Blinded clinical trials only became standardized into their current form in the 1980s. The history before this very recent phenomenon has become almost invisible, even though it was challenged right from the start by the AIDS crisis. The challenge to the RCT as the principle image of clinical research continues with the Ebola crisis and Right to Try legislation.
Some types of studies require unblinded personnel at the site and a matching unblinded monitoring and study management team. This presentation provides a little background on blinding and then reviews best practices for unblinding.
Jonas Ranstam MedicReS World Congress 2013MedicReS
Practical and statistical aspects of randomization
and blinding in clinical trials
Jonas Ranstam PhD
Department of Clinical Sciences
Lund University
Sweden
Some types of studies require unblinded personnel at the site and a matching unblinded monitoring and study management team. This presentation provides a little background on blinding and then reviews best practices for unblinding.
Jonas Ranstam MedicReS World Congress 2013MedicReS
Practical and statistical aspects of randomization
and blinding in clinical trials
Jonas Ranstam PhD
Department of Clinical Sciences
Lund University
Sweden
An epidemiological experiment in which subjects in a population are randomly allocated into groups, usually called study and control groups to receive and not receive an experimental preventive or therapetuic procedure, maneuver, or intervention .
Choice of control group in clinical trialsNagendra SR
To describe the general principles involved in choosing a control group for clinical trials intended to demonstrate the efficacy of a treatment and to discuss related trial design and conduct issues.
Clinical study types and designs are terms which represent the way in which clinical trials are structured and formulated.
Since we all know that clinical research is an extremely complex topic and not everything can be explained in a simple way, here we’ll focus only on some of the most basic types of clinical study types and designs which involve human subjects or participants.
First of all, you should know that the most basic grouping of study designs is experimental (treatment) studies and observational studies.
As we can suppose from the names, in an observational study, researchers have less control over subjects and they’re just observing what happens to subjects, while in experimental studies, researchers are using different methods (such as randomization) to place subjects in separate groups. This gives experimental studies much more validity than observational studies.
In this guide, we’ll talk about the 2 possible types of studies, as well as different study designs within.
In 1982, a blind signature, as introduced by David Chaum. Blind Signature Scheme is a form of digital signature scheme which allows a person to get a message signed by another party without revealing any information about the message to the other party.
This presentation introduces the theory behind the blind signature scheme, how to implement it with RSA public cryptographic scheme and various practical aspects of the scheme.
An epidemiological experiment in which subjects in a population are randomly allocated into groups, usually called study and control groups to receive and not receive an experimental preventive or therapetuic procedure, maneuver, or intervention .
Choice of control group in clinical trialsNagendra SR
To describe the general principles involved in choosing a control group for clinical trials intended to demonstrate the efficacy of a treatment and to discuss related trial design and conduct issues.
Clinical study types and designs are terms which represent the way in which clinical trials are structured and formulated.
Since we all know that clinical research is an extremely complex topic and not everything can be explained in a simple way, here we’ll focus only on some of the most basic types of clinical study types and designs which involve human subjects or participants.
First of all, you should know that the most basic grouping of study designs is experimental (treatment) studies and observational studies.
As we can suppose from the names, in an observational study, researchers have less control over subjects and they’re just observing what happens to subjects, while in experimental studies, researchers are using different methods (such as randomization) to place subjects in separate groups. This gives experimental studies much more validity than observational studies.
In this guide, we’ll talk about the 2 possible types of studies, as well as different study designs within.
In 1982, a blind signature, as introduced by David Chaum. Blind Signature Scheme is a form of digital signature scheme which allows a person to get a message signed by another party without revealing any information about the message to the other party.
This presentation introduces the theory behind the blind signature scheme, how to implement it with RSA public cryptographic scheme and various practical aspects of the scheme.
changes and yet everything is completely different."
Clinical Research has evolved a long way since its inception, whether documented or not. It traverses a long and amazing journey. The recorded history of clinical trials dates back to the biblical descriptions of 500 BC. The journey encompasses from dietary therapy - legumes and lemons - to drugs.
EBM is the practice of integrating individual clinical expertise with the best available clinical evidence from systematic research to maximize the quality and quantity of life for individual patients.
