MCCRSCC Improving Enrollment Capstone

Research Capstone Outline

I am needing a draft of the Capstone Clinical Research Paper.

Topic: A review on how to improve enrollment for 1st time clinical trial pediatric subjects diagnosed with Duchenne Muscular Dystrophy

I have attached all information that has been provided and or completed so far and items that need to be reviewed and incorporarete into this draft including the grading rubric

Please select a Clinical Study Design to present the information from:

Brief Descriptions of Study Designs:

  • Qualitative: These types of research methods involve describing in details specific situation using research tools like interviews, surveys, and Observations.
  • Quantitative: These types of research methods require quantifiable data involving numerical and statistical explanations.
  • Correlation/Regression Analysis: This research method involves determining the strength of the relationship between two or more variables (e.g., are violent video games correlated with aggression in children).
  • Quasi-Experimental: This research involves the comparison of two groups, one which is influenced by an external source and another which is not influenced by an external force.
  • Experimental: Use of random assignment to place participants in two groups: an experimental group which receives intervention, and another control group without any intervention. It is using a positive control for you to base it or compare it in your result.
  • Meta-Analysis: This research method is useful for finding out the average impact of several different studies on a hypothesis.
  • Qualitative: These types of research methods involve describing in details specific situation using research tools like interviews, surveys, and Observations.
  • Quantitative: These types of research methods require quantifiable data involving numerical and statistical explanations.

  • Correlation/Regression Analysis: This research method involves determining the strength of the relationship between two or more variables (e.g. are violent video games correlated with aggression in children).
  • Quasi-Experimental: This research involves the comparison of two groups, one which is influenced by an external source and another which is not influenced by an external force.Experimental: Use of random assignment to place participants in two groups: an experimental group which receives intervention, and another control group without any intervention. It is using a positive control for you to base it or compare it in your result.Meta-Analysis: This research method is useful for finding out the average impact of several different studies on a hypothesis.

Please see the details below for the table of contents:

Chapter Page # Chapter 1 (


) t Chapter 2 (

Literature Review

) u Chapter 3 (Methods) v Chapter 4 (


) w Chapter 5 (Discussion) x References y Appendices z

Please see the following guidance that has been provided:

Capstone Goals and Objectives

  • Goals are broad statements of what the capstone hopes to accomplish. They create a setting for the capstone. Specific objectives are statements of the capstone research question(s). Objectives should be simple (not complex), specific (not vague), and stated in advance (not after the research is done). After statement of the primary objective, secondary objectives may be mentioned.

Study Design – **This section may not be applicable to your project

  • The scientific integrity of the capstone and the credibility of the study data depend substantially on the study design and methodology. The design of the study should include information on the type of study, the research population, or the sampling frame, and who can take part (e.g., inclusion and exclusion criteria, withdrawal criteria etc.), and the expected duration of the study.
  • The same study can be described in several ways, and as complete a description of the study as possible should be provided. For example, you may want to describe the study as being epidemiologic or health policy or health and social behavioral research. It may also be described as observational or interventional; if observational, it may be either descriptive or analytic, if analytic it could either be cross-sectional or longitudinal etc. If experimental, it may be described as a controlled or a non-controlled study (this is not a comprehensive list).


  • The methodology section is the most important part of the protocol. It should include detailed information on the interventions to be made, procedures to be used, measurements to be taken, observations to be made etc. If multiple sites are engaged in a specified protocol, methodology should be standardized and clearly defined.
  • Interventions should be described in detail, including a description of the intervention, education, training etc. provided to groups or individuals.
  • Describe the procedures to be conducted. For example: a questionnaire survey, carrying out a focus group discussion as part of formative research, observation of the participant’s environment, etc.).
  • Standardized and/or documented procedures/techniques should be described and bibliographic references, if not provided earlier should be provided. Instruments which are to be used to collect information (questionnaires, FGD (Focus Group Data) guides, observation recording form, case report forms etc.) must also be provided.
  • This week we will review these sections of the methodology chapter. Certain elements may not apply to your paper.
  • This section of your paper is a detailed account of exactly what you are going to do or what you did. The key is to be very specific and not miss anything. Think of this as a recipe from a cookbook. What you will write in your paper will tell your readers whether your results are valid, reliable, and could be used with confidence.
  • For a capstone, you need to share the rationale behind what you are doing and that each of your choices was a conscious and correct choice.
  • Remember – when writing this section, everything changes to future tense. This is what you are going to do!
  • Design of Study
  • Setting
  • People Involved and Resources Needed
  • Sample, including Access and Recruitment Methods

