Questions about
clinical trials
Does clinical research sound like a foreign language to you? Check out this clinical research glossary for simple definitions of common clinical research words.
Why do we need clinical trials?
Clinical trials help us learn new ways to treat and prevent diseases. Researchers test:
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New medicines or medical devices to treat a disease
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Different ways to use current medicines to treat a disease
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Ways to find out if someone has a disease, or has a chance of getting a disease
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Ways to prevent disease
When researchers find a promising treatment, they need people to test it to make sure it works and is safe to use in a larger number of people.
What safeguards are in place for clinical trials?
In the past, clinical trials did not have all the participant safeguards that are now in place. This is especially true for trials that included people of color. For a long time, these participants were lied to, treated unfairly, and even harmed during trials.
These injustices have been exposed and have led to really big changes in how researchers conduct clinical trials today.
Today, clinical trials follow strict guidelines and laws to ensure greater protections for trial participants.
Ethical guidelines for all trials
There are 2 key documents that set ethical guidelines for clinical trials worldwide:
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The Declaration of Helsinki, which was first created in 1964
and is actively updated -
The Belmont Report, which was made in 1978
Researchers and scientists commit to following these documents, which have:
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Changed how doctors and researchers around the world are trained
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Led to laws countries have passed to help make trials safer
Safety before a trial can even start
Researchers must first create a clinical research protocol. This is a detailed plan for how researchers will do the trial to make sure everyone in the trial follows the same steps. A protocol includes:
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Why the research is important
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Who can join – such as medical conditions people can or can’t have
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How the trial will happen – such as where, when, and how often participants will have visits or tests
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What the researchers will do – such as the specific tests, treatments, or surveys researchers will give participants
Next, the protocol must be reviewed and approved by both:
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Health authorities, such as the FDA
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An Institutional Review Board (IRB), also called an Ethics Committee, which is a team of independent experts and members of the community that make sure the rights and welfare of the trial participants are protected
Safety while a trial is ongoing
Many groups continue to check the trial and its participants to help keep participants safe:
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The FDA and IRB, which continue to review how a trial is going
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The study team of doctors, nurses, and other experts who carefully check the participants’ health
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A group called the Data Monitoring Committee (DMC), or Data Safety Monitoring Board (DSMB), which reviews data to protect participants' safety
If the clinical trial team, IRB, or DMC think the risks of the trial are more than the benefits, they will stop the clinical trial.
Source: Clinical Research: Benefits, Risks, and Safety, NIH National Institute on Aging
¿Qué medidas de seguridad existen para los ensayos clínicos?
En el pasado, los ensayos clínicos no contaban con todas las medidas de seguridad para los participantes que existen ahora. Esto es especialmente cierto en el caso de los ensayos que incluían a personas de color. Durante mucho tiempo se les mintió a estos participantes, se les trató injustamente e incluso se les perjudicó durante los ensayos.
Estas injusticias han salido a la luz y han dado lugar a cambios realmente importantes en la forma en que los investigadores realizan los ensayos clínicos hoy en día.
Hoy en día, los ensayos clínicos siguen directrices y leyes estrictas para garantizar una mayor protección a los participantes de los ensayos.
Ethical guidelines for all trials
There are 2 key documents that set ethical guidelines for clinical trials worldwide:
-
The Declaration of Helsinki, which was first created in 1964
and is actively updated -
The Belmont Report, which was made in 1978
Researchers and scientists commit to following these documents, which have:
-
Changed how doctors and researchers around the world are trained
-
Led to laws countries have passed to help make trials safer
Safety before a trial can even start
Researchers must first create a clinical research protocol. This is a detailed plan for how researchers will do the trial to make sure everyone in the trial follows the same steps. A protocol includes:
-
Why the research is important
-
Who can join – such as medical conditions people can or can’t have
-
How the trial will happen – such as where, when, and how often participants will have visits or tests
-
What the researchers will do – such as the specific tests, treatments, or surveys researchers will give participants
Next, the protocol must be reviewed and approved by both:
-
Health authorities, such as the FDA
-
An Institutional Review Board (IRB), also called an Ethics Committee, which is a team of independent experts and members of the community that make sure the rights and welfare of the trial participants are protected
Safety while a trial is ongoing
Many groups continue to check the trial and its participants to help keep participants safe:
-
The FDA and IRB, which continue to review how a trial is going
-
The study team of doctors, nurses, and other experts who carefully check the participants’ health
-
A group called the Data Monitoring Committee (DMC), or Data Safety Monitoring Board (DSMB), which reviews data to protect participants' safety
If the clinical trial team, IRB, or DMC think the risks of the trial are more than the benefits, they will stop the clinical trial.
