Addressing Challenges in the
Recruitment and Retention of
Rare Disease Research Cohorts
Guidance Document, Version 1
Prepared by PCORnet Rare Diseases Task Force
Intent and audience: This guidance document is intended for PCORnet-affiliated researchers who are
planning or implementing recruitment and retention activities for PCORnet rare disease research
cohorts. The document was collectively written and edited by the inaugural members of the PCORnet
Rare Diseases Task Force (RDTF). The content includes expert guidance from the authors and
contributors. The information contained in this document is not presumed to be exhaustive,
representative, or authoritative. It is envisioned that this document will be updated periodically as new
resources and approaches become known. When the document has been approved by PCORI program
officers, it will be made available across PCORnet on the Central Desktop. Broader dissemination as part
of the NIH Collaboratory Living Textbook for Pragmatic Trials
(http://sites.duke.edu/rethinkingclinicaltrials/) can be considered. In addition, this content, plus some
description of the PCORnet and the RDTF, might be of interest to rare disease–specific journals (e.g.,
Orphanet), and if time and interests permit, RDTF members will consider adapting it to a manuscript for
submission and review in the future.
Submitted to the PMO
Approved by the PMO
Submitted to PCORI
Comments received by PCORI
Approved by PCORI
The Rare Diseases Task Force
13NOV2014
09DEC2014
09Dec2014
03Jan2015
20May2015
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Overcoming Challenges in Rare Disease Cohorts
Rare Diseases Task Force
Addressing Challenges in Recruitment and Retention of
PCORnet Rare Disease Cohorts
CONTENTS
OVERVIEW OF PCORNET ................................................................................................................- 3 OVERVIEW OF THE PCORNET RARE DISEASE TASK FORCE ................................................................- 3 RECRUITMENT AND RETENTION .....................................................................................................- 4 LAYING THE GROUNDWORK ........................................................................................................................ - 5 RECRUITMENT........................................................................................................................................... - 7 RETENTION ............................................................................................................................................. - 10 REFERENCE LIST ....................................................................................................................................... - 12 RESOURCES.................................................................................................................................. - 13 -
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OVERVIEW OF PCORNET
PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the
Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s
capacity to conduct comparative effectiveness research efficiently by creating a large, highly
representative network for conducting clinical outcomes research.
PCORnet seeks to transform clinical research by engaging patients, care providers, and health systems in
collaborative partnerships to improve healthcare and advance medical knowledge. By bringing research
and patient care together, this innovative distributed research network will be able to improve the
efficiency with which research that explores the questions that matter most to patients and their
families is conducted.
PCORnet will integrate data from multiple Clinical Data Research Networks (CDRNs) and PatientPowered Research Networks (PPRNs), all selected by and contracted by PCORI. The CDRNs are working
to develop the capacity to conduct randomized comparative effectiveness studies using data from
clinical practice in a large, defined population. These established or newly developed networks involve
two or more healthcare systems, with plans to function as integrated research network. The PPRNs
comprise patients and/or caregivers who are motivated to build an ideal network and play an active role
in patient-centered comparative effectiveness research (CER). A description of the inaugural networks
can be found at http://pcornet.org/member-networks/.
In addition to the CDRNs and PPRNs, PCORnet also includes a Coordinating Center. The major
components of the Coordinating Center serving PCORnet include an Executive Leadership Committee, a
Program Management Office, and 11 integrated Task Forces covering essential functions. In particular,
the Rare Diseases Task Force was established to support CDRNs and PPRNs in identifying populations,
developing research priorities, designing, and implementing studies for rare diseases. Please visit
www.pcornet.org for additional information.
OVERVIEW OF THE PCORNET RARE DISEASE TASK FORCE
Rare Disease Task Force description: The Phase 1 PCORnet Rare Disease Task Force is comprised of 37
members from more than 26 organizations representing conditions such as (but not limited to)
Duchenne and Becker muscular dystrophy, Phelan-McDermid Syndrome, hypothalamic hamartoma,
Dravet Syndrome, and tuberous sclerosis, co-infection with HIV and hepatitis C, Hypoplastic left heart
syndrome, and amyotrophic lateral sclerosis (ALS). For a complete list of rare disease conditions
represented by the Rare Disease Task Force, please refer to pcornet.org.