Modeling Alzheimer’s Disease research claims, evidence, and arguments from a ...jodischneider
Presentation: Jodi Schneider and Novejot Sandhu, “Modeling Alzheimer’s Disease Reseach Claims, Evidence, and Arguments from a Biology Research Paper.” 9th International Conference on Argumentation, International Society for the Society of Argumentation, Amsterdam, Netherlands, July 5, 2018
Abstract: Argument visualization may help make research papers easier to understand, which could both speed quality assessment within a discipline and help build interdisciplinary knowledge networks. This paper presents a case study of the arguments in a single high-profile paper on Alzheimer's disease research. Within this one paper, we analyze and hand-annotate the main claim, which is supported by 4 subclaims, in turn supported by data, methods, and materials. We also investigate how the paper imports and uses knowledge claims from other research papers. We create a specialized argument-based knowledge representation called a micropublication. In future work, we will investigate automatic argumentation mining for experimental biology research papers. Our long-term vision is to create literature-scale claim-argument networks that help more quickly use new knowledge about human health.
Information explaining what is a clinical trial and what it means to participate in one. Ins and outs, pros and cons of taking part in a clinical trial.
Case reports and studies may be defined as the non-experimental description of an individual or a few of cases in terms of new or unusual presentation of the diseases, an unexpected disease course or pathophysiology, and new effects (either beneficial or detrimental) of existing medications or procedures. Although they suffer from the non-experimental nature and other potential bias and errors, case reports and studies have played and will continue to play an important part in the advancement of medicine. They often serve as "primers" leading to discoveries of new diseases/disease pathophysiology as well as development of new preventive and therapeutic measures. Case reports and case studies are also employed as a platform for the training of medical students and/or resident doctors in scientific writing and critical thinking. Although the significance of case reports and studies in medicine has being recognized since the early stage of development of clinical medicine, their value needs to be appreciated in the context of modern clinical research design and the hierarchy of strength of evidence for guiding patient care. This paper discusses case reports and studies within the big picture of clinical research, research design, and evidence-based practice.
Published Research, Flawed, Misleading, Nefarious - Use of Reporting Guidelin...John Hoey
Much published health sciences literature is misleading and biased
Efforts to correct this include use of reporting guidelines- criteria for doing science and reporting the results properly
Also discussion of conflicts of interest - how to report them.
"Black Swans" are unknown unknowns- but the majority of things that affect your study are known risks. Let's look at some cases over my career and some current situations around the world to see what was predictable and what you can predict could affect your study and your career in the future.
How to solve problems in Microsoft Excel. Discussion of nested IF statements, VLOOKUP and HLOOKUP. Discussion of text analytics using Miscrosoft Excel.
How to follow Twitter even without an account. How to create an account. How to tweet and participate in a conversation. How to use Storify or Evernote to save tweets. How to use Hootsuite to set up a conference dashboard.
Leading the Way in Nephrology: Dr. David Greene's Work with Stem Cells for Ki...Dr. David Greene Arizona
As we watch Dr. Greene's continued efforts and research in Arizona, it's clear that stem cell therapy holds a promising key to unlocking new doors in the treatment of kidney disease. With each study and trial, we step closer to a world where kidney disease is no longer a life sentence but a treatable condition, thanks to pioneers like Dr. David Greene.
CRISPR-Cas9, a revolutionary gene-editing tool, holds immense potential to reshape medicine, agriculture, and our understanding of life. But like any powerful tool, it comes with ethical considerations.
Unveiling CRISPR: This naturally occurring bacterial defense system (crRNA & Cas9 protein) fights viruses. Scientists repurposed it for precise gene editing (correction, deletion, insertion) by targeting specific DNA sequences.
The Promise: CRISPR offers exciting possibilities:
Gene Therapy: Correcting genetic diseases like cystic fibrosis.
Agriculture: Engineering crops resistant to pests and harsh environments.
Research: Studying gene function to unlock new knowledge.
The Peril: Ethical concerns demand attention:
Off-target Effects: Unintended DNA edits can have unforeseen consequences.
Eugenics: Misusing CRISPR for designer babies raises social and ethical questions.
Equity: High costs could limit access to this potentially life-saving technology.
The Path Forward: Responsible development is crucial:
International Collaboration: Clear guidelines are needed for research and human trials.
