Healthcare Research Value, Relevance, and Regulation

In contrast to the last chapter, which looked at the importance of privacy, this one looks at the importance and usefulness of healthcare research. As indicated in the introduction to Chapter 2, the committee views the principles of privacy and healthcare research as complementary. In an ideal world, society would seek to permit both for the benefit of both individuals and society as a whole.

This chapter defines healthcare research, discusses its advantages to individuals and society, and provides a historical overview of federal research regulations in place long before the Privacy Rule went into force. Because a substantial percentage of medical research is covered by a variety of federal rules, it is vital to understand how the various laws overlap or differ. The chapter also addresses how various government constraints have restricted the scope of research and drawn a line between it and some closely related health practise activities, like as quality improvement programmes, that also use health data.

The chapter also analyses current survey data on public perceptions of health research and emphasises the need of patient and public education on healthcare research.

CONCEPTS IN HEALTHCARE RESEARCH AND THEIR VALUE

Definitions

The term “research” is defined in the HIPAA Privacy Rule and the Common Rule as “a systematic investigation, including research development, testing, and evaluation, designed to develop or contribute to generalizable knowledge.” This is a broad definition that may include biomedical research, epidemiological studies, and health services research, as well as studies of behavioural, social, and economic factors that affect health.

Clinical trials, in which people participate freely in studies to assess the efficacy and safety of innovative medical interventions, are possibly the most well-known kind of health research. However, information-based research is becoming a substantial part of the health sciences. A typical element of many studies is the analysis of data and biological samples that were initially collected for diagnostic, therapeutic, or financial purposes, or that were collected as part of previous research initiatives and are now being used for new research. Secondary3 data use is a common research methodology in fields such as epidemiology, health services research, and public health research (Lowrance, 2002; Lowrance and Collins, 2007). It includes the analysis of disease patterns, determinants, and natural histories, the evaluation of health care interventions and services, drug safety surveillance, and some genetic and social studies.

The Importance of Healthcare Research

Healthcare research, like privacy, is very vital to society. It may give critical information on disease trends and risk factors, treatment outcomes or public health initiatives, functional abilities, care patterns, and healthcare costs and utilisation. The different research approaches provide complementary viewpoints. Clinical trials may be used to compare and improve the usage of drugs, vaccines, medical equipment, and diagnostics by limiting the variables that might influence the study’s outcomes. This method, however, requires input from real-world clinical experience. If a drug is authorised by the Food and Drug Administration (FDA) for a certain indication, it is based on a series of controlled clinical trials involving hundreds to thousands of people. However, once a medicine has been authorised, millions of people will be able to utilise it in a variety of conditions. As a result, tracking the drug’s clinical experience is critical for detecting relatively infrequent adverse effects and determining how well it works for different persons or scenarios. Furthermore, it is critical to record and assess clinical practises in order to establish best practise standards and ensure great patient care.

Collectively, these types of health research have resulted in significant discoveries, the development of novel treatments, and significant improvements in health care and public health.4 Economists have discovered that healthcare research can significantly improve people’s health and longevity, in addition to the personal benefits that accrue to individuals (Hatfield et al., 2001; Murphy and Topel, 1999). Important societal interests are jeopardised if research activity is impeded or weakens.

The development of Herceptin as a breast cancer drug (Box 3-1) is a notable example of the benefits of research employing biological samples and patient data (Slamon et al., 1987). The examination of medical records has changed the way medicine is done in numerous other cases. According to such studies, tens of thousands of Americans die each year as a consequence of medical errors committed in hospitals. Research has also contributed to the identification of methods to decrease medical mistakes via the use of health information technology, such as e-prescribing (Bates et al., 1998; IOM, 2000b). This kind of research has also shown that access to care is limited in rural and urban areas, and that these characteristics have a detrimental influence on health outcomes (Mick et al., 1994). Furthermore, research on medical records has shown that preventive services, such as mammography, significantly reduce mortality and morbidity at low cost (Mandelblatt et al., 2003), and has established a causal link between the nursing shortage and patient health outcomes by demonstrating that patients in hospitals with fewer registered nurses stay longer and are more likely to experience complications, such as urinary tract infections and upper gastrointestinal bleeding. All of these findings have influenced and led national policy decisions. As more individuals use electronic medical information, the pace of this kind of research is increasing, as are opportunities to contribute new knowledge about what works in healthcare (CHSR, 2008).

Because of advancements in health information technology, healthcare research is experiencing a revolution. This development may enable previously unfeasible investigations to be conducted, resulting in new insights regarding health and sickness. According to the National Committee on Vital and Health Statistics, clinically rich information is now more freely available, better ordered, and capable of being electronically shared throughout the health and health care continuum. As a result, the data may be used more effectively for research, quality improvement, and public health, ultimately benefiting people’s health and access to healthcare (NCVHS, 2007a). The informatics grid recently created with support from the National Cancer Institute (Cancer Biomedical Informatics Grid, or caBIG) is an example of how information technologies can facilitate health research by enabling greater sharing of health data while maintaining regulatory compliance and patient privacy.

Because science is advancing fast and becoming more difficult, a single researcher or single site cannot give all of the information required to generate and assess medical discoveries or to ensure their safety. As a consequence, good information transmission between institutions is now more important than it was before, when there were fewer novel therapies being produced.