Ethical/Safety Considerations**This section may not be applicable to your project

  • The protocol should have a description of ethical considerations relating to the study. This should not be limited to providing information on how or from whom the ethics approval will be taken, but this section should document the issues that are likely to raise ethical concerns. It should also describe how you, the investigator, plan to obtain informed consent from the research participants (the informed consent process).
  • The safety of research participants is foremost, whether online or in person. Safety aspects of the research should always be kept in mind and information provided in the protocol on how the safety of research participants will be ensured. This can include procedures for recording and reporting adverse events and their follow-up, for example. It is useful to remember that even administering a research questionnaire can have adverse effects on individuals – think about confidentiality or questions that may upset.

Additional Guidance Provided:

The first thing you will tell the reader is what kind of study or project you are doing and why it is a good fit for your capstone research question. These can be broad approaches such as qualitative, quantitative, mixed methods, or a specific design such as narrative.

Brief Descriptions of Study Designs:


: The proposed study will use a quasi-experimental design with electronic surveys administered to participants before and then 6 months following the implementation of the new teaching program.

You can use articles or books to back up your choices on your approach.

Capstone Setting:

Where is your study taking place? You will need to place it within its geographic setting as well as local ( such as a facility or organization).

Provide enough background information about the setting so the reader understands the context in which the study or problem is taking place.

Details can include:

  • Population demographics
  • Socioeconomic factors
  • Environmental factors
  • Cultural factors.

If you are doing a quality -improvement project, include all of the facility or organization information that the reader needs to understand the problem. This may include a description of the organizational culture in addition information related to:

  • Administration
  • Management
  • Leadership structures
  • Financial data
  • Staffing
  • Involved populations, such as patients.

Anything that could influence the implementation or outcomes of your project, for better or worse, should be discussed.

People Involved and Resources-

You may need to describe the people who are involved with your project.

  • Who are these people?
  • Who are the key stakeholders?
  • Who is instrumental in moving your project forward?
  • Who will ensure you have the resources, including staff and finance that you need to move forward?
  • Who will provide you the necessary data (such as admission reports)?
  • What are the resources you need to implement or move this project forward?Human resourcesMaterial Resources (this can include computer, software, teaching supplies, etc.)Financial

Sample Population

  • What population are you drawing your sample from?
  • Who is going to participate in your study or project?
  • How many participants do you need? Is there a minimum?
  • How are you going to get them?

You need to first look at the type of sample design you are going to use ( convenience, purposive, random, consecutive) and why you chose that design.

Next, you need to establish your sampling frame (the general population from which you will recruit your participants). Example:For a study of bereaved parents of estranged children, the sampling frame might be parents attending a grief support group in a particular region of the United States.

Then you must tell us who you want to recruit from that population.

You need to specify exactly who qualifies to participate

  • Develop inclusion and exclusion criteria (I/E)
  • Characteristics for I/E may includeDemographics (age, sex, educational level, marital status, ethnicity, occupation)Presence or absence of an illness or health conditionDuration of illness or health conditionNumber of years phenomenon being experience
  • Professional role
  • Primary language
  • Literacy
  • Previous experience with intervention
  • Location


The study will include a purposive sample of women in western Uganda who have experienced obstetric fistula. Inclusion criteria are women 18 years of age and older with a vesicovaginal or rectovaginal fistula secondary to prolonged obstructed labor. Exclusion criteria will be women who have had their fistula for longer than five years so that the data reflect current sociocultural norms.

Bias in Research

Bias can occur in theplanning, data collection, analysis, and publication phases of research. Understanding research bias allows readers to critically and independently review the scientific literature and avoid treatments which are suboptimal or potentially harmful.

