消费者临床试验指南

A clinical trial is a research study that involves human volunteers. Heralded as the best, if not the only way, to advance medicine, clinical trials are designed to evaluate new ways to prevent, detect, or treat disease. (1,2)

Every medicine on the market, every pill we swallow, every vaccine in use, was once the subject of a clinical trial; countless lives have been saved by penicillin and the smallpox vaccine, to reach back in history and name just two. Current advances include a long list of immunotherapies now approved for use by the U.S. Food & Drug Administration (FDA) totreat lung cancer, as well as other cancers.

在美国和世界各地进行的临床试验的数量高于迄今为止。目前共有291,895项研究ClinicalTrials.gov.,存在的最大临床试验数据库之一。其中，48,921是积极招募参与者。这是从1,255次试验中起来，总共有时National Institutes of Health (NIH)website first became available to the public in early 2000. (3,4)

尽管制药公司和公共卫生机构的努力，但教育人们对临床试验的潜在利益和招聘志愿者，但公众对临床试验的公众意识仍然相对较低 - 以及参与，特别是妇女，儿童和少数群体在研究中受到强名。（5,6,7)

A study published in May 2018 in theJournal of the American College of Cardiologyfound that while women were well-represented in trials of drugs forhypertensionand心房颤动,numbers of those participating in studies involvingheart failure,coronary artery disease，急性冠状动脉综合征显着低于受普通人群中这些条件影响的女性的相对数量。（8)Cardiovascular diseasecontinues to be the leading cause of death among women (and men) in the United States.

Indeed, recruitment in general is considered a major challenge for those leading these trials, and many studies never get off the ground due to a lack of volunteers. Studies involving cancer therapies are known for being hard hit. Over 12,000 cancer clinical trials are in the recruitment stage, but estimates put rate of participation among cancer patients at as low as 3 percent of potential trial candidates. A recent report found that a significant number of trials fail to enroll more than a single patient.

Among the most commonly cited reasons: simple lack of awareness among potential volunteers, mistrust of the process and fear of being a “guinea pig,” fear of not receiving the new therapy but rather the “standard of care” comparison therapy, restrictive inclusion criteria — for example, those who have had prior chemotherapy may be ineligible — and fear of side effects of new drugs. (9,10)

The history of clinical trials is said to date back to 1747, when British physician James Lind conducted a systematic trial among British sailors with scurvy — a disease now known to be caused by a deficiency invitamin C— by dosing some sailors on board a long sea voyage with a combination of orange and lemon juice to see if it helped cure the condition.

很快就会遵循各种研究，但直到二十世纪，当随机审判发挥作用时，这件事就迈出了大跃进。（11)随机化涉及偶然将参与者划分为单独的群体，而不是通过选择某些特征来比较不同的治疗方法。第一次公认的随机试验是在英国医学研究委员会的20世纪40年代中期进行的，以测试疗效antibioticstreptomycin againsttuberculosis。

This trial also featured two other methodological advances in experimentation in that it was double-blind (neither investigators nor patients knew whether they were getting the active drug) and placebo-controlled (some patients received an inactive substance).

Today, most large clinical trials involving drugs that are likely en route to FDA approval are randomized, double-blind, and placebo-controlled — a combination known in medicine as the gold standard. Still, trials involving medical devices and surgeries are frequently not randomized, and many, if not most, devices aren’t subject to the same clinical rigor as drugs.

绝大多数涉及药物的临床试验or biologics — medicinal preparations like vaccines and immunotherapies that stimulate the immune system to fight infection and disease. At last count, 133,007 such trials were registered with ClinicalTrials.gov,more than double the number of studies involving surgical procedures and medical devices combined. (4)

Related:7 Myths About Clinical Trials for Advanced Breast Cancer

临床试验通常是汽车ried out in humans after laboratory and animal testing demonstrate that a treatment is promising, according to the NIH. The trials move through a series of phases and follow protocols including plans to balance potential risks with benefits. Trials are typically conducted in four phases:

Phase 1Researchers test a treatment in a small group of people (about 20 to 80), for a period of several months. The main purpose is to learn about safety and to identify side effects, often as dosage levels are increased. About 70 percent of drugs pass this phase of testing.

Phase 2Usually (but not always) a larger group of people (100 to 300) receive the treatment to further assess efficacy as well as safety and side effects. About one-third of experimental drugs successfully complete both phase 1 and phase 2.