Co-led by clinicians and researchers with more than 50 years of collective and diverse experience in the
rare disease domain, the RDTF members bring perspectives and contribute insight from a variety of
experiences such as assessing the needs of rare disease communities, recruiting and retaining research
cohorts, and writing protocols and serving in other capacities to advance knowledge needed for the
treatment and management of rare conditions. While some Task Force members are new to the rare
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disease space, others have more than 20 years of experience in their roles as clinical researchers;
biomedical researchers, social scientists; and policy/legal specialists. Additionally, patients and patient
advocates are represented.
RECRUITMENT AND RETENTION
A key tenet of PCORnet is engaging patients in all steps of the research process, from the development
and prioritization of research questions, through data collection, dissemination and publication, to
integration of results into healthcare practices. Engaging patients with rare diseases in each of these
activities involves unique challenges and opportunities; however the Rare Disease Task Force (RDTF)
members have found that addressing challenges and highlighting opportunities related to recruitment
and retention are of particular importance. As such, this guidance document provides an overview of
potential strategies for increasing patient recruitment and retention.
Recruitment and retention of rare disease research cohorts is challenging because of several key factors.
Each rare disease affects a small percentage of the population; in the United States, a rare disease is
defined as one in which fewer than 200,000 Americans are affected.1 Identifying members of rare
disease populations is difficult because of broad, national and international geographic distribution of
patients. Together, these two factors contribute to the overall challenge of very small sample size.
Small sample size also impacts other areas related to recruitment and retention. For example, the
relatively small sample size introduces the need for a larger percentage of patients to achieve a
representative sample for many research studies. Large percentages of rare disease cohorts are difficult
to achieve when multiple studies compete for similar patients. Small sample size also introduces a need
for heightened measures to provide security and protection of participant anonymity because identity
cannot be ‘lost in the numbers’.
In addition to recruitment challenges, retention of participants in rare disease research in part because
rare disease research studies often extend over long periods of time and require a wide spectrum of
healthcare encounters. Participants with aggressive forms of a rare disease often have limited ability to
participate in studies with long duration. Other factors that impact retention of participants in rare
disease research include: The heightened need for transparent communication, the need to establish
trust and a culture that demonstrates how the preferences and priorities of patients and families are
valued by the research team.
Despite the many challenges associated with recruiting and retaining rare disease patients in research,
many opportunities exist. For example, individuals with rare diseases (or their parents, family members,
or caregivers) are often highly motivated to participate in research because understanding the disease
and treatment options is critical for managing the illness, increasing quality of life and extending
lifespan. Members of the RDTF noted anecdotally, that rare disease communities sometimes feel
neglected by researchers and are urgent for better disease understanding and new interventions. After
they capture the attention of researchers, however, such families can be at risk for research fatigue.
Fatigue can result from frequent requests to participate in research, the burden associated with
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participating in multiple studies, and the lack of follow up or information reported back to the patient
about how his/her participation can help him/her or someone else in their disease community. Heavily
researched communities may develop preferences for participating in particular types of research (e.g.,
comparative effectiveness over quality-of-life studies) or communities may develop an aversion to
certain research approaches (e.g., unwillingness to participate in clinical trials using a placebo-controlled
design). The availability of multiple clinical research opportunities often leads to difficult decisions for
patients and families, who may have to choose among potentially beneficial options using uncertain or
incomplete information.
LAYING THE GROUNDWORK
It is important to identify strategies to address the challenges, to share lessons learned from experiences
and to provide anecdotal references for understanding the complexities of recruitment and retention in
the rare disease population. Although many of these approaches are relevant to both common and rare
conditions, all the ideas suggested by RDTF members have been included below so that this compilation
might serve as an in-depth resource for recruitment and retention of trial participants from rare disease
cohorts.
As an organizing framework for these strategies, we adapted a conceptual model from Ford et al.2 that
describes three components that may promote participation in a clinical trial:
1. Awareness of clinical trials. This may include outreach and collaboration with rare disease
organizations to engage patients, offering information about the rare condition, discussing
trial/study possibilities, and presenting workshops or focus groups.
2. Opportunity to participate. This may include helping to address potential costs or burdens of
participation such as transportation concerns, time commitments, or other logistical issues.
3. Decision-making. This may involve promoting a willingness to participate by conveying benefits
or offering incentives (financial or nonfinancial).
Many of the approaches and strategies described here will be useful in all phases of engaging
participants—from laying the groundwork through recruitment and retention.
The following approaches may be helpful in laying the groundwork.