Public Education: Open discussions ensure informed decisions about CRISPR.
Prioritize Safety and Ethics: Safety and ethical principles must be paramount.
CRISPR offers a powerful tool for a better future, but responsible development and addressing ethical concerns are essential. By prioritizing safety, fostering open dialogue, and ensuring equitable access, we can harness CRISPR's power for the benefit of all. (2998 characters)
The Importance of Community Nursing Care.pdfAD Healthcare
NDIS and Community 24/7 Nursing Care is a specific type of support that may be provided under the NDIS for individuals with complex medical needs who require ongoing nursing care in a community setting, such as their home or a supported accommodation facility.
Deep Leg Vein Thrombosis (DVT): Meaning, Causes, Symptoms, Treatment, and Mor...The Lifesciences Magazine
Deep Leg Vein Thrombosis occurs when a blood clot forms in one or more of the deep veins in the legs. These clots can impede blood flow, leading to severe complications.
Antibiotic Stewardship by Anushri Srivastava.pptxAnushriSrivastav
Stewardship is the act of taking good care of something.
Antimicrobial stewardship is a coordinated program that promotes the appropriate use of antimicrobials (including antibiotics), improves patient outcomes, reduces microbial resistance, and decreases the spread of infections caused by multidrug-resistant organisms.
WHO launched the Global Antimicrobial Resistance and Use Surveillance System (GLASS) in 2015 to fill knowledge gaps and inform strategies at all levels.
ACCORDING TO apic.org,
Antimicrobial stewardship is a coordinated program that promotes the appropriate use of antimicrobials (including antibiotics), improves patient outcomes, reduces microbial resistance, and decreases the spread of infections caused by multidrug-resistant organisms.
ACCORDING TO pewtrusts.org,
Antibiotic stewardship refers to efforts in doctors’ offices, hospitals, long term care facilities, and other health care settings to ensure that antibiotics are used only when necessary and appropriate
According to WHO,
Antimicrobial stewardship is a systematic approach to educate and support health care professionals to follow evidence-based guidelines for prescribing and administering antimicrobials
In 1996, John McGowan and Dale Gerding first applied the term antimicrobial stewardship, where they suggested a causal association between antimicrobial agent use and resistance. They also focused on the urgency of large-scale controlled trials of antimicrobial-use regulation employing sophisticated epidemiologic methods, molecular typing, and precise resistance mechanism analysis.
Antimicrobial Stewardship(AMS) refers to the optimal selection, dosing, and duration of antimicrobial treatment resulting in the best clinical outcome with minimal side effects to the patients and minimal impact on subsequent resistance.
According to the 2019 report, in the US, more than 2.8 million antibiotic-resistant infections occur each year, and more than 35000 people die. In addition to this, it also mentioned that 223,900 cases of Clostridoides difficile occurred in 2017, of which 12800 people died. The report did not include viruses or parasites
VISION
Being proactive
Supporting optimal animal and human health
Exploring ways to reduce overall use of antimicrobials
Using the drugs that prevent and treat disease by killing microscopic organisms in a responsible way
GOAL
to prevent the generation and spread of antimicrobial resistance (AMR). Doing so will preserve the effectiveness of these drugs in animals and humans for years to come.
being to preserve human and animal health and the effectiveness of antimicrobial medications.
to implement a multidisciplinary approach in assembling a stewardship team to include an infectious disease physician, a clinical pharmacist with infectious diseases training, infection preventionist, and a close collaboration with the staff in the clinical microbiology laboratory
to prevent antimicrobial overuse, misuse and abuse.
to minimize the developme
The dimensions of healthcare quality refer to various attributes or aspects that define the standard of healthcare services. These dimensions are used to evaluate, measure, and improve the quality of care provided to patients. A comprehensive understanding of these dimensions ensures that healthcare systems can address various aspects of patient care effectively and holistically. Dimensions of Healthcare Quality and Performance of care include the following; Appropriateness, Availability, Competence, Continuity, Effectiveness, Efficiency, Efficacy, Prevention, Respect and Care, Safety as well as Timeliness.