Phase 3The treatment is given to still larger numbers of people (1,000 to 3,000) and can last several years. This phase is intended to provide investigators — and, ultimately, theFDA- 更完整的有关有效性和可能的​​不良反应范围的信息。70％至90％的药物进入第3期研究成功。经过成功的第3阶段试验后，制药公司可以要求FDA批准销售药物或生物学。

Phase 4This phase, also referred to as post-marketing surveillance, occurs after a treatment has been approved by the FDA and is made available to the public. At this stage, researchers are supposed to track safety in the general population and gather information on benefits and optimal use. If problems occur, restrictions may be placed on the drug or result in its being taken off the market. (12,13)

People say they participate in clinical trials for a variety of reasons, including to play an active role in their healthcare; to help others by contributing to medical research; to gain access to treatments before they are widely available; to get medical attention and care they might not ordinarily receive; and even to earn money, according to CenterWatch. (14,15)

参加临床审判的人的满意度高：2013年临床研究参与的信息和研究中心发表的报告（CISCRP）发现，95％的人在过去参加了审判的人他们将来会考虑将来加入另一个。（16）

Still, many potential first-time volunteers never move forward. There are a number of reasons for that. Some, particularly minorities, are disturbed by memory of missteps in the past, primarily the TuskegeeSyphilisExperiment (1932–1972), in which African-American men were told by the U.S. Public Health Service that they were being treated (at no charge) forsyphilis，但事实上，谁根本没有治疗可传染和经常致命的疾病。（17)

Still others are cognizant of past trials that resulted in products or treatments that subsequently turned out to be problematic for some reason, such as沙利度胺,sedativemarketed to pregnant women in the late 1950s for morning sickness that caused thousands of severe birth defects; and the Dalkon Shield, the intrauterine device (IUD) used in the early 1970s that prompted more than 200,000 lawsuits by women harmed by the device.

Other commonly cited concerns include: fears about side effects; doubts that the therapy will be any better than the standard of care; and worry about receiving a placebo — as in, why would anyone want to risk getting a placebo, especially if they are very ill? (14)

While experts in the field acknowledge that the risks of enrolling in a clinical trial are real and entail many unknowns, they also say that some concerns are unfounded. For example, placebos are rarely used in trials involving patients with more advanced or serious illness, saysKenneth Getz, founder of CISCRP, explaining that most use an active comparison drug, typically the FDA-approvedstandard of care。In addition, volunteers are free to withdraw from a trial at any time and for any reason. (18)

Over the years, and often in response to mistakes from the past, practices have been put into place to protect and inform patients. For example, the Tuskegee tragedy led to a process known as informed consent, which involves informing prospective volunteers about the intervention in question and the potential risks involved. Informed consent is now considered both a critical component of patients’ rights and clinical trials themselves. (6)

During the informed consent process, people should be provided with information that enables them to make an informed and educated decision about whether to enroll in the trial. That includes explaining to prospective subjects the purpose of the research, what their role would be and how the trial will work, and allowing them adequate time to ask questions and discuss things with family and friends. TheFDA also notesthat it’s important for people to understand their role as a “subject of research” — not as a patient.

Related:10 Cancer Clinical Trial Facts Every Patient Should Know

临床研究是以各种类型的地点进行，包括医院，大学，医生办公室和社区诊所。该地点取决于谁进行该研究。资助者可以包括政府机构，行业团体，个人，大学和其他组织，如基础和非营利组织。根据NIH的说法，现在，现在在所有50个州和世界各地的200多个国家都存在。（4）

可以找到试验，因为几乎可以想象的每个条件。虽然许多最常见的宣传审判往往涉及高赌注疗法，例如患有晚期癌症的疗法，较低赌注试验丰富。注册试验包括从痤疮到的一切的治疗方法insomnia焦虑。（4）

他们并非关于药品。例如，许多临床试验涉及与植物，矿物质补充剂和针灸等疗法的干预措施，例如，甚至是芳香疗法和用于热闪光的瑜伽。（4）

People with a preference for lifestyle interventions to prevent or treat illness can help play a role in moving those disciplines forward as well. A number of trials involving diet and exercise have been registered.

These include studies assessing the effects of low-fat vegan versusMediterranean diets论体重和胰岛素敏感性，由医生负责医学委员会营运，以及效果intermittent fastingin subjects consuming a Mediterranean or Western diet, part of ongoing research at the Washington University School of Medicine on the prevention and treatment of age-related diseases. (4)

最后，是否参加审判是个人决定，但也许最重要的外卖是决定是一个通知的。Whether the thinking is "buyer beware" or "well-informed is well-armed," the consensus is that it’s best to gather as much information and input as possible from trusted and valued sources, including doctors and other health professionals as well as family and friends.

“我们的座右铭是在参与前的教育，”Getz说。“我们鼓励患者及其家人和朋友聚集事实，与其他患者和专业人员交谈，并提出很多问题。”

There are several searchable databases that offer listings of available clinical trials, including:

• ClinialTrials.gov.
• CISCRP.org

Additionally, you may find searchable listings at local healthcare centers, on the websites of disease specific organizations, and even advertised in local newspapers. (14,19,20,21,22)

In most cases, participating will be at no cost to you. Sometimes, insurance may be billed for some devices, drugs, or services. These should be spelled out in the informed consent process.

What to AskCan you spell out what the cost will be to me?

Some clinical trials will not require travel. Others will require regular check-ins. Find out what will be required of you beforehand.

What to AskWill my travel costs be covered?

A treatment trial may be comparing a new treatment to one that is the standard, or to a placebo. You have no control over which arm of the trial you are assigned to.

What to Ask如果我没有新的治疗，我愿意参加吗？

You can opt out of a trial at any time.

What to AskHow do I handle the situation if I decide to opt out part way through the trial?

一些试验涉及多年的随访。

What to AskWhen does the trial end, and how long will my obligation last?