Awareness

Be transparent and honest: Describe what is known and unknown about the potential risks and
benefits of the research. Because patients and families are often desperate for treatment
advances related to their disease, managing expectations is critical.
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Engage with all organizations: When there are multiple groups serving a given rare disease, try
to develop an impartial work relationship to gain acceptance from the patient community as a
whole.
Plan ahead: Give organizations time to prepare their members to receive information about
potential research in the planning stages.
Be visible: Attend and speak at patient/disease advocacy organization conferences early in
research planning so their constituents can begin to know the investigators and research team
and provide the patient perspective early enough in the process. This helps to build trust and
loyalty for the team’s work in general as well as the in the current study.
Leverage existing relationships: Realize that when researchers have direct contact with the
organizations, organization leaders are better able to talk with their constituents about the
study. This lends credibility to the organization’s work, which in turn increases support for the
organization.
Cast a wide net: When there is no organization serving a particular rare disease, contact
“umbrella” groups that have platforms for many conditions or cross-conditions. There are
several such groups, including the Genetic Alliance3 and the National Organization for Rare
Disorders (NORD).4
Opportunity
 Consider the patient’s perspective: Develop or refine the research question to meet a true
need of the community.
 Patient-friendly approach: Use methods that are acceptable to the patient community.
 Minimize the patient investment: Consider the study design and proposed workflow from the
perspective of study participants; seek opportunities to reduce the time and resource
participants and their families must invest.
 Think outside of the box: Understand alternative paths for recruitment and dissemination of
information.
Decision-making
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Transparent motives: Help patients and family members know why you are interested in this
condition. Describe your work and show them why their help and participation are essential.
Patients appreciate knowing the project’s final goal, so discussing short-term, intermediate,
and long-term goals is critical. For example, a natural history study is considered an immediate
short-term goal, which then leads to the long-term goal of a new therapy.
Community engagement: Invite patients or patient representatives to be members or advisors
of the research team.
Show the impact: Utilize patient and community input to make decisions and be transparent
about where and how their input was impactful.
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RECRUITMENT
There is a limited patient pool for recruitment in rare disease communities, which amplifies the need for
customized recruitment activities. To develop focused strategies, it is important that researchers
understand the sentiments of the affected community, including their thoughts, attitudes, and beliefs
about research and their personal interactions with both healthcare and clinical research. Authentic
engagement involves both parties listening intently for mutual benefit, rather than reaching a
predefined end result.
In the high-stakes recruitment for clinical trials, sponsors need to be aware of competing research
opportunities and must adhere to the highest ethical and moral standards in recruitment. Conditionspecific foundations may be able to inform sponsors of competing research and suggest approaches to
engaging the community effectively and fairly.
Additionally, identifying potential participants for rare disease research can be challenging because of
widespread geographic distribution or lack of precise ICD codes or other signals in electronic health
data. Identification of new participants may require significant time and effort per individual case and
can often cost more per case than a research program for a common disease. Prospective researchers
should anticipate and budget for costs related to identifying individuals affected by rare diseases. In
many cases a confirmation (often molecular) of diagnosis is needed; it is recommended that this step be
built into the recruitment phase.
While there is no generic, standard approach for recruitment in rare diseases, the specific condition
might make some outreach approaches more feasible than others. For example, for rare conditions with
fewer than 100 patients, personal emails or phone calls might be feasible but likely would not be for
rare conditions affecting thousands of individuals. Often, rare disease networks are available on the
internet or social media that can help in identification and recruitment, but integrating messages about
recruitment into social support sites can be a challenge.
Successful strategies for identifying and recruiting patients involve engaging patients and patient
support organizations; two-way communication and seeking input early; respecting the patient’s limited
time and resources for research; and empowering patients with information and opportunities for
networking, learning, and action. When individuals are managing debilitating diseases that require
considerable time and effort, be mindful to impose as little additional burden to participation as
possible. The following approaches have proven useful for recruiting participants in rare disease
research initiatives and will be updated periodically by RDTF members as they complete their PCORnet
research activities.
Awareness

Step Out: Engage with the rare disease communities where they already convene (e.g.,
meetings, organizations, clinics, and community groups).
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Focus in: Conduct focus groups or other outreach with multidisciplinary stakeholders to identify
unique or particularly effective recruitment strategies. (See PCORnet investigators from
Vanderbilt for examples of the Patient Engagement Studies for one model.5)
Ask questions: Engage with communities to ask (and convey your intent to answer) questions
that are important to the disease community.