Because of the proliferation of treatment options and the growing expense of new treatments, further scrutiny of true efficacy is necessary once efficacy has been shown. For this, registries of patient characteristics, outcomes, and unfavourable events are required. Large populations are required to compare patient groups and estimate risk/benefit ratios. INTERMACS6 (Interagency Registry for Mechanically Assisted Circulatory Support) is a statewide registry for patients having mechanical circulatory support device therapy to treat severe heart failure. This registry was developed in collaboration with the National Heart, Lung, and Blood Institute, the Centres for Medicare & Medicaid Services, the Food and Drug Administration, clinicians, scientists, and industry representatives. The data analysis is expected to aid in improved gadget development as well as better patient care and evaluation. The registry’s findings are expected to have an influence on current research as well as aid in the correct regulation and payment of such devices. The Extracorporeal Life Support Organisation (ELSO), a global association of medical experts and researchers focused on the development and assessment of novel therapies for the support of failing organ systems, maintains a registry of extracorporeal membrane oxygenation and other cutting-edge methods of organ system support. Registry data is used to help regulatory agencies, clinical care, and research. Another example is the United Network for Organ Sharing (UNOS) database, which was created for the collection, storage, analysis, and publication of data related to patient waiting lists, organ matching, and transplants.8 Launched in 1999, this secure, web-based system contains information on every organ donation and transplant event that has occurred in the United States since 1986.

There are several advantages to information-based research, such as study that uses healthcare information databases (Lowrance, 2002). It has the ability to evaluate extremely big data sets and may discover unanticipated occurrences or variations across subpopulations that would not be included in a controlled experimental investigation. Controlled trials are commonly employed when they are simply unfeasible owing to ethical, technological, or other constraints. It may also be used to assess the success of a certain test or intervention in actual praxis, rather than only its theoretical effectiveness. It is also feasible to review data obtained in previous research endeavours, such as clinical trials, in order to answer to new enquiries quickly and inexpensively. However, information-based healthcare research has limitations. It often has less statistical rigour than controlled clinical studies because it lacks the scientific control over the initial data collection, quality, and format that prospective experimental healthcare research may impose from the start. In addition to these scientific constraints, obtaining individual consent for the research may be difficult or impossible due to its relational and, at times, physical distance from the data subjects, as well as the huge amount of information involved.

Advances in information-based healthcare research may also aid in the shift to personalised medicine, increasing the relevance of healthcare research for individuals. Personalised medicine strives to offer prevention programmes and treatments that are particularly matched to each individual’s genetic composition and medical history. Despite breakthroughs in health care via innovative treatments, it is well acknowledged that the bulk of pharmaceuticals only function in a tiny fraction of people who have the ailment for which they are given. Furthermore, a small percentage of people are likely to have unpleasant side effects with drugs that have been shown to be safe for the majority of people when taken at the recommended dose. The variety of the patient population causes these two phenomena. More customised approaches to health treatment are becoming feasible with the potential to provide “the appropriate drug, at the appropriate dose, to the appropriate patient, at the appropriate time,” owing to breakthrough developments in the study of genetics and other indicators of health and illness. Achieving the aims of personalised medicine will improve the efficacy and safety of medical therapy.

Public Perceptions of Healthcare Research

Numerous studies have been undertaken to ascertain the public’s view of research and the factors that influence people’s motivation to participate in research. The primary focus of this chapter’s surveys is interventional clinical trials. The second chapter examines survey questions used to gauge public acceptability of allowing their medical information to be used for research purposes.

The public values healthcare research.

Numerous polls show that the vast majority of Americans support medical research and believe it improves society. A recent Harris poll found that around 80% of respondents were interested in the results of health research, which was consistent with prior survey findings (Westin, 2007). According to a 2005 study that assessed data from 18 national polls and 70 state surveys, the majority of Americans believe that maintaining US superiority in health research is vital.

According to 78% of respondents, it is highly essential, while 17% believe it is moderately important. Only 4% of Americans believe it is critical for the United States to maintain its position as the global leader in healthcare research (Woolley and Propst, 2005). A 2007 study found similar results: 76% of respondents said science is very important to our health, and 78% said science is very important to our capacity to compete (Research!America, 2007).

According to Virginia Commonwealth University’s 2004 Life Sciences Survey, the majority of Americans embrace research. In this study, 90% of respondents agreed that scientific advances have improved society; 92% agreed that “scientific research is essential for improving the quality of human lives”; and 84% agreed that “the benefits of scientific research outweigh the harmful results” (NSF, 2006).

Overall Participation in Research Experience

The feelings of persons who have participated in healthcare research are generally unknown. However, the evidence that is presently available indicates that research participants had generally positive experiences. According to a recent Harris Poll, 13% of respondents had participated in some kind of health research, and 87% were happy with the experience (Westin, 2007). In a cancer study, 93 percent of participants stated they had a very positive experience, and 76 percent said they would recommend a clinical trial to someone with cancer. According to the majority of clinicians surveyed for this study, clinical trial participants get the best treatment possible and have outcomes that are at least as good as those of cancer patients having conventional cancer therapy (Comis et al., 2000). According to another poll, 55% of people who participated in research studies claimed they would do so again in the future (Trauth et al., 2000).

Capability to Participate in Research

According to public opinion surveys, the majority of Americans are willing to participate in clinical research trials. A collection of research articles! ordered was published in 2001. According to a Woolley and Propst poll done in 2005, 63 percent of Americans claimed they would be willing to participate in clinical research. This percentage has remained constant throughout time. A Study from 2007!According to a poll done in the United States, 68 percent of respondents said that their desire to better their own or others’ health was a major factor in deciding whether or not to engage in a clinical research project (Research!America, 2007). Another poll conclusion was that 63 percent of Americans would be extremely willing to participate in a clinical research study if they were asked.

