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TEDMED 2012 Recap – Part 1

Like children who were suddenly forced to go home after spending three and a half days in Disney World, the delegates slowly made their ways towards the exit of the J.F. Kennedy Center of the Performing Arts as one of the greatest conferences for healthcare innovation concluded. Following the format of the original TED Talks, TEDMED featured “21 women, 45 men, 1 monster, 32 performers up on stage” – actual tweet from @TEDMED.Imagination, innovation and inspiration are the three words used to describe the mission of this community of passionate, leading-edge thinkers who come from every discipline within the fields of health and medicine, as well as from business, government, technology, academia, media and the arts. This mixture provides a very unique way of learning across disciplines towards a common goal.

For all of you who couldn’t come or who couldn’t catch any of the simulcast spots spread around the US, we provide you with a summarized recap of the most interesting presentations we saw. The TEDMED team will start to upload the official videos in 3-4 weeks, they’ll go up in batches of 5-6, once every week.

 

Session 1: “Embracing the Unconventional”

An astonishing act by the Traces acrobats set the pace for the first group of speakers, starting with Bryan Stevenson the executive director of Equal Justice Initiative, who explained the importance of creating an identity that resonates in an honest way, as a way of trying to prepare us for the upcoming talks by opening our eyes and minds in order to determine who are all these new technologies and advancements intended to benefit – those with wealth or those without. Later on, Rebecca Onie followed with an inspiring talk aboutHealth Leads an organization which provides a system where doctors are able to prescribe not drugs to manage disease, but solutions that improve health, helping patients to access basic resources needed for healthy lifestyles. As an example, she asked why prescribe antihistamines to an allergic kid who will return to a house full of allergens? in-stead, why not also prescribe removing the allergens from the house in the first place? So simple, yet no one ever thought about it, or did something.

 

Session 2: “Taking the Long View” Jill Sobule and Francis Collins 290x290 TEDMED 2012 Recap   Part 1

The next day kicked-off with a great surprise, the Director of the National Institutes of HealthFrancis Collins rocking his blues guitar (which had a cool design of the DNA double helix on its fretboard) along with Jill Sobule singing “Disease don’t care.” Collins then put down his guitar and switched to the “serious stuff” giving us one of the best talks of the conference. The NIH is focusing part of their efforts in making the new drug discovery process faster. He invited to stage an inspiring 15-year old boy who suffers from progeria and he shared his experience and feelings about participating in clinical trials and offered his opinion on the role he is playing to help find new treatments and a cure. Collins then continued with an idea about how some drugs that were originally discovered for one disease can be rescued and used for other diseases. At the end he called for more resources, new partnerships and the commitment of young professionals (count me in!).

Later on John Hoffman from HBO Documentaries and Judith Salerno of the Institute of Medicine tackled the obesity epidemic and showed a preview of a new documentary called “The Weight of the Nation.”

 

Session 3: “The Missing Ingredients”

The Director of the Centers for Disease Control & PreventionThomas Frieden spoke about the importance of prevention and the importance of focusing on what makes a difference locally, beyond statistics.

“You diagnose many patients… but how many do you actually cure?” – Thomas Frieden, MD. Director of CDC.

Then Dr. Ivan Oransky from Reuters Health stepped up to the stage holding a baseball and presented an interesting analogy using the recent Moneyball movie in how doctors trying to predict patients outcomes is similar to scouts predicting young baseball players’ performance on the big leagues. Next, Todd Park the US Chief Technology Officer, gave his first of two talks about how electronic medical records implementation have shown beneficial results. After him came one of our favorite talks of all, Dr. Jacob Scott was introduced with an impressive background of having been an astrophysicist, a Navy submarine nuclear reactor operator, earned his M.D., became a radiation oncologist and is now earning a PhD in math at Oxford. He explained how all this process from astrophysics through medicine and to math was driven by his creative desire. He criticized how med schools systematically discourage creativity among their candidates even before selecting them (MedCrunch published a post on Creativity). His talk really shook the ground and received a standing ovation an also some reaction from the AAMC. We interviewed him and got to know him throughout the conference sharing more insights about the importance of changing medical education for the physician that the future needs (we will publish the interview in the next couple of days). Jacob Scott 2 600x400 TEDMED 2012 Recap   Part 1

 

Session 4: “The Shape of Things”