ICH Guidelines for Pharmacovigilance.pdfNEHA GUPTA
The "ICH Guidelines for Pharmacovigilance" PDF provides a comprehensive overview of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guidelines related to pharmacovigilance. These guidelines aim to ensure that drugs are safe and effective for patients by monitoring and assessing adverse effects, ensuring proper reporting systems, and improving risk management practices. The document is essential for professionals in the pharmaceutical industry, regulatory authorities, and healthcare providers, offering detailed procedures and standards for pharmacovigilance activities to enhance drug safety and protect public health.
1. Clinical Trial Blinding:
History and Analysis
of an
Imperfect Research Method,
with new
21st Century Challenges
SoCRA RTP, 01 June 2015
Mary K.D. D’Rozario
MSCR, MBA, CCRP, RAC, CCRA
President, CRP Social Media by Clinical Research Performance, Inc.
mary.drozario@crplink.com
www.crplink.com
@marydrozario
marydrozario
marykddrozario
2. Blinding: Do we know it when we see it?
Results:
“Physician respondents identified 10, 17 and 15
unique interpretations of single, double, and triple
blinding, respectively, and textbooks provided 5, 9
and 7 different definitions of each.”
PJ Devereaux, BJ Manns, WA Ghali et al. Physician interpretations and textbook definitions of blinding terminology in randomized
controlled trials. Journal of the American Medical Association. 2001;285(15):2000-2003
3. Blinding: Do we know it when we see it?
• Some suggest “masking” rather than blinding. [1]
• Some suggest blinding be specifically explained
in the study report. [1]
KF Schulz, I Chalmers, DG Altman. The landscape and lexicon of blinding in randomized trials. Annals of Internal Medicine.
2002;136:254-9
4. Blinding: Why do we do it?
• scientific legitimacy
• persuasion
• competition for resources
• flight to quality
• subject retention
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
5. Blinding: How well are we doing?
• Of 200 randomly chosen studies from 2001, 19%
stated they were “double blind” but had not
blinded either patients, health care providers, or
data collectors. [1]
M Haahr, A Hróbjartsson. Who is blinded in randomized clinical trials? A study of 200 trials and a survey of authors. Clin Trials.
2006;3:360-5
6. Blinding: How well are we doing?
Literature Review 1 (2004) 2 (2000) 3 (2004)
Type of study
evaluated:
Placebo-controlled Antidepressant Nicotine Replacement
Therapy
Number of studies: 97 91 73
Number of studies
that evaluated the
blind:
7 8 17
Number which noted
the blind
compromised:
5 4 12
1. “Turning a blind eye: the success of blinding reported in a random sample of randomized, placebo controlled
trials,” British Journal of Medicine, doi:10.1135/bmj.37952.631667.EE published 2004
2. “Critical approach to antidepressant trials: Blindness protection is necessary, feasible and measurable,” The
British Journal of Psychiatry, 2000:177:47:51
3. “The blind spot in the nicotine replacement therapy literature: Assessment of the double-blind in clinical trials,”
Addictive Behavior, 2004:29:673-684
7. History of Blinding: How we got here.
• Blinding Discovered and Re-discovered at several
times and places.
• Contributing issues include:
competing concepts of scientific truth and medical
treatment
social barriers between different groups of
scientists and physicians
language barriers
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
Shapero AK, Shapero E. The powerful placebo: From Ancient Priest to Modern Physician. Baltimore: The Johns Hopkins
University Press. 1997
Hacking I. Telepathy: Originals of Randomization in Experimental Design. Isis. Sept 1988, A Special Issue on Artifact and
Experiment (79);3:427-51
8. Galen: Canon of Medicine
–Galen (129 – c. 200)
–Canon compiled by Ibn
Sīnā (Avicenna) in Arabic,
completed 1025
–Considered authoritative
on some subjects into the
early 20th century
9. James Lind’s Treatise of the Scurvy, 1753
• Detailed study of the
available literature.
• Detailed study of actual
patients.
• Empirical study of
treatments.