According to previous studies, there is a high degree of interest in participating in research on certain conditions. A poll found that 88% of people would be ready to participate in a study to learn more about cancer, 86% about heart disease, 83% about a deadly ailment that cannot be treated, 79% about addiction, 78% about depression, and 76% about schizophrenia (Trauth et al., 2000). Respondents’ willingness to participate grew as their research expertise increased (Trauth et al., 2000). A separate study found that if they were diagnosed with cancer, 8 out of 10 Americans would consider participating in a clinical trial. More than two-thirds of respondents would be willing to participate in a healthcare research targeted at cancer prevention (Comis et al., 2000).

Furthermore, Americans seem to be strongly in support of genetic data-based medical research. According to a 2007 research conducted by the Genetics & Public Policy Centre, 93% of Americans felt that genetic testing should be used to assist scientists in developing new techniques to detect, prevent, or cure sickness. According to two separate surveys (Genetics & Public Policy Centre, 2007; study!America, 2007), 66% of Americans are willing to donate their genetic material for healthcare research. Despite this apparent good attitude towards genetic research, 92 percent of Americans claimed they were concerned about their genetic information being used in a “harmful way” (Genetics & Public Policy Centre, 2007).

A person’s motivation to participate in a medical research study may be impacted by a range of factors, including concerns about confidentiality and privacy (Genetics & Public Policy Centre, 2007; Research!America, 2007). Those with higher incomes, undergraduate or graduate degrees, or families with children were more inclined to participate in research, according to the Trauth survey. Age had an effect on willingness to participate: just 31% of respondents 65 and older were eager to join in research, compared to 57% of respondents 18 to 34 (Trauth et al., 2000).

Race and ethnicity are two more factors that may influence a person’s motivation to do research. Minorities, as is commonly known, participate in healthcare research at a far lower rate than white Americans. This disparity may be the result of many linguistic, economic, and cultural barriers (Giuliano et al., 2000), and study on the subject has shown conflicting results. Several studies (Braunstein et al., 2008; Corbie-Smith et al., 1999; Farmer et al., 2007; Grady et al., 2006; Shavers et al., 2002) have shown that racial and ethnic minority groups participate in medical research at a lower rate than the general population. This is assumed to be due to their distrust of the medical research community.

Additional statistics indicate to minority groups’ lack of access to the research community as a contributing reason to the low number of minorities participating in healthcare research (Brown et al., 2000; Wendler et al., 2006; Williams and Corbie-Smith, 2006). As a result, it is likely that ineffective minority recruitment techniques are at least largely to blame for the low number of minorities participating in medical research.

According to a cancer research study, one of the biggest hurdles to individuals engaging in research is not knowing about the presence of clinical trials. Nearly 6,000 cancer patients were questioned, and 85% said they were unaware of the opportunity to participate in a healthcare research study. Participants in the survey stated that they chose to participate because they believed one of the following: trials provide patients with the highest quality of care (76 percent); their participation would help future cancer patients (72 percent); they would receive newer and better treatments (63 percent); or they would receive more care and attention (40 percent) (Comis et al., 2000).

A doctor’s suggestion may also impact participation. According to one survey, 48 percent of Americans responded that a doctor’s recommendation would be a major factor in deciding whether to participate in a research study. Furthermore, more than three-quarters of those polled felt that the reputation of a study should be considered when deciding whether or not to participate (Research!America, 2007). In an Italian public opinion survey, 20% of respondents said that having a doctor as a reference during a research study influenced their willingness to participate (Mosconi et al., 2005).

Overall, surveys demonstrate that most Americans prefer medical research, believe it helps society, and are interested in the findings of health research. Despite the fact that little is known about the attitudes of people who have actually participated in medical research, the evidence that is presently available indicates that the majority of study participants had positive experiences. According to polls, the majority of Americans are willing to participate in clinical research trials. Surveys reveal that, similar to the results in Chapter 2, a range of variables, such as the kind of study and personal characteristics such as health condition, age, education, and race, might possibly impact a person’s desire to engage in medical research. Individuals who were more acquainted with the study methodology were more open to participating.

HEALTHCARE RESEARCH CONTROL

Historical Development of Federal Healthcare Information Research Protections

In response to previous infractions in biomedical research, international agreements, federal legislation, and federal rules limiting the use of human subjects were often formed (reviewed by Pritts, 2008) (Box 3-3). The most well-known examples were the Tuskegee syphilis study, which began in 1932 and withheld effective treatment from affected African American men long after a cure for syphilis had been discovered, and the reported abuse of concentration camp inmates in Nazi experiments during World War II. As a consequence, they focus on the concepts of consent and autonomy. Although the principles apply to research that uses identifiable health information, its primary goal is not information-only research.

BOX 3-3

The Foundation for Human Subjects Protection in Biomedical Research. The Nuremberg Code, created by the international community during the Nazi War Crimes Trials, is considered as the first codification.

The Belmont Report was arguably the most important inquiry of human subject protection in research in the United States. The Belmont principles were the cornerstone for institutional control of human subjects research in the United States, and they have been elaborated upon in a variety of situations. Governments do not directly control the bulk of researchers’ activities (Burris et al., 2003). The Department of Health and Human Services (HHS) did, however, implement the Belmont Commission’s recommendations in 1979 in Subpart A of 45 C.F.R. 46 (“Subpart A”), which was recognised as a model policy for federal organisations. The President’s Commission for the Study of Ethical Problems in Medicine, Biomedical and Behavioural Research recommended10 in December 1981 that all federal agencies adopt Subpart A.