As you develop your project, take time to see if you have identified any potential “bias” in your approach, in your population, and in your analysis. How will you approach bias that you identify?


  • Topic Overview
  • ChallengesSite Concerns and ChallengesPotential Participant Concerns and Challenges

Literature Review

  • Review of existing literature
  • Additional of new literature that supports the challenges
  • Additional of literature that


  • Needs to be decided upon


  • Proposed recommendations/ focus on techniques to be used

Discussion Conclusion

Author: Jeff Somers, Mediaplanet
Read online.
Uniting to Meet the Challenge of Rare
Diseases in 2022 and Beyond
Around the world, a renewed focus on rare diseases and
orphan drugs is beginning to move the needle – but there is
more work to do.
A common misconception about rare diseases is that they are extremely rare, but in reality
these conditions are not as rare as one may think. They affect people from all walks of life and
in all economic circumstances. Most rare diseases are genetic and present throughout a
person’s entire life. Many of these disorders are chronic, have no effective treatment, and may
require complex care.
There are more than 7,000 rare diseases in existence, and nearly 300 million people around
the world are living with a rare disease.
The Global Rare Ecosystem
“Rare disease is a public health issue,” says Durhane Wong-Rieger, Ph.D., president and CEO of
the Canadian Organization for Rare Disorders. “More people have a rare disease than have all
cancers combined, cardiovascular disease, or diabetes.”
Shannon Resetich, Global Franchise Head of Rare Diseases at Sanofi agrees and emphasized
that “while the recent pace of discovery and advancement of new therapies and technologies is
impressive, there is still a long way to go to address the unmet needs of patients.”It’s vital to
keep patients top of mind when considering the true impact of this public health issue. “When
I think about the global rare ecosystem, I first think about the long, painstaking journey to
diagnosis,” says Resetich. “Patients often experience a winding diagnostic odyssey which can
sometimes take up to 10 years to receive a proper diagnosis. We know that rare diseases are
not only difficult to diagnose, study, and treat, but also heart-breaking for the person receiving
the life-altering diagnosis.”Sanofi has both witnessed and contributed to changes in the rare
disease landscape dating back to the 1980s. Over these past 40 years, the company has
developed different therapies for the treatment of many rare diseases. Even with that
pioneering legacy, there is more work to do.
After jumping over the hurdle of an accurate diagnosis, challenges still remain for patients. “The
way we have designed our systems is that healthcare is seen as expenditure—as opposed to an
investment in society,” Wong-Rieger notes. “We do have amazing therapies, but can we
continue to invest in their development if they’re not going to have a return on that
The challenge of investment is closely related to the idea of access, which is currently a ‘onesize-fits-all’ approach. “We’ve got about 600 new therapies in the U.S. and 200 in Europe,”
notes Wong-Rieger, “and we continue to shove them into the old model for access. My fear is
that we have reached the breaking point there.”
“Making access equitable is a huge challenge,” adds Flaminia Macchia, executive director of
Rare Diseases International (RDI). “The commitment is to leave no one behind—which means
equity. And equity does not mean giving the same to everybody—it means giving according to
people’s needs. Equity means to do more for those who have less.”
These challenges are real, but Macchia also sees reason for optimism. “I think we are currently
in a good trend,” she says. “We have several new or recent global policy documents, like the
United Nations Political Declaration on Universal Health Coverage that was adopted a couple of
years ago. Another element that makes me positive is the global patient movement, which is
getting stronger.”
Resetich further reinforced the importance of equity of global access, noting “At Sanofi, we are
especially proud of our Rare Humanitarian program, which works to develop sustainable
healthcare systems and increase access to medicines. Further – our focus now and in the future
is to increase the centrality of the total patient in securing information to help guide the
continued pursuit of true innovative solutions.”
Where We Go From Here
It turns out, patient engagement and inclusion is a trend across the board.
Wong-Rieger also highlights the rising inclusion of patients in the rare disease conversation.
“We’re highly engaging the patient as a partner,” she says. “What we’re seeing in the future is
empowering people to actually take a much bigger role in their own health.”
Resetich added “The total patient must be at the core of informing and guiding how we all
approach our work—as we navigate this, our patients will become even more instrumental in
providing invaluable perspectives to help improve our study designs, clinical approach, and the
overall healthcare ecosystem toward better care.”
The amplification of the patient voice is crucial both for gaining attention and resources, and
because medicine is increasingly personal. “The future, in terms of therapy, is going to be
genetically based, highly targeted, and highly personalized,” Wong-Rieger notes. “Our vision for
rare diseases is that each and every patient will have a totally individualized care and treatment
Another hope for the future evolved out of the COVID-19 pandemic. “There has been an
acceleration of the regulatory processes for the COVID vaccines—that could potentially be a
useful model for other disease areas,” says Macchia. “We have also seen innovative access
schemes such as joint procurement. Why not think about a joint procurement type of model for
orphan medicines?”
Scrapping the Silo Mentality
Tackling the systemic and economic challenges to rare diseases and their treatment will require
cooperation. “The global rare ecosystem involves multiple stakeholders—healthcare providers,
patients, patient advocacy organizations, regulators, payors, academia, policymakers, and
more,” notes Resetich. “Uniting is the best chance we have at bettering the lives of patients and
their care partners. The global rare disease community can benefit greatly from all of us
working together towards a common goal, and we owe it to them to do so.”
One way these stakeholders come together is through the World Orphan Drug Congress
(WODC), an annual conference taking place this year July 11-13 in Boston. “The WODC is kind of
a unique beast,” Wong-Rieger says. “It’s an opportunity to advance the research. It is
competitive, cooperative, and collaborative. It is both the for-profit and the not-for-profit
coming together. And there aren’t many other conferences of this type in which the patients
can play such a major role and have such equal access.”
Resetich sees hope in that community. “It is gatherings like the WODC that ensure we all come
to the table to further progress,” she says. “When it comes to thinking about those affected by
rare diseases, I can’t think of anything more important than a collective effort to address