10. Mesmerism
• 1784
• Commission appointed by
King Louis XVI
• Headed by Benjamin Franklin
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
11. Nuremberg Salt Test
• 1835
• Test of
homeopathy
Stolberg M. Inventing the randomized double-blind trial: the Nuremberg salt test of 1835. J R Soc Med. 2006;99:642-643
12. Test of “Smallest Discernible Difference in
Sensation”
• 1884
• Charles Sanders Peirce
(founder of the philosophy
known as American
pragmatism)
• student Joseph Jastrow
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
13. Subcutaneous Injection of Animal Testicles
• 1889, announced to
the Société de
Biologie, Paris, by
Charles E. Brown-
Séquard
• Tested by M.G. Variot
at the Hôtel Dieu
• Controlled for
“suggestion”
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
14. Diphtheria Trial (randomized, not blinded)
• 1897
• Designed by Johannes
Fibiger, age 28 (Dutch)
• Systematic randomization
to either standard
treatment or standard
treatment plus serum
16. Blinded Test of Diphtheria Serum
• 1914
• Adolf Bingel
• First major blinded trial of
a pharmaceutical product
• 937 randomized subjects
• Double-blind
• Control for “suggestion”
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
Braunschweig, Germany, circa 1900. Library of Congress.
17. 1920’s English Speaking
• Michigan Tuberculosis Trial (1926-1931) [1]
Single blind
Control for instability of disease state
• Cardiac Department of London Hospital Angina Study,
1930 [1]
Control for variations of symptoms
• Claude Bernard’s paper “An Introduction to the Study of
Experimental Medicine” translated and published in New
York, 1927 [2]
Used the term “comparative method” and described blinding
1. TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the
History of Medicine. 1998;72.3:389-433
2. Shapero AK, Shapero E. The powerful placebo: From Ancient Priest to Modern Physician. Baltimore: The Johns Hopkins
University Press. 1997
18. Paul Martini, Methodenlehre der
therapeutischen Untersuchung
• 1932
• “exclude suggestive or
other irrelevant factors in
the unwissentliche
Versuchsanordnung”
• Literally, “unwitting test” or
“test without knowing”
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
Stoll S, Raspe H. Paul Martini and the development of clinical epidemiology in Germany. The Paul Martini Foundation. [poster]
19. R.A. Fischer, The Design of Experiments
• 1935
• Botanist
• Inventor of the field of
experimental design
Made experiment “scientific”
• Considered randomization
important
• Blinding less important and
easy to do
20. Harry Gold: The Invention of “Pharmacology” and
the Gold Standard of Blinding for Research
• 1937, conducted first blinded study
• No previous exposure to blinding
methods
• Got the word “blinding” from a cigarette
advertisement
• First person assigned a professorial
position in “pharmacology,” at Cornell
University
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
Shapero AK, Shapero E. The powerful placebo: From Ancient Priest to Modern Physician. Baltimore: The Johns Hopkins
University Press. 1997
Gold H. Clinical pharmacology: historical note. Journal of Clinical Pharmacology and Journal of New Drugs. 1967(7):309-11
21. Streptomycin Clinical Trial
• 1948
• Austin Bradford Hill
• “deliberately left out the words
‘randomization’ and ‘random
sampling numbers’ at the time
because… I might have scared
them [collaborating physicians]
off.”
TJ Kaptchuk, "Intentional Ignorance: A History of Blind Assessment and Placebo Controls in Medicine", Bulletin of the History
of Medicine. 1998;72.3:389-433
22. Acceptance of Randomized Controlled Clinical
Trials Grows
• 1976, JAMA Special Article: Randomized Clinical
Trials: Perspectives on Some Recent Issues
“Ethical considerations suggest that randomized
trials are more suitable than uncontrolled
experimentation in protecting the interests of
patients. Randomized clinical trials remain the
most reliable method for evaluating the efficacy of
therapies.”
23. ICH Definition (ICH launched 1990)
“Blinding is an important means of reducing or minimizing the risk of
biased study outcomes. A trial where the treatment assignment is not
known by the study participant because of the use of placebo or other
methods of masking the intervention, is referred to as a single blind
study. When the investigator and sponsor staff who are involved in the
treatment or clinical evaluation of the subjects and analysis of data are
also unaware of the treatment assignments, the study is double blind.”
24. Declaration of Helsinki, October 2013
Amendment, Section 33: Use of Placebo
The benefits, risks, burdens and effectiveness of a new intervention must
be tested against those of the best proven intervention(s), except in the
following circumstances:
Where no proven intervention exists, the use of placebo, or no
intervention, is acceptable; or
Where for compelling and scientifically sound methodological
reasons the use of any intervention less effective than the best proven
one, the use of placebo, or no intervention is necessary to determine the
efficacy or safety of an intervention
and the patients who receive any intervention less effective than
the best proven one, placebo, or no intervention will not be subject to
additional risks of serious or irreversible harm as a result of not receiving
the best proven intervention.
