101 Facts About Clinical Research

1 Facts about 0 1 Published July 2005

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Volunteer Participation Public Perception Clinical Research Process Cost of Research Adverse Events Physician Involvement Minorities Women Elderly Pediatric Cancer Heart Disease HIV/AIDS Lung Disease

More than 2.3 million people participate in approximately 80,000 total clinical trials every year throughout the US. (CenterWatch)

2.

50,000—60,000 of these trials are industry sponsored, while the remaining are government sponsored. (Institute of Medicine)

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In the past 20 years, the average number of patients per trial increased from 1,700 to more than 4,000.

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In order to meet expected demand, the number of respondents to clinical study promotions will have to increase sevenfold, from 2.8 million in 1999 to 19.8 million in 2005. (Applied Clinical

(PharmaExec.com)

9.

Trials)

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From 2001 to 2004 the number of people who stated that they had the opportunity to participate in a clinical research study increased by almost 50% from 13% to 19% (Harris Interactive, The average number of participants needed for a new drug trial jumped from 2270 participants in the 1980s to 3,700 participants in the 1990s. (Food & Drug Administration)

7.

Studies have shown that while 44% of people find out about studies through the media, only 14% gain the information from their physicians. (Harris Interactive)

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80% of total trials are delayed at least one month because of unfulfilled enrollment. (CenterWatch)

In 2003, 22% of studies were completed with a one month delay, while a whopping 72% of studies saw a delay of over one month. (CenterWatch)

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Surveys have shown a trend toward poor volunteer retention in studies, as overall only one out of every four (73.2%) of volunteers stick with a study until its completion. Most dropouts occur during phases II and III.

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From 1997 to 2003, the percentage of studies completed on time decreased from 18% to 6%. (CenterWatch) Only 30% of clinical trial participants report that they first learned about a clinical trial from their primary/specialty health care provider – most information comes from media or the internet. (Institute of Medicine)

May 2004)

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1-15 16-23 24-38 39-49 50-55 57-59 60-65 66-71 72-76 76-78 79-91 92-95 96-98 99-101

(CenterWatch, 2003)

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One study showed that 10% of volunteers did not look at the informed consent before signing it; 18% signed without input from their personal physician, nurse, family member, or trusted; and 70% reported that, at the outset of the informed consent process, they did not know what questions to ask. (Applied Clinical Trials) Most adult cancer patients who participate in clinical trials say that a physician had a great deal of influence on their decision to participate. (Harris

20.

Interactive)

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Even though most people do not rely on their physicians for information, 78% of the public say the physician is their most trusted source of information. (CenterWatch)

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22. 16.

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In a recent poll, 94% of people recognize the importance of participating in clinical research in order to assist in the advancement of medical science. Yet 75% of the general public state that they have little to no knowledge about the clinical research enterprise and the participation process. (CenterWatch) Despite distrust, from 1995 to 2003 the percentage of people who believed that clinical research was important in advancing science increased from 83% to 94%. (Carlton Research and Harris Interactive) The major factors people look at when considering participation in clinical trials includes “If it would benefit me or someone else” (58%). “If I knew all about the risks” was second (48%); “If the risk was minimal or if the reward outweighs the risk” was third (35.3%); “For a cure” was fourth (35.2%); and “If my doctor recommended it” was fifth (34.5%) (Harris

23.

(CISCRP/OCD Survey)

24.

25.

Interactive)

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An overwhelming majority of people (77%), say that they would consider getting involved in an appropriate clinical research study if asked; however, only 10% of those eligible to participate in clinical trials do so in the United States. (Harris Interactive)

Only 12% of survey respondents stated that they have spoken with their doctor or healthcare provider about clinical research. (CISCRP/ODC Survey, 2004) From 1996 to 2002, the percentage of the public who distrusted information received from clinical research professionals increased from 28% to 75%. (Research America / Harris Interactive) Two-thirds (66%) of Americans think that clinical research studies are safe for those who participate. (CISCRP/ODC Survey) More than half of respondents would have greater trust in clinical research information if the results were made available on a public website registry.

26.

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It takes approximately 10 years of study in test tubes and laboratory mice to reach the point where a treatment might be tested for its safety and effectiveness in humans. (Ken Getz, Informed Consent) A clinical trial is sometimes called a clinical research study or a research protocol, but a clinical trial primarily refers to the location where a study protocol is being tested. In other words, a single protocol involves multiple locations across a variety of cities, states and even countries where clinical trials are conducted. (Star-Ledger Staff) Approximately one in 50 drugs that enter pre-clinical testing prove safe enough and effective enough to be tested in people. (Ken Getz, Informed Consent) In the 1960s, it took 8.1 years to develop a new drug; in the 1990s it took researchers 15.3 years, or nearly double the time. (Association of Clinical Research Professionals)

28.