Save 60% to the World Orphan Drug Congress USA 2022 with discount code,
Improvements in Recrutiment and Enrollment
My Dashboard
Arizona State University
Survey Overview
Viewed Started Completed Completion Rate Drop Outs (After Starting) Average Time to Complete Survey
2 minutes
Arizona State University
Q2. What is your age range? (Select 1 Option)
1. 18-25
2. 25-30
3. 30-35
4. 35 and Older
Mean : 3.429 Confidence Interval @ 95% : [2.937 – 3.920]
Standard Deviation : 0.938
Arizona State University
Standard Error : 0.251
Q3. What is your ethical/racial affiliation you most identify with? (Select
1 Option)
1. White
2. African American
3. Latino
4. Asian
5. American Indian
6. Alaskan Native
7. Hawaiian or Other Pacific Islander
Arizona State University
8. Other
Mean : 2.214 Confidence Interval @ 95% : [1.497 – 2.931]
Standard Deviation : 1.369
Arizona State University
Standard Error : 0.366
Q4. What is the role you most closely align with for Clinical Trials
(Interventional/Observational) (Select 1 Option)
1. Principal Investigator
2. Sub-Principal Investigator
3. CRC
4. CRA
5. Project Manager
6. Data Manager
7. Nurse
Arizona State University
Mean : 4.235 Confidence Interval @ 95% : [3.532 – 4.939]
Standard Deviation : 1.480
Arizona State University
Standard Error : 0.359
Q5. How long have you been in your Clinical Trial Role (Select 1
1. 1-3 Years
2. 3-5 Years
3. 5-7 Years
4. 7-10 Years
5. 10 or More Years
Mean : 2.429 Confidence Interval @ 95% : [1.589 – 3.269]
Standard Deviation : 1.604
Arizona State University
Standard Error : 0.429
Q6. Who is primarily involved in the studies you are involved with?
(Select All that Apply)
1. Pediatric
2. Adolescent
3. Adult
Mean : 2.500 Confidence Interval @ 95% : [2.167 – 2.833]
Standard Deviation : 0.761
Arizona State University
Standard Error : 0.170
Q7. Are you familiar with Duchenne Muscular Dystrophy (Select 1
1. Yes
2. No
Mean : 1.571 Confidence Interval @ 95% : [1.302 – 1.840]
Standard Deviation : 0.514
Arizona State University
Standard Error : 0.137
Q8. What types of Recruitment Practices do your studies utilize?
(Select All that Apply)
1. Advertisements-Written/Post Publications
2. Advertisements-Radio
3. Advertisements-Commercials
4. Site Data Base Search
5. Recruitment Companies
Mean : 3.174 Confidence Interval @ 95% : [2.550 – 3.798]
Standard Deviation : 1.527
Arizona State University
Standard Error : 0.318
Q9. What are some typical concerns/issues you see with recruitment?
(Select All that Apply)
1. Financial Reimbursement
2. Travel Concerns
3. Scheduling Concerns
4. Lack of Trust with Clinician
5. Lack of Trust with Personal Information
6. Lack of Education on the Clinical Trial Process
Arizona State University
Mean : 3.861 Confidence Interval @ 95% : [3.319 – 4.403]
Standard Deviation : 1.659
Arizona State University
Standard Error : 0.276
Q10. What do you feel would help increase recruitment and enrollment
into studies? (Select All that Apply)
1. Cold Calling
2. External Marketing Campaigns
3. External Information Publications
4. Remote Platforms (Teleconferencing/Video Calls)
5. Additional Resources (i.e., Social Workers)
Mean : 3.857 Confidence Interval @ 95% : [3.458 – 4.257]
Standard Deviation : 1.079
Arizona State University
Standard Error : 0.204
Q11. What Advances do you think your site and or studies could
implement to increase recruitment/enrollments? (Select All that Apply)
1. Recruitment Companies
2. Partnership with Patient Advocacy Groups
3. Technology Platforms
4. Increased External Marketing Techniques
Mean : 2.621 Confidence Interval @ 95% : [2.227 – 3.015]
Standard Deviation : 1.083
Arizona State University
Standard Error : 0.201
Arizona State University
Improving Enrollment for First-Time Clinical Trial Pediatric Subjects Diagnosed with
Duchenne Muscular Dystrophy
Clinical trials for different conditions and drug development require a lot of rigor for
validity and reliability. The incorporation of patients’ voices in drug development as well as the