The President’s Office of Science and Technology Policy formed the Committee for the Protection of Human Research Subjects in 1982 to fulfil the commission’s recommendations. The committee concluded that it is desirable for federal regulations on human subject protection to be standard in order to eliminate unnecessary requirements and encourage greater understanding among institutions that conduct federally funded or regulated research. As a consequence, in 1991, 18 federal agencies joined HHS in establishing a unified set of rules for the protection of human research subjects that were similar to Subpart A of 45 C.F.R. 46 and became known as the “Common Rule.”

An Overview of the Common Rule

The Common Rule governs most federally funded human subjects research, which strives to protect participants’ rights at all phases of a study. The Common Rule attempts to minimise bodily and psychological harm while underlining the importance of individual liberty and consent. It also requires an Institutional Review Board (IRB) to conduct an impartial review of research. Privacy and confidentiality precautions are seen as key risk concerns in research, despite the fact that they are not officially and completely defined.

The informed consent of the study participant and the IRB review of proposed research serve as the foundation for meeting the goal of protecting human subjects. This section discusses the essential components of the Common Rule (Pritts, 2008 review). Chapter 4 contains in-depth information on particular obligations related to the HIPAA Privacy Rule.

The Goal of the Common Rule

As previously stated, research is defined as “a systematic investigation, including research development, testing, and evaluation, designed to develop or contribute to generalizable knowledge.”13 As a result, the Common Rule is generally only applicable to federally funded research on human subjects.

According to the Common Rule, a “human subject” is “a living individual about whom an investigator… conducting research obtains (1) Data through intervention or interaction with the individual, or (2) Identifiable private information,” with “Identifiable private information” defined as “private information that can be used to identify the subject or that can reasonably be used to identify the subject by the investigator or that is associated with the information.”

The Common Rule applies to the vast majority of federally funded human subjects research projects, but it also has a broader impact because the vast majority of institutions that accept federal funding sign a contract with HHS (known as a Federalwide Assurance, or FWA) pledging to follow the Common Rule’s guidelines for all research projects, regardless of funding source. However, some privately funded human subjects research is conducted outside of the purview of government regulation (Goldman and Choy, 2001; Williams, 2005). Businesses and other groups are allowed to choose whether or not to apply the Common Rule freely to their research initiatives, and many do. However, when compliance is optional, HHS does not monitor or sanction research programmes (Goldman and Choy, 2001; Williams, 2005).

Consent Recognized

Before participating in a research, a subject must grant informed consent (typically in writing) according to the Common Rule (Williams, 2005). In order for a person to freely accept or decline participation in a research project, informed consent is obtained via a method in which a person learns critical data about the investigation, including potential risks and benefits.

The standards for informed consent in the Common Rule focus greater emphasis on the components and supporting evidence of informed consent than on the process by which it was gained. In terms of process, the rules require that informed consent be obtained only when the prospective subject has sufficient time to decide whether or not to participate. According to the Common Rule, informed consent information must be supplied in a language that the subject understands, and assent should not be regarded as renouncing legal rights or absolving the investigator of duty for carelessness committed during the course of the study.

Furthermore, the Common Rule specifies a number of components that must be provided when informed consent is sought. These elements are as follows:

  • an explanation of the research’s goals,
  • the topic’s projected participation timeline,
  • the research’s potential benefits and drawbacks,
  • how information will be kept confidential,
  • the engagement is entirely optional,
  • Who the participant should contact if they have any questions about the study or their rights as a research participant.

The Common Rule allows for informed authorization to be granted for future undefined research under specific circumstances. The Common Rule permits for the use of informed consent to get a person’s permission to analyse personally identifiable information stored in a repository for hypothetical future research aims, for example (HHS, 2003).

The required components of informed consent apply to all types of research, though some are more relevant to biomedical research (for example, the consent must include a disclosure of appropriate alternative procedures or courses of treatment, if any, that might be advantageous to the subject). Informed permission is particularly important in research including personally identifiable health information. An informed consent must contain a statement detailing how, if at all, records identifying the subject will be kept secret, according to the Common Rule.

Institutional Review Boards
The Common Rule, which incorporates the Belmont Report’s principles, requires that protocols for human subjects research be reviewed by an IRB (Box 3-4) before research can begin. The IRB must meet certain membership requirements, including having members with different specialties and at least one member who is not affiliated with the investigator’s institution. The Common Rule specifies the IRB review levels necessary for various types of research, as well as the elements the IRB should consider while completing the review. Even though it does not specify the methodology an IRB must use when assessing protocols, the Common Rule requires the IRB to develop written processes for how it will analyse protocols and record IRB decisions.

BOX 3-4

Institutional Review Boards. According to the Department of Health and Human Services (HHS) Institutional Review Board (IRB) rules, “the IRB is an administrative body designed to safeguard the rights and welfare of human research participants (more…).”

Before accepting a research proposal, an IRB must assess whether the following requirements are satisfied, according to the Common Rule:

  • The dangers to subjects are maintained to a minimum.
  • Subjects’ risks are justified in light of the prospective benefits, if any, and the importance of the information that may be anticipated to emerge.
  • The subjects were selected fairly;
  • In accordance with the rules, informed permission will be obtained and documented.
  • When required, the research approach contains appropriate mechanisms for monitoring the information acquired to ensure the well-being of participants.
  • When appropriate, adequate safeguards are put in place to protect subject privacy and data confidentiality.