Source: Clinical Research: Benefits, Risks, and Safety, NIH National Institute on Aging
Quelles mesures de protection sont en place pour les essais cliniques ?
Par le passé, les essais cliniques ne comportaient pas toutes les mesures de protection des participants qui sont maintenant en place. C’est particulièrement vrai pour les essais impliquant des personnes de couleur. Pendant longtemps, on a menti à ces participants, on les a traités injustement et on leur a même fait du mal pendant les essais.
Ces injustices ont été mises en évidence et ont conduit à de très grands changements dans la façon dont les chercheurs mènent aujourd’hui des essais cliniques.
Aujourd’hui, les essais cliniques sont encadré par des directives et des lois strictes afin d’assurer une meilleure protection des participants aux essais.
Lignes directrices éthiques pour tous les essais
Deux documents clés établissent des lignes directrices éthiques pour les essais cliniques dans le monde entier :
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La Déclaration d’Helsinki, qui date de 1964 et qui est activement mise à jour
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Le Rapport Belmont, qui date de 1978
Les chercheurs et les scientifiques s’engagent à suivre ces documents, qui :
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Ont modifié la façon dont les médecins et les chercheurs du monde entier sont formés
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Sont à l’origine de lois que des pays ont adoptées pour aider à rendre les essais plus sûrs
La sécurité avant même le début d’un essai
Les chercheurs doivent d’abord créer un protocole de recherche clinique. Il s’agit d’un plan détaillé de la façon dont les chercheurs mèneront l’essai pour s’assurer que toutes les personnes impliquées dans l’essai suivent les mêmes étapes. Un protocole comprend :
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Pourquoi la recherche est importante
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Qui peut participer – comme les problèmes médicaux que les personnes peuvent ou ne peuvent pas avoir
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Comment l’essai se déroulera – comme où, quand et à quelle fréquence les participants auront des visites ou des tests
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Ce que les chercheurs feront – comme les tests, les traitements ou les enquêtes spécifiques auxquels les chercheurs soumettront les participants
Ensuite, le protocole doit être examiné et approuvé à la fois par :
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Les autorités de santé, telles que la FDA
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Un comité d’examen institutionnel (IRB), également appelé comité d’éthique et de recherche (CER), qui est une équipe d’experts indépendants et de membres de la communauté qui s’assurent que les droits et le bien-être des participants à l’essai sont protégés
Sécurité pendant un essai clinique
De nombreux groupes continuent de surveiller l’essai et ses participants pour aider à assurer la sécurité des participants :
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La FDA et l’IRB, qui continuent d’examiner le déroulement d’un essai
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L’équipe de l’étude, composée de médecins, d’infirmiers et d’autres experts qui vérifient soigneusement la santé des participants
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Un groupe appelé le Comité de surveillance des données (DMC, ou Conseil de surveillance de la sécurité des données (DSMB), qui examine les données pour protéger la sécurité des participants
Si l’équipe de l’essai clinique, l’IRB ou le DMC pense que les risques de l’essai sont plus importants que les avantages, ils mettront fin à l’essai clinique.
Source: Clinical Research: Benefits, Risks, and Safety, NIH National Institute on Aging
Participating in a clinical trial is always your choice. You can decide not to join. Or you can join and later choose to leave at any time. If you decide to leave, it’s helpful to share your reasons with the clinical trial team. This feedback can help make the trial process better for you and for others in the future.
Read about your rights as a research participant:
Research Participants’ Bill of Rights, The Multi-Regional Clinical Trials Center
What are the phases of clinical trials?
Each of the 4 phases add more participants to answer specific questions. A trial treatment can only move on to the next phase if the trial shows that it may work and that the benefits may outweigh the risks.
Before Phase 1
The trial treatment is tested in lab and animal studies (not on people).
Phase 1
Researchers give the trial treatment to 20-100 participants with or without the disease.
Researchers answer questions such as:
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Is it safe for people?
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Are there any side effects?
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How much of the trial treatment is needed?
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This can last for several months.
Chance of advancing to the next phase:
About 7 of 10 trials move to Phase 2.
Phase 2
Researchers give the trial treatment to several hundred participants with the condition.
Researchers answer questions such as:
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How well does it work?
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Are there any new side effects?
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Are there new safety concerns? (This can last for several months to 2 years.)