testing of drug efficacy and safety are crucial components of clinical trials required to develop
drugs that are responsive to patient needs and characteristics of the target health condition.
Recruitment of patients for Duchenne Muscular Dystrophy (DMD) clinical trials has been
challenging and hence has significantly limited current research on the health condition and
drugs for it. Therefore, this challenge necessitated research on appropriate recruitment
approaches, especially for pediatric patients to become subjects in clinical trials. This paper
reviews the current literature and evidence of the health condition and research approaches for
effective study participants’ recruitment.
Disease Characteristics and Epidemiology
DMD is a genetic x-linked neuromuscular disorder primarily characterized by
progressive muscular wasting. The epidemiology of the condition is relatively low with the
prevalence of 1 out of 5000 males (Paquin et al., 2019). The condition is often diagnosed
between two and five years and can be noted as weakened proximal muscles, delay in motor
development, and abnormal gait (Guiraud & Davies, 2017). It is the most common of muscular
dystrophy conditions and typically affects males (Paquin et al., 2019). The condition affects
approximately 250,000 people in the United States, with thousands of others in other countries
and regions or with unreported diagnoses (Ryder et al., 2017). The quality of life for people with
DMD is significantly affected by muscular dystrophy hence the need for ongoing research on its
treatment and slowing the condition’s progress.
The rate of progression for DMD may vary from one patient to another. Typically, the
patient is diagnosed with delayed motor development and abnormal gait with muscular
dystrophy (Guiraud & Davies, 2017). This will then progress to loss of independent motor
function, with the patient eventually requiring assistive technology for ambulation (Paquin et al.,
2019). Loss of arm function is also experienced at the same time. Eventually, the patient will
succumb to cardiovascular and pulmonary failure (Guiraud & Davies, 2017). During the
relatively short lifespan, loss of independent function and progressive muscle dystrophy have
significant challenges on the patient and their caregivers. The average lifespan is between 20 and
30 years, and hence the patients experience premature death (Guiraud & Davies, 2017).
Non-curative treatments have been developed and implemented in the US with little
success. Treatments can be classified as therapies targeting the DMD defect and those mitigating
secondary pathologies and mechanisms. Currently, only two treatments targeting the DMD
defect have been approved in the US. The first is corticosteroid therapy used to slow the progress
of symptoms and muscular degeneration (Paquin et al., 2019). The second therapy is genetic
therapy, specifically targeting mutations in the DMD gene (Paquin et al., 2019). While
corticosteroids are indicated for all patients, genetic therapy is limited to only about 15% of the
population with DMD, but the efficacy is not yet ascertained (Paquin et al., 2019). Thus, urgent
research on drugs and therapies for DMD is needed to address the current gap in treatment.
Challenges in Previous Studies
With only 250,000 people in the United States (US) was captured by Ryder et al. (2017),
it is challenging to access a sample population that can amount to a credible and reliable research
outcome. Previous researchers have encountered numerous enrollment challenges, especially
because DMD is a rare disease, and there is also consideration for treatment-amenable genetic
subtypes (Peay et al., 2016). Even in cases where patients are obtained, spatial challenges in
conducting research are common (Duan et al., 2021). Additionally, genetic subtypes also differ
in DMD, and enrolling patients for a specific subtype has proved further challenging (Peay et al.,