For low-risk research, an IRB may exempt the need for informed consent or permit a revision to the permission form, and in exceptional cases, the IRB may even waive the requirement for formal consent.

Personal Particulars

The use of “private identifiable information” is regarded as human subjects research under the Common Rule, as previously stated, if the subject’s identity is or may be easily discovered by the investigator or linked to the information accessible by the researcher if the data are regarded personally identifiable.20 However, the Common Rule exempts from its obligations research that involves:

The collection or analysis of previously collected data, records, papers, pathology samples, or diagnostic samples, provided that these sources are available to the public or that the material is documented in a manner that prohibits direct or indirect identification of subjects.21

If otherwise identifiable data is coded and certain other requirements are met, it may be deidentified or “anonymized” for the purposes of the Common Rule (HHS, 2004). Information is “coded” if identifying data (such as a name or Social Security number) that would allow the researcher to easily determine the identity of the person to whom the private information or specimens pertain has been changed.

If the following requirements are met, research using just coded private data or samples is not considered to include human subjects under the Common Rule:

Interactions with or interventions on living individuals did not provide private data or samples, especially for the research aim presently being suggested; and

Because of factors such as: the investigator(s) are unable to quickly establish the identification of the person(s) to whom the coded private information or specimens relate.

—Before beginning the research, the key to breaking the encryption is deleted;

—Until the persons involved have died, the investigators and the owner of the key agree not to release the key to the investigators under any circumstances.

—the dissemination of the key to investigators is banned by established norms and operational procedures for an IRB-approved repository or data management centre until the persons implicated have died;

Other legal limitations prevent the investigators from gaining access to the key until the individuals are deceased.
When a researcher accesses or gets data that has been coded but does not have access to the identifying key, this study is not considered human subjects research and is not necessary to acquire informed permission or have an IRB review and approve a protocol.

Human Research Subjects Protection under the FDA

The FDA is also responsible for ensuring the rights, safety, and welfare of human subjects who participate in clinical studies25 involving items covered by the Federal Food, Drug, and Cosmetic Act26 (the Act), as well as studies that support requests for research or marketing authorizations for FDA-regulated goods such as medications, medical devices, and biological products for human use (Box 3-5).

BOX 3-5

FDA Protection of Human Subjects Regulations The Food and Drug Administration’s (FDA) Protection of Human Subjects Regulations are intended to protect the rights of persons who engage in research utilising commodities that are within its authority (more…).

While HHS adopted the Common Rule regulations on the protection of human research subjects in January 1981, the FDA adopted regulations governing informed consent of human subjects27 and regulations establishing standards for the composition, operation, and responsibilities of IRBs that review clinical trials involving human subjects28, which were harmonised with the Common Rule to the extent permitted by law in 1991.

DIFFERENCE BETWEEN HEALTH RESEARCH AND PRACTISE

The Common Rule and Privacy Rule artificially separate health research and some closely related health care practises, such as public health practise, quality improvement activities, programme evaluations, and utilisation reviews, all of which may involve the gathering and analysis of personally identifiable health information. For IRBs, Privacy Boards,32 researchers, and health care practitioners, however, determining which activities fall under the Common Rule and Privacy Rule can be difficult.

IRBs and Privacy Boards must correctly identify the numerous actions that are or are not governed by the various sections of the Privacy Rule and Common Rule. Only research requires official IRB or Privacy Board assessment and informed consent33. Important health care activities, such as public health practise and quality improvement, may become difficult or impossible to carry out if an activity is incorrectly classified as research.

Standard criteria are urgently needed to distinguish between health research and related activities, and the committee advises HHS to interact with key stakeholders in order to create such standard criteria. This requirement has been recognised by HHS, which has produced a working paper titled “What Is Research?A number of different models have also been proposed to assist decide whether efforts in the disciplines of public health and quality improvement should be classed as research, as mentioned below, and they might be useful in formulating HHS recommendations. However, it seems that progress on this project has been postponed for unclear reasons (NCURA, 2007). Any standards adopted by HHS should be evaluated on a regular basis to ensure that they are helpful and achieve the desired goals.

Some ongoing attempts to develop such criteria in the public health and quality improvement areas are mentioned in the sections that follow. The committee’s purpose is to illustrate the challenges in drawing these distinctions and adopting clear standards, rather than to endorse any one paradigm.

Comparing and contrasting public health research and practises

In the Belmont Report (CDC, 1999), health practises were defined as “interventions designed solely to improve the well-being of the person, patient, or client, and which have a reasonable expectation of success.” To apply this notion to “public” health practises, the target beneficiary of the intervention must be broadened to encompass advantages to the community as a whole rather than just a single person. Public health research is not formally specified under either the Common Rule or the Privacy Rule; rather, it is included by the broad idea of study. The Privacy Rule applies to public health practises, but not to public health research (see Chapter 4).
Early models for distinguishing public health practise from research focused on the purpose of the activity, noting that the purpose of public health research is to “contribute to or generate generalizable knowledge,” whereas the purpose of public health practise is to “conduct programmes to prevent disease and injury and improve community health” (Snider and Stroup, 1997). The Centres for Disease Control and Prevention developed a similar technique for a larger purpose review. For example, the model says that in public health research, the project’s intended benefits extend beyond the study participants, and the data acquired surpasses the individuals’ care requirements. However, in the context of public health practise, the intended benefits of the project are primarily for the participants in the activity or the community of the participants, and the only data collected are those required to evaluate or improve a public health programme or service, the health of the participants, or both. The approach also assumes that public health practise is non-experimental and depends on time-tested medical approaches (CDC, 1999). Both of these models, however, have been criticised for being excessively subjective and dependant on the person doing the activity (Gostin, 2008; Hodge, 2005).
A new, more comprehensive model was recently released as a more objective checklist to be used by IRBs, Privacy Boards, and interested parties (Hodge, 2005; Hodge and Gostin, 2004). This model combines elements of the preceding two. This model is founded on clear definitions of public health research, defined as “the collection and analysis of identifiable health data by a public health authority for the purpose of generating knowledge that will benefit those beyond the participating community who bear the risks of participation,” and public health practise, defined as “the collection and analysis of identifiable health data by a public health authority for the purpose of protecting the public.”
The approach is based on two key tenets. First, the person carrying out the disputed behaviour is a federal, tribal, state, or municipal government official, agent, agency, or organisation.
Second, the disputed action involves the collection, use, or disclosure of personally identifiable health information.
BOX 3-6
a framework for differentiating public health practise and research Public health stage 1 practise:

Health research versus quality improvement

Quality improvement is defined as “systematic, data-guided activities designed to bring about immediate, positive change in the delivery of health care in a particular setting” (Baily, 2008).35 Quality improvement efforts are not subject to IRB or Privacy Board clearance, according to the Common Rule and Privacy Rule, which identify quality improvement as a part of health care operations.

However, it can be difficult for medical professionals, institutional review boards, and privacy boards to determine whether a specific action is solely for quality improvement or includes research. In one survey36, physicians working on quality improvement were found to be less likely than IRB chairs to believe that IRB review was required for a specific hypothetical activity or that informed consent was required.

BOX 3-7

To reduce the occurrence of catheter-related bloodstream infections, Peter Pronovost of Johns Hopkins University (JHU) led a quality improvement project at 103 intensive care units (ICUs) in Michigan hospitals. (more…)
Others have identified specific criteria to separate quality improvement activities from research, while others in the health care sector have advocated for requiring all prospective quality improvement programmes to be evaluated externally (Bellin and Dubler, 2001).

For example, Casarett and colleagues developed a two-part test to identify quality improvement initiatives. The first criterion is whether “the knowledge to be gained” from the effort is expected to directly assist the majority of patients. This implies that the patients must gain something useful from the information acquired during the examination, rather than simply receiving the procedure itself.

The Hastings Centre recently published a paper on the parallels and differences between research and quality improvement, highlighting three key qualities of research and three fundamental qualities of quality improvement. According to the authors, all patients want high-quality medical treatment, and the success of a quality improvement activity depends on the collaboration of all patients.

The study, on the other hand, argues that participation in research should be optional, and that decisions to participate should be based on researchers’ complete disclosure of all the benefits and drawbacks. Furthermore, the authors argue that most research has no immediate benefits for the institution conducting it, and that it is intended to provide new information about human health rather than relying solely on what is already known.

The authors came to the conclusion that IRBs are not the proper entity to oversee the morality of quality improvement initiatives. They contend that since these activities vary from research in how they are carried out, IRBs needlessly impose large transaction costs on them. For instance, in research, any modifications to the approach need for further IRB clearance. On the other hand, quality improvement activities often modify the intervention, measurement, and activity objectives in light of the investigators’ experience. The amount of time and effort needed to perform the initiative and generate useful data is considerably increased if the investigator is forced to go back to the IRB every time a minor alteration is necessary. Additionally, the investigators engaging in quality improvement efforts are often already caring for participants clinically and are accountable for the effectiveness and safety of an intervention. The authors contend that the extra control provided by an IRB is not necessary to ensure participant safety.

The report instead advocated integrating the ethical oversight of quality improvement activities into the ongoing management of a facility’s health care delivery system, suggesting that clinical care organisation managers could be in charge of overseeing quality improvement, and that consent to receive treatment should also include consent to participate in any low-risk quality improvement projects.

In May 2008, the IOM and the American Board of Internal Medicine co-sponsored a conference to address the persistent ambiguity about when quality improvement becomes research and requires IRB oversight. Attendees included prominent members of the quality improvement community, and both immediate and long-term solutions to this issue were proposed. However, no formal report was generated from this conference, and no broad agreement was achieved.

THE VALUE OF EFFECTIVE PUBLIC COMMUNICATION

The great majority of Americans, according to polls, feel that health research is essential and are interested in the results of research studies, as was previously mentioned in this chapter. Additionally, the majority of patients seem to be open to taking part in health research, whether it be by volunteering for a study to test a medical procedure or by granting permission to access their medical files or stored biospecimens, under certain circumstances. Their desire to engage is based on their confidence in researchers to protect patients’ rights and wellbeing, including assurances of privacy and confidentiality, and their conviction that the project is useful and justifies their participation. Patients, however, are seldom told about study findings that can directly affect their health and often lack knowledge about how research is carried out. The committee’s recommendations in this section are meant to address the public’s desire for more information about health research as well as to contribute to the achievement of two of the report’s major objectives: (1) enhancing the security and privacy of patient information; and (2) enhancing the efficacy of health research.

Distribution of Health Research Results

Long-term community involvement in health research projects has been recommended by ethicists, as has increased dissemination of study findings (reviewed by Shalowitz and Miller, 2008a,b). Transparency, which is the need to clearly explain how and why personally identifiable information is acquired, is a critical component of comprehensive privacy safeguards, according to the IOM committee.