Send the right message: Work with organizations to develop the recruitment message. When
possible, customize materials to address pervasive concerns or overcome ambivalent or
negative attitudes toward research, while taking care not to overstate potential benefits or
create coercive communications.
Leverage technology: Use social media to announce study options. This may be most effective
and acceptable to regulatory bodies when done in collaboration with a disease-focused
foundation.
Leverage existing forums: Take advantage of rare disease community resources; for example,
host webinars or other meetings through foundation networks or attend conferences and
events sponsored by the community.
Opportunity
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Plan for the future: Analyze comments and suggestions provided by patients and families for
themes for future research of specific interest to the disease community.
Consider the usability factor: Develop protocols and materials that are sensitive to patients’ and
family members’ perspectives.
Be realistic: Involve patients and caregivers in the clinical study design to ensure that inclusion
and exclusion criteria, number of study visits, and study procedures are both acceptable and
reasonable for participants.
Write for the readers: Explore meaningful, but minimally burdensome, consent processes that
take into account disease-imposed limitations, if relevant. There is a critical balance between
making consent simple and understandable while keeping patients fully informed of the risks
of research. We encourage -PCORnet members to share new platforms, strategies, and examples
in this area.
‘Need to know’ vs ‘Nice to know’: Ensure that informed consent documents are complete with
regard to key points that could affect the patient. To the extent allowed by your IRB, avoid the
use of inconsequential notices and warnings that detract from the reader’s ability to
distinguish between real and theoretical risks and between risks of high and low likelihood or
impact. PCORnet members are encouraged to share example consents on the PCORnet Central
Desktop to help others in this area.
Integrate research and care delivery: Integrate recruitment activities into the support and
clinical care flow for patients when possible.
Use a multifaceted approach: Collect information needed to connect with patients (or their
families) by several methods (phone, text, email, etc.) to reduce the chance that they will be
lost to follow-up.
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Engage locally: When blood draws are needed, make arrangements for participants to have
them drawn locally. Cover costs of sampling and provide prepackaged/labeled containers with
all necessary information and instructions.
Empower the local experts: Identify clinicians who see patients with the condition and enlist
their support. Familiarizing them with your research lets them have meaningful discussions
when a patient brings up the proposed study in a medical visit.
Home court advantage: When research participants are children or adults unable to travel
alone, it may be difficult for a parent or caregiver to take time off work to go to a research
location, even if they are motivated. Efforts to minimize travel requirements and distance are
appreciated. Examples include partnerships with local clinicians or researchers to ease travel
burden.
Leverage available resources: When travel to the research location is unavoidable and study
funds are not provided, help participants connect with other resources to expedite travel (e.g.,
Miracle Flight). Or, for families of children, provide low-cost or no-cost accommodations (e.g.,
Children’s Inn, Ronald McDonald House). Meal vouchers and information about local patient
organizations and connections to medical social work teams can be helpful and are often
appreciated.
Go where the people are: Leverage existing meeting opportunities and resources. For
example, integrate with other groups who serve the affected individuals; engage in
established meetings such as Individualized Education Plan (IEP) discussions or with established
online resources such as web-based disability resources, etc. Leaders of organizations can assist
with this.
Go paperless: Keep paperwork to a minimum and send it to potential participants in advance.
Move toward paperless and consider getting information via interview rather than in writing, or
give participants a choice in mode of information collection.
Connect the dots: When connections are made with people who are not affiliated with an
organization, offer information about relevant organizations. If patients have trouble making
contact, offer to facilitate.
Knowledge is power: Offer incentives, if possible. The rare disease community is often
incentivized by knowledge more than by money. If a monetary incentive is offered, give them
the option of having it in the form of a donation to the organization.
Decision-making

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Explain the rationale: Include in the information flow a message that informs patients what the
research can mean to them. Even if the knowledge gained through research is general, people
affected by the condition want to know the research will enable providers to make informed
treatment decisions and plan future research. Researchers can help patients and families
understand that although research is not the same as clinical treatment, it is beneficial and
important to them and to their future.
Create a value statement: Communicate the benefits of research participation to the community
and to the individual participant (e.g., in the form of access to information, expertise, and
support).
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Build trust: Ensure that the data will be collected, maintained, and distributed by a trusted
source. In the registry context, this should be a community source that aims to speed diseasespecific research. Communicate this message to research sponsors and partners, including
patients and disease advocacy groups. Research participants and their families want to know
that their data will be used to support future research, and knowing that a research team is
committed to sharing data and results can enhance participation.