2016). Therefore, the primary challenge experienced is the rarity of the disease and accessing
appropriate population.
The practical and ethical challenge of conducting placebo arms for this condition can be a
major hurdle. DMD is a progressive condition leading to ultimate death and almost all the
studies aim to slow down progression or stop the progression overall (Goemans et al., 2020).
Therefore, to determine drug effectiveness in real-world data and natural history data, it is
required that a control group is maintained to ensure that changes in the intervention group can
be properly attributed to the treatment (Goemans et al., 2020). It is unethical to withhold
potentially life-saving medications from patients who face imminent death from a health
condition (Goemans et al., 2020). Consequently, recruiting and maintaining patients for both the
intervention and placebo groups remains a major challenge.
The concept of informed consent and deliberations for ongoing controlled trials is another
issue. Informed consent is an ongoing process of deliberation where the decision-makers access
information, appraise it, and make decisions based on it (Peay et al., 2016). DMD trials are
typically conducted with pediatrics, and this which means informed consent can only be granted
by a parent or guardian (Peay et al., 2016). Therefore, When recruiting subjects for participation
in clinical trials, the concept of consent must be considered at the point of view of the parent
from the parent’s point of view. New and emerging information may dissuade parents from the
trials and disagreements between pediatrics and their parents on participation may also hinder the
process (Peay et al., 2016). Despite efforts to use patient-focused drug development (PFDD),
informed consent and subjective patient experiences are not adequately captured in such trials
(Crossnohere et al., 2020).
Perceived Benefits or Harms
Parents and their children considering entering a DMD or another clinical trial will
consider benefits and costs of participating, which will significantly affect their willingness to
participate. Benefits of participating can be defined as any additional gain the patient or family
has from the study and range from better health outcomes to access to therapy options (Peay et
al., 2016). Harms include any psychological or physiological harm caused, including worsened
health condition or disappointment from the trial (Peay et al., 2016). The patient and their
family’s understanding of the history of disease, research, and participation in prior studies may
affect their willingness to participate in DMD trials (Naarding et al., 2020).
Parents of children with life-limiting disorders mostly decide to participate in clinical
trials with the hope for the individual benefit of the child and the perceived benefit of treatment
at no extra costs (Peay et al., 2016). The possibility of health improvement from participating in
clinical trials is a motivating factor for most parents. Parents with children with life-limiting
disorders such as DMD find it challenging to decline participation compared to those of children
in stable health (Naarding et al., 2020). Most parents also expect that if the trial is a success, their
children are receiving treatment at no extra costs. This presents a challenge as Crossnohere et al.
(2020) noted that many parents do not differentiate treatment options and clinical trials
participation options. While clinical trials may offer free drugs or treatment under certain
conditions, this is not always the promise in all trials.
Additionally, hopefulness and access to the latest treatment options is another motivator
established by Crossnohere et al. (2020). The psychology of parents and others participating in
clinical trials is that they maintain hope in obtaining a helpful treatment in various ways. Hope,
therefore, is a concept central to the recruitment process. It is closely linked to access to the latest