Only one in five drugs that enter clinical trials will prove safe and effective enough to receive FDA approval. And some of these drugs end up being most effective for patients with different diseases than those that they were originally created to treat. (Ken Getz,

36.

Informed Consent)

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More than 120 new remedies get the FDA’s stamp of approval every year, including a handful of “breakthrough” drugs that provide the first effective treatment ever for a variety of medical conditions. (Ken Getz, Informed Consent) Even relatively small improvements to existing drugs, which represent over 40% of new drugs approved by the FDA each year, provide important health benefits to patients. (Ken Getz,

Informed Consent)

37.

Informed Consent)

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According to the FDA, approximately 70% of new medical treatments pass Phase I testing stage. (Ken Getz,

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Only about one-third of drugs that enter clinical testing ever successfully complete phase II and progress to larger-scale phase III studies. This stage provides hard, statistical facts about a drug. (Ken Getz, Informed Consent) For every, 10,000 molecules screened, an average of 250 enter pre-clinical testing, 10 make it through to clinical trials, and only one is approved by the regulator. (Economist, June 18, 2005) Phase III clinical trials involve extensive testing to assess safety, efficacy and dosage levels in a large group of patients facing a specific illness. The study drug is tested on as many as several thousand people over a period of two to five years. (Ken Getz,

34.

Informed Consent)

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Therapies that have reached phase III have already passed toxicity testing and have proved to be at least somewhat effective. But subjects in phase III trials still usually have no better than a 50% chance of getting the investigational treatment versus a placebo or standard therapy. (Ken Getz, Informed Consent)

About 80% of drugs that enter phase III will successfully complete this stage. (Ken Getz, Informed Consent)

38.

Informed Consent)

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The FDA review period usually lasts about one year for most New Drug Applications (NDAs). The FDA also has an expedited review process for priority drugs—usually lasting under six months. Priority drugs are those that represent a notable treatment benefit for critical and severe illnesses. Approximately 60% of all NDAs are approved by the FDA. (Ken Getz,

While the pharmaceutical industry has increased research and development spending over the past 20 years, to a record $30.5 billion in 2001, the number of new drugs has not increased in proportion. (Applied Clinical Trials)

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Drug companies have been increasing their R&D spending by about 6% a year since 1995. The Center for Medicines Research International project that will total $55 billion at the end of 2005. (Economist, June 18, 2005) The average HIV drug cost $479m to bring to market. (Economist, June 18, 2005) The average cost to bring a rheumatoid arthritis drug to market is $936 million. (Economist, June 2005)

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The average American spends almost $5,000 a year on health care. Less than $20 of that amount is invested in research to prevent, treat and cure diseases and disabilities. (Research

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In all, it may take as much as 20 years to bring a single new drug treatment from its initial discovery through to the market. (PhRMA)

America)

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The projected 2005 spending for R&D by phase: preclinical $12b, phase I $4.7B, Phase II $16.2b, Phase IIIb/IV $8.7b. (CenterWatch; Goldman Sachs) According to a Yale University Study, in 1980 32% of biomedical research and development in the US was funded by industry. By 2000, the figure had soared to 62%. (Business Week, June

54. 55.

Administration)

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2004)

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On average, one out of every 30 study subjects per NDA will have a serious adverse event. (Food & Drug Administration) Each year, 800 Serious Adverse Events (SAEs) are typically reported to the FDA for newly approved drugs (Food & Drug

R&D funding by therapeutic area: Cancer and Endocrine 24%, Central Nervous 23%, GI/GU 15%, Infectious Disease 12%, Cardiovascular 12%, Other 10%, Nutrients 5%, Respiratory 4%, Biologicals 3%. (CenterWatch

In reports from industry to the FDA, one out of 10,000 study subjects has died as a result of study drug effects while participating in clinical research studies. This compares to 1 in 3,000 people who die from an accidental injury each year, and 1 out of every 6,000 people who die from a motor vehicle accident annually. (Food & Drug Administration)

Estimates 2004; BCC, 2003)

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Trials funded by companies tend to be four times more likely to have results favorable to them than those funded by other sources. (Guardian Unlimited: Foregone Conclusions)

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In 2002, the National Institutes of Health (NIH) and pharmaceutical and biotechnology companies spent more than $50 billion on research and development, from the discovery phase to FDA approval, on thousands of potential medical treatments. (Ken

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One in eight physicians (13%) curently serve as a clinical investigator. (Harris