Chance of advancing to the next phase:
About 3 of 10 trials move to Phase 3.
Phase 3
Researchers give the trial treatment to 300-3,000 or more participants across multiple locations.
Researchers answer questions such as:
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Does it work better than current treatments?
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Does it work to prevent, diagnose, or treat the disease?
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What more is learned about known or new side effects?
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Are there new safety concerns? (This can last for 1-4 years.)
Chance of advancing to the next phase:
About 3 out of every 10 clinical trials move forward for approval decisions, such as by the FDA.
In other countries, government authorities review for approval to use by people in their countries.
Phase 4
If the health authority approves the treatment for public use, researchers will continue to do studies to learn more about the treatment. Thousands of patients will get the treatment.
Researchers answer questions such as:
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Does it work for all types of people?
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What is the best way to use it?
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Are there new safety concerns when taken over a longer period of time?
FDA Approval
At this point, a sponsor (who funds the research) may submit the results to a health authority (such as the FDA) to have the trial treatment approved for people to use. The authority may approve the trial treatment if the results show that the trial treatment is safe and works well.
Source : Step 3: Clinical Research, FDA.gov
¿Cuáles son las fases de los ensayos clínicos?
Cada una de las 4 fases añade más participantes para responder a preguntas específicas. Un tratamiento del ensayo solo puede pasar a la siguiente fase si el ensayo demuestra que puede funcionar y que los beneficios pueden superar los riesgos.
Antes de la Fase 1
El tratamiento del ensayo se prueba en estudios de laboratorio y con animales (no en personas).
Fase 1
Los investigadores administran el tratamiento del ensayo a unos 20 a 100 participantes con o sin la enfermedad.
Los investigadores responden a preguntas como las siguientes:
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¿Es seguro para las personas?
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¿Hay algún efecto secundario?
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¿Qué cantidad del tratamiento del ensayo se necesita?
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Esto puede durar varios meses.
Posibilidad de pasar a la siguiente fase:
Aproximadamente 7 de cada 10 ensayos pasan a la Fase 2
Fase 2
Los investigadores administran el tratamiento del ensayo a varios cientos de participantes con la enfermedad.
Los investigadores responden a preguntas como las siguientes:
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¿Qué tan bien funciona?
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¿Hay nuevos efectos secundarios?
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¿Existen nuevos problemas de seguridad? (Esto puede durar de varios meses a 2 años).
Posibilidad de pasar a la siguiente fase:
Aproximadamente 3 de cada 10 ensayos pasan a la Fase 3.
Fase 3
Los investigadores administran el tratamiento del ensayo a entre 300 y 3,000 participantes o más en varios lugares.
Los investigadores responden a preguntas como las siguientes:
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¿Funciona mejor que los tratamientos actuales?
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¿Funciona para prevenir, diagnosticar o tratar la enfermedad?
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¿Qué más se sabe sobre los efectos secundarios conocidos o nuevos?
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¿Existen nuevos problemas de seguridad? (Esto puede durar entre 1 y 4 años).
Posibilidad de pasar a la siguiente fase:
Aproximadamente 3 de cada 10 ensayos clínicos siguen adelante para las decisiones de aprobación, como por la FDA.
En otros países, las autoridades gubernamentales revisan la aprobación para el uso por parte de la población en sus países.
Fase 4
Si la autoridad de salud aprueba el tratamiento para uso público, los investigadores seguirán haciendo estudios para saber más sobre el mismo. Miles de pacientes recibirán el tratamiento.
Los investigadores responden a preguntas como las siguientes:
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¿Funciona para todo tipo de personas?
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¿Cuál es la mejor manera de utilizarlo?
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¿Existen nuevos problemas de seguridad cuando se administra durante un periodo de tiempo más largo?
Aprobación de la FDA
En este punto, un patrocinador (que financia la investigación) puede presentar los resultados a una autoridad de salud (como la FDA) para que el tratamiento del ensayo sea aprobado para su uso por las personas. La autoridad puede aprobar el tratamiento del ensayo si los resultados muestran que el mismo es seguro y funciona bien.
Fuente: Paso 3: Investigación clínica, FDA.gov
Quelles sont les phases des essais cliniques ?
Chacune des quatre phases ajoute plus de participants pour répondre à des questions spécifiques. Un traitement à l’essai ne peut passer à la phase suivante que si l’essai montre qu’il peut fonctionner et que les avantages peuvent l’emporter sur les risques.