treatment options by assuming that newer treatments will be more effective or safer than older
treatments (Crossnohere et al., 2020). One downside of hopefulness is that patients and their
families may experience negative psychological outcomes in cases when trials are not successful
(Peay et al., 2016). As a result, failed clinical trials may increase desperation, anxiety, and
depressive symptoms for patients and their parents.
Some of the harms considered include randomization, time, and inconveniences caused
by the studies (Crossnohere et al., 2020). Randomization is the process of allocating patients
randomly to the intervention and placebo arms of a clinical trials. In blinded studies, the patients
and their parents are not aware which group they belong to (Crossnohere et al., 2020). Time
taken in clinical trials that may span years may also come with inconveniences such as the need
to travel and collect data (Crossnohere et al., 2020). A sense of helplessness and hopelessness
may also be present in some patients who have participated in previous studies to no success
(Crossnohere et al., 2020). These perceived harms may discourage patients and their parents
from participating. Generally, current literature shows that most parents will consider the costbenefit analysis as an effective means of determining whether to participate.
DMD clinical trials are currently challenged in recruiting pediatric research subjects for
many reasons. The condition is progressive, leading to loss of functional independence and
ultimate death. Current treatments using corticosteroids aim to reduce the progression while
genetic therapy is still ongoing to target the gene mutations for the disease. Current literature
shows that researchers face significant challenges when recruiting participants, and participants
and their parents conduct a risk-benefit analysis of the clinical trials to determine whether to
participate. Further research on means of motivating participants while minimizing potential
harms is needed to support DMD research.
Crossnohere, N. L., Fischer, R., Crossley, E., Vroom, E., & Bridges, J. F. (2020). The evolution
of patient-focused drug development and Duchenne muscular dystrophy. Expert Review
of Pharmacoeconomics & Outcomes Research, 20(1), 57-68.
Duan, D., Goemans, N., Takeda, S. I., Mercuri, E., & Aartsma-Rus, A. (2021). Duchenne
muscular dystrophy. Nature Reviews Disease Primers, 7(1), 1-19.
Goemans, N., Signorovitch, J., Sajeev, G., Yao, Z., Gordish-Dressman, H., McDonald, C. M., &
Mercuri, E. (2020). Suitability of external controls for drug evaluation in Duchenne
muscular dystrophy. Neurology, 95(10), e1381e1391.
Guiraud, S., & Davies, K. E. (2017). Pharmacological advances for treatment in Duchenne
muscular dystrophy. Current Opinion in Pharmacology, 34, 3648.
Naarding, K. J., Doorenweerd, N., Koeks, Z., Hendriksen, R. G., Chotkan, K. A., Krom, Y. D.,
& Kan, H. E. (2020). Decision-making and selection bias in four observational studies on
Duchenne and Becker muscular dystrophy. Journal of Neuromuscular Diseases, 7(4),
Paquin, R. S., Fischer, R., Mansfield, C., Mange, B., Beaverson, K., Ganot, A., & Peay, H. L.
(2019). Priorities when deciding on participation in early-phase gene therapy trials for
Duchenne muscular dystrophy: A best–worst scaling experiment in caregivers and adult
patients. Orphanet Journal of Rare Diseases, 14(1), 1-9.
Peay, H. L., Scharff, H., Tibben, A., Wilfond, B., Bowie, J., Johnson, J., & Biesecker, B. B.
(2016). “Watching time tick by…”: Decision making for Duchenne muscular dystrophy
trials. Contemporary Clinical Trials, 46, 1-6.
Ryder, S., Leadley, R. M., Armstrong, N., Westwood, M., De Kock, S., Butt, T., & Kleijnen, J.
(2017). The burden, epidemiology, costs and treatment for Duchenne muscular
dystrophy: An evidence review. Orphanet Journal of Rare Diseases, 12(1), 1-21.

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