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87% of physicians are not involved in clinical trials - 38% attribute lack of opportunity as the main reason for not serving as a Principal or Sub Investigator in a clinical trial and 32% feel that the time commitment is too much (Harris Interactive, May 2004) Federal agencies, who along with drug companies rely on community doctors for about 80% of patients participating in studies. (WebMD Medical News)

Interactive, May 2004)

Getz, Informed Consent)

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The money spent on clinical trials is used to pay for the research professionals managing the projects, for equipment and facilities and study grants to research centers conducting the projects. In 2002, more than $4 billion—of the original $10 billion in 2002—will be paid as grants to investigators within these research centers. (Ken Getz, Informed Consent) The pharmaceutical industry currently spends well in excess of U.S. $30 billion a year in worldwide drug safety, financing a boost in the number of drug safety reviewers from 109 to 134.

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(The Boston Globe, 2005)

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An average of 3.6 deaths attributed to study drug effects are reported to the FDA each year. (Food & Drug

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One adverse event per study subject per New Drug Application (NDA) is typically reported to the FDA. (Food &

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Each year, 17,200 adverse events (AEs) are reported to the FDA for new drugs. (Food & Drug Administration)

Administration)

Drug Administration)

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African-American, Hispanic and Asian trial participants represent over 1/3 of our population, yet in 1999 only 6% African-Americans, 1% Asians and 1% Hispanics, for a total of 8%, participated in trial; down from 12% in 1995. This situation is on great concern because the information we get from clinical trials is of greater value when trial participants more closely represent our population. (First Clinical Research) The involvement of minority physicians has been shown to have a positive impact on minority involvement in clinical trials. At this time, only about 7% of all physicians in the United States belong to a minority group and a very small percentage are actively involved in clinical research. Several medical societies and associations are now looking for ways to encourage minority physician involvement in clinical trials. (Ken Getz, Informed Consent)

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In 1996,African-American patients represented 11% of all cancer trial participants, by 2002 that number declined to 7.9 percent. Hispanics, who make up 9.1% of the U.S. population, made up only 3% of participants in clinical trials in 2002, down from 3.7% in 1996. (Healthday News,

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June 2004)

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Certain minority populations are more likely to suffer from specific diseases, such as diabetes and hypertension. Also, minorities often respond to medications differently. In response, the government has made minority inclusion mandatory for trials that it sponsors. (Ken Getz,

The close of the previous decade saw 8 out of the 10 prescription drugs withdrawn from the U.S. market because they caused statistically greater health risks for women than men. (Science, June 10, 2005) Between 2000 and 2003, the U.S. National Institutes of Health awarded an average of 3% of its grants per year for the research on sex differences. (Science, June 10, 2005)

71.

Men are more likely than women to enroll in colorectal cancer trials and lung cancer trials. (JAMA)

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Approximately to 20% of all participants in drug trials are over the age of 65. (AARP

73.

While the elderly make up two-thirds of patients with either breast, colorectal, prostate, or lung cancer, they represented only one-third of clinical trial participants.

Informed Consent)

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According to some studies, three quarters of Latinos get their information about drugs from pharmacists, not doctors. Usually, pharmacists are not included in the wide net that companies use to recruit patients into trials. (Forbes.com) The National Institutes of Health (NIH) first published guidelines in 1987 for including more minorities in government-funded clinical studies, but these guidelines do not apply to industry-funded research.

Bulletin Online: June 2003)

(Healthday News)

74.

(First Clinical Research)

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A 1993 Federal Law requires that the women be included in every clinical trial involving a disease that affects them and that the NIH make sure studies are designed to collect and analyze data on gender differences. (www.healthywomen.org) Many diseases disproportionately affect women; among them: breast cancer, Alzheimer’s disease, rheumatoid arthritis, multiple sclerosis, osteoporosis, diabetes and depression. (Ken Getz, Informed Consent) From 1977 to 1993, the FDA forbade earlystage testing of most medications on women of childbearing potential for fear of causing birth defects. Now, women are recruited for trials even if they are of childbearing age, but are informed of any potential risks of untested medication on fertility- and pregnancy- related issues. (www.healthywomen.org)

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More than 700 of the 1,000 or so drugs now being evaluated in clinical trials are aimed at treating diseases or conditions associated with aging, according to the Pharmaceutical Research and Manufacturers of America. (AARP) There is a strong relationship between age and enrollment fraction, with trial participation for people 30 to 64 years of age representing 3.0% of incident cancer patients in that age group, in comparison to 1.3% of 65-74 year olds and 0.5% of patients 75 years and older. This inverse relationship between age and trial enrollment fraction was consistent across racial and ethnic groups. (JAMA)

83.