When patients give permission for the use of their medical information in a specific study, health researchers should put more effort into informing study participants about the outcomes, as well as the relevance and value of those results. Patients may feel more a part of the process and be more likely to join in future trials if they are informed about clinically significant results from a research in which they have participated.

Nonetheless, giving participants useful feedback is hampered by a number of factors other than cost. While required and appropriate, a summary of the findings alone may be viewed as a token and raise concerns about topics such as the best way to create summaries, when results should be shared, and how to communicate research with uninformative results.

There are some guidelines for communicating and explaining study results to research participants, but they differ in specifics due to a lack of data on the subject and, as a result, a lack of standards (Partridge and Winer, 2002; Partridge et al., 2008; Shalowitz and Miller, 2008b; Zarin and Tse, 2008).

Research registries

By registering trials and other studies in public databases, one may increase the public’s access to information regarding research investigations. In particular, where research is undertaken with an IRB/Privacy Board authorised waiver of permission or authorisation (see Chapter 4), HHS should support such registration of trials and other studies. There are currently several clinical trial registries, and enrollment has grown recently (reviewed by Zarin and Tse, 2008). ClinicalTrials.gov, a clinical trials registry created by the National Library of Medicine in 2000, has grown to contain data from a number of additional trial registries and serves as the FDA’s mandatory site for submissions about clinical studies subject to the FDA databank requirement. The FDA Amendments Act of 200738 created the first government financed trials outcomes database and broadened the scope of needed registrations at ClinicalTrials.gov. It requires the registration of all FDA-regulated medicines, biologics, and devices that are the subject of controlled clinical research, except Phase I trials.

Furthermore, prospective trial registration is required for publication, according to an ICMJE guideline implemented in the autumn of 2005 (DeAngelis et al., 2004). According to Zarin et al. (2005), this approach resulted in a 73 percent increase in trial registrations from all intervention types, with approximately 45,000 trials recorded by autumn 2007.

Although the creation of such registries is a critical first step towards making high-quality clinical trial information available to the public, there is currently no centralised system in place to disseminate information about ongoing studies being conducted with drugs or other interventions. As a result, it can be challenging for consumers and their healthcare providers to locate ongoing research. The international policies of the ICMJE or the World Health Organisation, which require the registration of all interventional studies in humans regardless of the type of intervention (Laine et al., 2007; Sim et al., 2006), are more comprehensive than the current statutory requirements for registration and data reporting in the United States.

Furthermore, these databases often do not include noninterventional studies, such as observational studies, which are becoming increasingly important in biomedical research. Inclusion of such studies in a registry might be a significant strategy for raising public awareness of such research since many noninterventional studies are carried out with an IRB/Privacy Board authorised waiver of permission or authorisation.

Public Awareness of Research Methods and Value

As previously stated, clinical trials are the most well-known form of health research, but a significant portion of information-based health research entails analysis of thousands of patient records in order to comprehend human diseases, assess the efficacy of treatments, and discover harmful side effects of therapies. As the selection of electronic documents grows, this type of study will likely become more common.

In a recent study that used focus groups to examine veterans’ opinions on the use of medical records in research, for example, it was discovered that the majority of participants (75%) were unaware that “under some circumstances, [their] medical records could be used in some research studies without [their] permission,” despite the fact that a notice of privacy practise was provided.

Furthermore, polls show that many patients, in addition to notification, want the opportunity to decide whether to agree to such study using their medical information, and that patients who want their consent for each study to be sought are most concerned about the negative effects of improper disclosure of their personally identifiable health information, such as discrimination when applying for jobs or life or health insurance.

Strengthening security safeguards for health data could lower the likelihood of security breaches and their possible harmful effects, as mentioned in Chapter 2, and should thus assist to allay patients’ fears in this respect. The public’s continuous engagement in health research depends on patients’ continued faith in the research community, which may be increased by providing patients with information about how health research is done, monitored, and reported on. For instance, researchers are often given datasets without clear identifiers like name and Social Security number. Additionally, IDs are not included in study findings publications. Additionally, under both the Privacy Rule and the Common Rule, a waiver of consent and authorization is only permitted when the research entails minimal risk and when obtaining individual consent and authorization is impracticable (see the previous section and also Chapter 4). A waiver is only granted in these circumstances. Finally, it is required by professional ethics that researchers protect data and respect privacy.

Patients’ understanding of the importance of medical record research will be critical, as surveys show that people are more receptive to research that affects them and their loved ones. Education initiatives should also emphasise the negative effects of inadequate datasets on research results, as datasets will not accurately reflect the entire population if some people refuse to provide their health information, even if representative samples are required.

BOX 3-8

The selection bias in health research. It is likely that patients will be unwilling to provide researchers access to their medical information if they are required to get permission or consent beforehand.

In medical records research, a general demand for permission or authorization results in inadequate datasets, biassed findings, and incorrect conclusions. Even though permission and consent rates at certain significant medical institutions (such as the Mayo Clinic) may reach as high as 80%, the 20% of patients who decline have different demographic and clinical features.

Many instances of significant study discoveries from medical records research would not have been possible if direct patient agreement and authorization were always required (Box 3-1). For example, a review of medical records revealed that newborns exposed to the hormone diethylstilbesterol (DES) in the first trimester of pregnancy had a higher risk of developing breast, vaginal, and cervical cancer, as well as reproductive abnormalities as adults.
HHS and the health research community should collaborate to educate the public about the importance of research and how it is conducted. Every stakeholder, including professional associations, charitable foundations, and patient organisations, has a vested interest in informing their respective populations.