Report back: Provide feedback to disease community resources, such as how many people
have participated in the research so far and how many more you need, or when scientific
results will be presented (e.g., at meetings). Organizations can relay this information so that
individuals receive it from several sources.
You’re invited: Host patient-focused meetings or reunions and incorporate research discussions
at those times.
Peer to peer communication: Ask participants to help spread the word to other potential
participants.
Teach the man to fish: Provide participants with vetted resources (e.g., educational materials,
online tools) for assistance with issues they may encounter as part of their condition.
Facilitate connections: If an organization does not currently exist to serve people with the
condition, join with a patient or family to organize support group meetings at your location or
find a way for participants who are geographically distant to connect with each other. Help
them build a community that you can re-connect with in the future.
Remember that the quality of interaction you have with people involved in the study, and their families
and caregivers as appropriate, is important. They need to know they are not alone, that they are doing
a great job, are valued, and have unique talents. Give lots of positive reinforcement.
RETENTION
Given the low number of people with rare conditions and the difficulty recruiting, it is critical that
participants remain in the study or registry and continue to answer questions and update information.
However, such patients are often coping with serious disorders, or dealing with a healthcare system that
is unequipped or uninformed about how to provide them with care, or needing social services and
supports that are typically difficult to access or maintain.
Patients with rare diseases are often strong advocates for research, yet they must perceive some
benefit—or only limited burden—to remain engaged in the process. RDTF members have identified the
following successful strategies for retaining participants in research.
Awareness


Impact the future: Empower study participants; emphasize how they are making a difference
not only in their own lives but also in every patient diagnosed after them.
Be an ambassador: Motivate participants to understand and address unanswered questions in
the community.
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Engage the support systems: Conduct outreach to “navigators”—people who help patients
traverse the condition from point of diagnosis until they are independently managing life with
the condition.
Leverage technology: Use social media to provide updates on studies (e.g., recruitment efforts,
preliminary results, upcoming activities).
Foster connections: Host events for participants and researchers to gather and meet one
another.
Capture the moment: Take photos and post on social media (with photo permissions in
advance).
Learn from the experts: Invite leaders of organizations to your events and ask them to speak.
Share results: During study design and start-up, researchers and stakeholders can
collaboratively develop a dissemination plan for real-time updates to study participants and
stakeholders. Share results of final analyses at advocacy conferences, via webinars, or by
creating a brief description for social media, newsletters, etc. Note: Brief lay summaries often
can be shared without having an adverse impact on the researcher’s ability to publish.
Opportunity
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Patients can decide: Consider forms of dynamic and granular engagement that allow the
participant to make choices about what information they will share, how they will share it,
and with whom it can be shared.
Leverage technology: Reduce the burden of updating information by using relevant
technologies.
Give a gift Tie requests for new or updated information with a “give back” to the participant
such as an educational or support tool.
Stay local: Minimize the number of visits requiring travel to clinic.
Engage patients in prototype development: Develop user-friendly web-portals and pilot-test
them with participants.
Compensate for travel: Provide financial support or other forms of assistance to facilitate travel
to study sites when required.
Celebrate: Send birthday and holiday cards from the study team as a way to show your
continued interest and to keep up-to-date contact information.
Decision-making

Provide context: Allow participants to view their own score for an assessment in the context of
an aggregate score for measures submitted by all comparable participants. Alternatively,
provide participants with some framework in which they can interpret their responses to
individual items on a questionnaire in the context of the responses submitted by the greater
participant community.
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Build on prior work: Explore opportunities to repurpose registry data in clinical tools for
patients or health care providers; similarly, explore opportunities to leverage data that is
available from patients and health care providers for use in research.
Expand authorship: Name the organization and their leaders as coauthors of your research.
This approach enhances collaboration and allows them a larger stake in the research as well as
helping with patient retention and follow-up.
Lessons learned: Ask research participants what they would do differently if they were the
researcher.
Share resources: If the research team finds a new resource, send it to the organization or
directly to the patients in the study.
Spread the word: Formally publish research results and share the manuscripts with study
participants.
Describe the use: Document and report the number of researchers who cite these publications
to facilitate connections across research initiatives.