Extreme care must be taken to avoid abuse of this option.
25. Some other thinking on blinding:
• Kolahi J. Abrishami M. Multiple-blind: Towards a new blinding protocol for
future generations of clinical trials. Med Hypotheses. 73(2009):843-5
Suggest expanding blinding, including requiring blinded creation of dual study reports and
blinding evaluation of study reports. (Some of these techniques were implemented by
Gold, but did not catch on for general use. [1])
• Bang H et al. Assessment of blinding in clinical trials. Controlled Clinical
Trials 2004;(25):143-56
Provides a method for arriving at a blinding index.
• JR Rees, TJ Wade, DA Levy, JM Colford Jr., JF Hilton. Changes in beliefs
identify unblinding in randomized controlled trials: a method to meet
CONSORT guidelines. Contemporary Clinical Trials. 2005;26:25-37
Suggest adding patient preference arms to studies.
• -D’Rozario MKD, Unblinded monitoring programs: design and education.
SoCRA Source. February 2011: 70-75
Discusses the need for unblinded monitoring and how to implement an unblinded
monitoring program.
1. Shapero AK, Shapero E. The powerful placebo: From Ancient Priest to Modern Physician.
Baltimore: The Johns Hopkins University Press. 1997
26. HIV Activism • 1984
FDA credited (some were unofficial “look
the other way”) with first allowing
compassionate use.
• 1987
AIDS Quilt first displayed on the National
Mall
Drugs typically took 10 years to be
approved.
AZT was the first fast-track (law itself not
established until 1997) drug, approved in
25 months.
Lengthened life by about a year.
First Study:
• 12 centers. All with different standard-of-
care protocols.
• Had been designed as a 6 month blinded
study.
• Mean participation of 120 days when study
was unblinded.
• Claims that they study was never blinded
because of obvious adverse events, product
testing by subjects, product pooling, etc.
*Highlighted information from a variety of
sources, not necessarily covered in this
book.
27. HIV Activism
• 1989: Compound Q
Open-label patient-led study of an
herbal product.
Closed after patient deaths.
Made famous in the movie Dallas
Buyers Club.
*Highlighted information from a variety of
sources, not necessarily covered in this
book.
28. Ebola
• 11 AUG 2014: Advisory Panel meets, creates
publication “Ethical considerations for use of
unregistered interventions for Ebola virus
disease”
Determine that it is ethical to offer unproven therapies
• Consent required
Therapies should be distributed fairly
Communities should be involved in distribution
decisions
Moral requirement to collect data from such
interventions
29. Ebola
• 20-21 OCT 2014: WHO Ethics Working Group
meets, creates publication “Ethical issues related
to study design for trials on therapeutics for Ebola
virus disease
“monitored emergency use of unregistered and
experimental interventions (MEURI)” instead of
“compassionate use”
Trials should not take away from the care received by
other patients
Real time data collection and sharing
Provides a worksheet to compare a randomized
controlled trial against other options.
30. Right to Try
• Current Law in 19 states
Arizona Arkansas Colorado Indiana Louisiana
Maine Michigan Minnessota Mississipi Missouri
Montana Nevada North Dakota Oklahoma
South Dakota Tennessee Utah Virginia
Wyoming
Link to all current and draft legislation:
http://www.raps.org/regulatory-
focus/news/right-to-try
31. Right to Try Example: Arkansas
Terminally ill
No current treatment
No clinical trial within 100 miles of home
Manufacturer can charge for the product
• Insurance may, but not required, to pay
for product
• Insurance must pay for all other
treatment
• Debt related to product cannot be
collected from family
32. Right to Try Example: DRAFT North Carolina
Terminally ill
No current treatment
Detailed informed consent requirements
Manufacturer protected from liability
Prescribing doctor protected from sanctions
Manufacturer can charge for the product
• Insurance may, but not required, to pay
for product
• Insurance must pay for all other treatment
• Debt related to product cannot be
collected from family