivers need to understand patients' decision-making contradictions and beliefs in participating in trials, especially those participating in early (Phase I) clinical trials, to be clear about the design concepts and related care of early trials.