In summary, the PCORnet RDTF offers a collection of recommendations to support the recruitment and
retention of patients in rare disease research. Themes including empowerment, education, and
transparency emerge as essential components of an effective plan to recruit and retain qualified
participants in efforts that advance knowledge and science needed to improve the health and quality of
life for patients, families and caregivers whose lives are affected by rare conditions.
REFERENCE LIST
1.
U.S. Food and Drug Administration. Available at:
http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/
SignificantAmendmentstotheFDCAct/OrphanDrugAct/default.htm. Accessed November 10, 2014.
2.
Ford JG, Howerton MW, Bolen S, et al. Knowledge and access to information on recruitment of
underrepresented populations to cancer clinical trials. Evid Rep Technol Assess (Summ)
2005(122):1-11. PMID:15989377.
3.
Genetic Alliance. http://www.geneticalliance.org/.
4.
National Organization for Rare Diseases. https://www.rarediseases.org/.
5.
Wilkins CH, Joosten Y. Improving Patient Engagement and Understanding Its Impact on Research.
NIH Collaboratory Grand Rounds Presentation. August 2014. Available at:
https://www.nihcollaboratory.org/Pages/Grand-Rounds-08-29-14.aspx. Accessed November 10,
2014.
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RESOURCES
A number of general resources exist to support the recruitment and retention of participants in clinical
research. This list is not exhaustive or limited to rare diseases but should be helpful to inform general
approaches and guidance.
Resource Name
Location
1
Brescia BA. 2013. Two Sides of the Rare
Disease Coin and Three Insights Into Patient
Retention
http://innovations.bbkworldwide.com/bid/176
555/Two-Sides-of-the-Rare-Disease-Coin-andThree-Insights-Into-PatientRetention#gsc.tab=0
2
Chapter 20: Rare Disease Registries. In
Registries for Evaluation of Patient Outcomes,
3rd Edition: A User’s Guide
http://www.ncbi.nlm.nih.gov/books/NBK2086
09/
3
DeWard SJ, et al. 2014. Practical Aspects of
Recruitment and Retention in Clinical Trials of
Rare Genetic Diseases: The Phenylketonuria
(PKU) Experience
http://www.ncbi.nlm.nih.gov/pubmed/240141
52
4
Forsythe LP, et al. 2014. A Systematic Review
of Approaches for Engaging Patients for
Research on Rare Diseases
http://www.ncbi.nlm.nih.gov/pubmed/250473
93
5
Gupta S, et al. 2011. Rare Lung Disease
Research: Strategies for Improving
Identification and Recruitment of Research
Participants
http://www.ncbi.nlm.nih.gov/pubmed/220458
77
6
Kaye J, et al. 2012. From Patients to Partners:
Participant-Centric Initiatives in Biomedical
Research. Nature Reviews Genetics, 13, 371376.
http://www.nature.com/nrg/journal/v13/n5/a
bs/nrg3218.html
7
Kaye J, et al. 2014. Dynamic Consent: A Patient
Interface for Twenty-First Century Research
Networks. Eur J Hum Genet.
http://www.ncbi.nlm.nih.gov/pubmed/248017
61
8
Orphan Reach: Patient Retention
http://orphan-reach.com/rare-diseasefocus/optimize-patient-retention
9
Participant Engagement: Tools to Meet People
Where They Are (NIH Collaboratory Grand
Rounds Presentation by Sharon Terry)
https://www.nihcollaboratory.org/Pages/Gran
d-Rounds-02-21-14.aspx
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Resource Name
Location
10
Partnering With and Engaging Families in
Clinical Research (NIH Collaboratory Grand
Rounds Presentation by Megan O’Boyle)
https://www.nihcollaboratory.org/Pages/Gran
d-Rounds-08-15-14.aspx
11
Strange C, et al. 2014. Social Impact Identified
In and By the Alpha-1 Antitrypsin Deficiency
Community. Rare Diseases and Orphan Drugs
http://rarejournal.org/rarejournal/article/view
/59
12
Terry SF, et al. 2013 The Haystack is Made of
Needles. Genet Test Mol Biomarkers
http://www.ncbi.nlm.nih.gov/pubmed/?term=
23428177
13
The Use of Social Media in Clinical Research
(NIH Collaboratory Grand Rounds Presentation
by Stephanie Startz)
https://www.nihcollaboratory.org/Pages/Gran
d-Rounds-03-21-14.aspx
14
WikiAdvocacy, Functions of Advocacy
Organization
http://wikiadvocacy.org/index.php/Functions_
of_Advocacy_Organization#Support
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