Avant la phase 1
Le traitement à l’essai est testé en laboratoire et sur des animaux (et non sur des humains).
Phase 1
Les chercheurs administrent le traitement à l’essai à 20 à 100 participants atteints ou non de la maladie.
Les chercheurs répondent à des questions telles que :
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Est-ce sans danger pour les êtres humains ?
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Y a-t-il des effets secondaires ?
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Quelle quantité de traitement est nécessaire ?
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Cela peut durer plusieurs mois.
Probabilité de passer à la phase suivante :
Environ 7 essais sur 10 passent à la phase 2.
Phase 2
Les chercheurs administrent le traitement à l’essai à plusieurs centaines de participants atteints de la maladie.
Les chercheurs répondent à des questions telles que :
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Dans quelle mesure est-ce efficace ?
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Y a-t-il de nouveaux effets secondaires ?
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Y a-t-il de nouveaux problèmes de sécurité ? (Cela peut durer de plusieurs mois à deux ans.)
Probabilité de passer à la phase suivante :
Environ 3 essais sur 10 passent à la phase 3.
Phase 3
e 3 Les chercheurs administrent le traitement à l’essai à 300 à 3 000 participants de plus répartis dans plusieurs emplacements géographiques.
Les chercheurs répondent à des questions telles que :
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Est-ce que cela fonctionne mieux que les traitements actuels ?
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Est-ce que cela fonctionne pour prévenir, diagnostiquer ou traiter la maladie ?
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Qu’apprend-on de plus sur les effets secondaires connus ou nouveaux ?
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Y a-t-il de nouveaux problèmes de sécurité ? (Cela peut durer entre un et quatre ans.)
Probabilité de passer à la phase suivante :
Environ 3 essais cliniques sur 10 passent à la phase d’attente de décision d’approbation, par exemple par la FDA.
Dans d’autres pays, les autorités gouvernementales examinent l’approbation de l’utilisation par les personnes de leur pays.
Phase 4
Si l’autorité sanitaire approuve le traitement pour un usage public, les chercheurs continueront de faire des études pour en savoir plus sur le traitement. Des milliers de patients recevront le traitement.
Les chercheurs répondent à des questions telles que :
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Est-ce que cela fonctionne pour tous les types de personnes ?
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Quelle est la meilleure façon de l’utiliser ?
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Y a-t-il de nouveaux problèmes de sécurité lorsque le traitement est pris sur une longue durée ?
Approbation de la FDA
À ce stade, un promoteur (qui finance la recherche) peut soumettre les résultats à une autorité sanitaire (telle que la FDA) pour que le traitement à l’essai soit approuvé pour les êtres humains. L’autorité peut approuver le traitement à l’essai si les résultats montrent que le traitement à l’essai est sûr et fonctionne bien.
Source : Step 3: Clinical Research, FDA.gov
How do I know if joining a clinical trial is right for me?
Before joining a study, it is important that you understand:
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Why the study is being done
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The possible benefits and harms
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What you will have to do
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The rights you have
You may want to talk to the study doctor or staff, your own medical team, family or friends before deciding.
If you choose to join the clinical trial, the study team will help you through all the steps to get started.
How will I know if a clinical trial I join will be successful?
Comment saurai-je si un essai clinique auquel je participe sera couronné de succès ?
¿Cómo sabré si el ensayo clínico en el que participo tendrá éxito?
Not every clinical trial leads to a new medicine. In fact, most don’t. But every clinical trial adds to our understanding of diseases, such as SCD.
We can learn many things from a clinical trial – What works? What doesn’t work? What might work? What works better? And more.
The goal is to continue learning so we can help people living with SCD live longer, healthier lives.
Clinical trials can even lead to unexpected outcomes.
For example, in the 1980s, several studies showed that giving daily penicillin to children under age 5 with SCD greatly lowered their chance of pneumonia. This unexpected but positive finding showed the importance of knowing if a child has SCD early in life. This discovery led doctors to recommend SCD screening of babies right after birth. Today, every state requires newborns to be tested for SCD!
Clinical trials are like stepping stones on a path of discovery!
What kind of treatment will I receive?
During a clinical trial, you may be assigned to get the trial treatment, a placebo, or the standard of care for your condition. Sometimes you will get the trial treatment in combination with the standard of care. What you get depends on the design of the trial and the treatment being studied.
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Trial treatment: The drug or vaccine that the researchers are studying to see if it works.
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Placebo: Something that looks like the study treatment but has no active medicine in it. Researchers use placebos to help measure the real effects of the trial treatment.