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Only five of the 80 drugs most frequently used in newborns and infants are labeled for pediatric use.

84.

(CenterWatch)

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An estimated 20-30% of drugs approved by the FDA are labeled for pediatric use. (Food and Drug Administration) Including vaccines, the pediatric drug market reaches close to $20 billion worldwide. (CenterWatch)

Finding a cure to cancer would be worth about $47 trillion to the U.S. economy alone. (University of Chicago study) Nearly 9 million Americans have survived cancer. Today in the US there are close to 400 new cancer therapies in pre-clinical and clinical development.

85.

(Bristol-Myers-Squibb)

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Approximately 1.3 million people will be diagnosed with cancer in the US this year; about 50,000 (less than five percent) will enroll in cancer clinical trials. Because of this limited enrollment, it will take about 14 years to bring cancer research out of the lab and into the lives of cancer patients. (Coalition

87.

The five year survival rate for cancer patients has increased to 64% from 59% just ten years ago. (National Cancer Institute

88.

In the past three decades, the cure rate for childhood cancer has improved from less than 10% to 75%, thanks to medical advances made by clinical research.

and Centers for Disease Control and Prevention)

(CureSearch)

89.

Although only 12% of the eligible population participates in overall industry-funded clinical research, 54% of eligible pediatric leukemia patients participate in cooperative studies. (NIH and IOM)

90.

of National Cancer Cooperative Groups, Inc.)

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Clinical trial participants are credited with helping to develop treatments that now save the lives of 70 to 80 percent of the victims of childhood leukemia. (Reader’s Digest)

(Coalition of National Cancer Cooperative Groups, Inc.)

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Only 3% of adult cancer patients sign up for trials even though 20% typically meet eligibility requirements, according to a 1999 study by the American Society of Clinical Oncology. (WebMD Medical News) Although 61% of new cases of cancer occur among the elderly, recent studies indicate that the elderly comprise only 35% of participants in cancer clinical trials. (Journal of Clinical Oncology, 2003) Today, over 60% of pediatric patients with cancer are enrolled in clinical trials.

Less than 5 percent of cancer patients participate in clinical trials. If 10 percent participated, studies could be completed in one year, instead of the three-five years that studies currently require. (National Cancer Institute) 91.

According to a survey of nearly 6000 cancer patients conducted in Spring 2000, Eighty four percent of respondents said they were unaware that participation in a clinical trial was a treatment option; however the majority of these individuals said they would have been receptive to the idea of a clinical trial if they knew it was an option. (Harris Interactive) According to the same survey, Ninety seven percent of respondents who participated in clinical trials reported they were treated with dignity and respect and received excellent or good quality care, while seventy six percent of trial participants said they would recommend clinical trial participation to someone with cancer. (Harris Interactive)

98. 92.

Trials volunteers are credited with helping to develop the treatments that have cut the male death rate from coronary heart disease by 50 percent.

Although only 12% of the eligible population participates in overall industry-funded clinical research, a whopping 41% of eligible AIDS patients participate in cooperative studies. (NIH and IOM)

(Reader’s Digest)

93.

Death rates from the nation’s number one killer, heart disease, have been cut by more than 60% in the past 50 years.

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Thanks to ongoing clinical trials, the American Lung Association anticipates important asthma treatment breakthroughs in the next five years.

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In 2004, four percent of the total funding spent on research and development was spent on respiratory therapies.

(Research America)

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On average, it takes 5 years to develop, from IND filing to NDA submission, Cardiovascular therapies. (CenterWatch) Health economists estimate that the thousands of lives saved by preventing and treating heart disease and stroke from 1970 to 1998 were worth $1.1 trillion each year. (Research America)

It is estimated that 14% of adults receiving care for HIV infection participated in a medication trial or study; 24% had received experimental medications; and 8% had tried and failed to obtain experimental treatments. (New England Journal of Medicine)

97.

(Reader's Digest)

(CenterWatch)

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Over the last 80 years, the Nationwide American Lung Association has granted millions of dollars to scientific studies that have formed the basis for new treatments and helped further our understanding of lung disease and ways to prevent it. Since 1985, the association has invested more than $58 million in lung disease research, funding more than 2,000 scientists. (Breathe Easy Magazine, 2004)

Of the drugs granted “Fast Track” status by the FDA, 10% were for HIV/AIDS drugs. (CenterWatch)

This publication was compiled by The Center for Information and Study on Research Participation (CISCRP), an independent nonprofit group dedicated to educating and informing the public, patients, the media, and policy makers about clinical research participation.

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