This could be accomplished by utilising community-based participatory research more frequently, which entails community-based organisations or groups including people from the community as partners in the research process to assist in the design of studies and the dissemination of the information acquired39. These organisations assist researchers by combining their understanding of local health issues and their ability to create activities that the community is likely to find valuable.

RECOMMENDATIONS AND SUMMARY

The committee decided on a second overarching concept to direct the formulation of recommendations after reviewing the data detailed in this chapter. The group emphasises the importance of preserving and enhancing the efficacy of health research. Findings from research are essential to prolonging the length of healthy lives. Increased life expectancy, decreased infant mortality, a reduction in the impact of infectious diseases, and improved outcomes for patients with heart disease, cancer, diabetes, and other chronic diseases are all results of research into the causes of disease, methods of prevention, techniques for diagnosis, and new approaches to treatment. Clinical research that is patient-centered and examines novel concepts enables quick advancements in medicine. As a result of recent advancements in our understanding of the genetic causes of illness, the pace of discovery is quickening, and we are on the cusp of a spectacular moment of investigative potential. Genomic research is creating new opportunities for disease prevention and the creation of safer, more efficient medical treatments that may someday be customised for particular patients. It will be necessary to leverage a significant quantity of already-existing health-related data and archived tissue specimens in order to make further advancements in linking genetic information to illness propensities and treatment responses. The usage of electronic medical records is expanding, which will speed up discovery and make it easier to generate new information via research. For these initiatives to be successful, a large number of patients must participate in the study, and data must be shared widely in order for the findings to be reliable and relevant to many demographic groups. Collaborations between patient populations, their doctors, and research teams to advance scientific understanding will directly benefit citizens of our nation and other nations.
According to surveys, the majority of Americans are interested in the results of research studies, believe that healthcare research is important, and are willing to participate in it. Patients, on the other hand, are rarely informed about study findings that may directly affect their health and frequently lack knowledge about how research is carried out. Effective communication may increase the public’s confidence in the research community, which is critical because public engagement in research is essential.

As a result, the committee recommends that when patients agree to the use of their medical information in a specific study, health researchers should make a greater effort to educate study participants about the findings as well as their relevance and significance. While more people using electronic health records may be beneficial, standards and norms for communicating study findings to research participants or different segments of the population are also required.

In order to make information on research studies more widely accessible to the public, HHS should also promote the registration of trials and other studies in public databases, especially when research is carried out with an IRB/Privacy Board authorised waiver of consent or authorization. Although there are many clinical trial registries and the number of registrations has grown recently, there is currently no centralised system for disseminating information about drug or other intervention clinical trials, making it challenging for consumers and their healthcare providers to locate ongoing studies. Furthermore, these databases often do not include noninterventional studies, such as observational studies, which are becoming increasingly important in biomedical research. The inclusion of such research in a registry might be a significant strategy for raising public awareness of such studies since many noninterventional studies are carried out with an IRB/Privacy Board authorised waiver of permission or authorisation.

The most well-known types of health research are interventional clinical trials, but much information-based health research involves studying thousands of patient records in order to better understand human diseases, assess the efficacy of treatments, and identify unfavourable side effects of therapies. As electronic health information becomes more widely available, this type of study is expected to become more common.

According to surveys, many patients want not only notification but also the opportunity to choose whether to agree to such study using their medical data; however, many patients are likely unaware that their medical records are being used in information-based research. Strengthening security safeguards for health data, as mentioned in Chapter 2, could lower the likelihood of security breaches and their potentially harmful effects, and should thus help to allay patients’ fears.

Furthermore, HHS and researchers must emphasise the negative effects of incomplete datasets on research results, as well as the importance of healthcare advances resulting from medical records research. Health research requires representative samples to be credible and generalizable, but if some people hide their health information from researchers, databases will not accurately reflect the community as a whole.

It will be crucial for HHS to also provide further advice on how to differentiate the various activities in order to guarantee that good health research and associated activities continue to be carried out with adequate supervision under federal rules. The Privacy Rule distinguishes between health research and those activities that are closely connected to it, such as quality improvement and public health initiatives, which may also require gathering and analysing personally identifiable health information. These activities, which seek to safeguard the public’s health and enhance the standard of patient care, are regarded as health care “practise” as opposed to health research under the Privacy Rule (as well as the Common Rule). As a result, they may be carried out without permission, authorisation, or an IRB/Privacy Board waiver of such. However, it can be difficult for IRBs and Privacy Boards to determine which activities fall under or do not fall under the different provisions of the Privacy Rule and the Common Rule. As a result, poor decisions may prevent important activities from being carried out or may even allow the improper disclosure of personally identifiable health information.

To address these issues, a number of models have been developed that specify the standards that IRBs and Privacy Boards must follow in order to differentiate between practise and research. For example, one recent model provides a comprehensive checklist for IRBs and Privacy Boards to use in determining whether an activity is public health practise that does not require IRB/Privacy Board approval or public health research that must adhere to the Pri
HHS should gather the relevant parties in order to provide uniform standards for IRBs and Privacy Boards to apply in determining whether procedures include research or practise. The law should be flexible enough to allow significant initiatives to proceed with the appropriate amount of monitoring. It will be critical to assess how well these criteria work in assisting IRB/Privacy Board evaluations of proposed procedures and if they result in the right judgements for these bodies.

Making the aforementioned changes a prerequisite for getting funds from HHS and other research sponsors, as well as providing some additional funds to cover the expenditure, might be accomplished without changing HIPAA.

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