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Standard of care: The same care or usual treatment doctors typically use to treat a condition.
What is the point of a placebo?
Researchers use placebos to help measure the real effects of the trial treatment.
When people take a new medicine, they may feel better just because they believe it will help them. This is called the placebo effect.
To make sure a trial treatment is working — and not that people are feeling better because of the placebo effect — researchers compare it to a placebo. If the trial treatment works better than the placebo, then researchers can be more confident that the improvement is because of the treatment.
How will they decide which treatment I get?
In most trials, study participants are placed into different study treatment groups by chance (like flipping a coin). This is called randomization.
Typically, you, your doctor, and the research team will not know if you are getting the trial treatment or the placebo. This is called blinding – and it helps make sure that knowing the treatment you are getting does not influence or change the results.
What if I get assigned a placebo?
If you get assigned to take a placebo, you will still get a high level of care and monitoring. You may also get the standard of care (usual treatment) for your condition. Many clinical trials offer the trial treatment later in the trial to participants who got the placebo.
If you are assigned to take a placebo, you are still helping researchers learn about the trial treatment.
What do I need to do before I join a clinical trial?
Before you join a clinical trial, you’ll go through a process called informed consent. The researchers will talk with you using a document called an informed consent form that explains details such as:
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Why researchers are doing the clinical trial
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What you will need to do during the clinical trial, such as:
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How long you will be in the trial
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How many trial visits you will have
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What tests and exams you may have during trial visits
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Risks and benefits of taking part in the trial
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Any payment you might get for taking part, such as for parking, travel, and meals
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How your personal information will be kept private
If new safety information comes up during the clinical trial, researchers will update the informed consent form. If that happens, you will be asked to review and sign it again to make sure you know about the changes.
Before you join a trial, carefully consider if it is right for you:
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Ask your questions. The researchers are there to help you understand what’s involved and why your participation is important. They can answer your questions both before you join and at any time during the trial.
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Talk about the clinical trial with your doctor, healthcare team, family, and friends.
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Make sure you have answers to all of your questions so you, your family, and your healthcare team can decide together.
What happens during a clinical trial visit?
The word "clinical" might sound impersonal - however, throughout the clinical trial, you’ll have regular visits with a team of doctors and nurses who you will get to know over time. This gives you a chance to get comfortable with the people supporting you.
Clinical trial visits may be in the clinic or at home. These may be similar to a visit to your doctor, where you go into an exam room and talk with a clinical trial nurse or doctor. You may be asked to do things like:
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Have a physical exam
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Answer questions about your health
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Have your blood drawn for testing
Remember: Clinical trials are completely voluntary. You can decide to leave the trial at any time. Before you do, please share any problems or worries with the trial team. There may be more support to help you.
How much time will a clinical trial take?
Most clinical trials last between 6 months - 1 year, though some may be shorter or longer. Your number of trial visits and how long they will take can vary.
Before you join, you can ask how much of a time commitment the trial will be. The trial staff will also give you a visit schedule so you can plan for them.
How can people change how researchers do clinical trials?
Today, more communities are sharing their priorities and concerns directly with researchers. And researchers are paying attention.
More than ever, researchers are embracing a "patients as partners" approach. They are working with communities to make research more inclusive and meaningful.
Here are some examples of how they are doing this:
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Community-based participatory research (CBPR): This is an approach that brings together researchers, community members, and local partners. Together, they design research studies that center around the community’s needs.
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Example: The Sickle Cell Disease Collaborative Conversations works with partners to help improve care for people with SCD. They work to build a collaborative healthcare provider and community network and find ways to help people get better access to health care.
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Building trust through community health workers: Researchers are working with community health workers — trusted members of the community who connect people to healthcare services and information. These workers help teach the community about clinical trials, answer questions, and help people make decisions about their health.
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Sharing clinical trial results with the community: When you join a clinical trial, you might wonder, “Will I ever find out the results?” More and more, the answer is “Yes!” More researchers are sharing the outcomes of trials with the community in a simple, easy to understand summary. In some countries, this is even a requirement!
To learn more about how everyday people are making a difference in how research is done, visit:
Where can I learn more about clinical trials?
You can learn more about clinical trials from these sites:
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Clinical Trials.gov, Learn About Studies
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CISCRP Clinical trial videos:
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Finding Treatments Together, A 3-part series on how clinical research is done
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Pediatric Education Series: Overview of Clinical Trials
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U.S. Department of Health and Human Services, NIH, Clinical research trials and you