As a way of showing us all how the shape and morphology of things (such as the human body) can generate the most important creations, the dancers Momix impressed everyone with a magnificent performance. Albert-Lázló Barabási from the Center for Complex Network Research of the Northeastern University used a map of Manhattan to illustrate the networks that make the biological processes of cells work. He told us to forget about the physical aspect of diseases and to start thinking about the connections that form a network, a system that manifests as a disease. Another MedCrunch favorite followed, Seth Cooperfrom the University of Washington and one of the creators of FoldIt spoke about how the power of play and gamification can solve real world problems, such as how players have helped scientists design new synthetic proteins that are more efficient than native ones (MedCrunch interviewed him, you can see it here).

JH21015 290x290 TEDMED 2012 Recap   Part 1

The NYU team stepped in and spoke about a whole new way of learning anatomy. Marc Triola, MD and John Qualter are the creators of Biodigital Human, a fascinating 3D model of the whole human body anatomy that is not only easy to use but also web based and free. We had the chance to interview them and demo the technology (they always looked cool with the 3D glasses on their booth). The program allows for recreation of different pathologies such as a heart attack and also medical procedures like an appendectomy.

David Icke amazed the crowd with his presentation about pliable electronics, wich could be worn on the skin and measure vitals or register many other kinds of biological data and deliver it wirelessly to your smartphone or directly to your physician on real time. Finally, and what a way to drastically shake everyone off their seats! Diane Kelly shared her results (and very explicit pictures) of her research about mammalian penises… yep we said penises.

 

Session 5: “Matters of the Mind”

Session 5 highlight came from Virginia Breen who is the mother of Elizabeth Bonker, a 14-year old autistic child who has struggled with the desire to comunicate and not being able to. Elizabeth story opened the minds and hearts of the audience and taught us that sometimes established and trusted systems (such as an IQ test) are not always a reflection of the truth. The day concluded with Miguel Nicolelis from the Duke Center of Neuroengineering, who showed everyone how monkeys played video games controlling cursors using brain waves (look mama-monkey no hands!). As if that wasn’t enough, they showed another monkey that was in North Carolina and who controlled a robot that was 6 times bigger and heavier that was all the way across the world in Japan.

 

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TEDxWarwick – Kevin Warwick – Implants & Technology — The Future of Healthcare?

httpvh://www.youtube.com/watch?v=Z8HeFNJjuj0

Kevin Warwick is Professor of Cybernetics at the University of Reading, where he carries out research in artificial intelligence, control, robotics and cyborgs. He is a Chartered Engineer and a Fellow of the IET.

Kevin was born in Coventry, UK and left school to join British Telecom, at the age of 16. He took his first degree at Aston University, followed by a PhD and research post at Imperial College, London. He subsequently held positions at Oxford, Newcastle and Warwick Universities before being offered the Chair at Reading.


As well as publishing over 500 research papers, Kevin’s experiments into implant technology led to him being featured as the cover story on the US magazine, Wired.
Kevin has been awarded higher doctorates (DSc) both by Imperial College and the Czech Academy of Sciences, Prague, and has received Honorary Doctorates from Aston University, Coventry University, Bradford University and Robert Gordon University. He was presented with The Future of Health Technology Award in MIT, was made an Honorary Member of the Academy of Sciences, St. Petersburg, and has received The IEE Senior Achievement Medal, the Mountbatten Medal and the Ellison-Cliffe Medal. In 2000 Kevin presented the Royal Institution Christmas Lectures, entitled “The Rise of the Robots”.

Kevin’s research involves the invention of an intelligent deep brain stimulator to counteract the effects of Parkinson Disease tremors. The tremors are predicted and a current signal is applied to stop the tremors before they start — this is to be trialled in human subjects. Another project involves the use of cultured/biological neural networks to drive robots around — the brain of each robot is made of neural tissue.

Kevin is perhaps best known for his pioneering experiments involving a neuro-surgical implantation into the median nerves of his left arm to link his nervous system directly to a computer to assess the latest technology for use with the disabled. He was successful with the first extra-sensory (ultrasonic) input for a human and with the first purely electronic telegraphic communication experiment between the nervous systems of two humans.

In the spirit of ideas worth spreading, TEDx is a program of local, self-organized events that bring people together to share a TED-like experience. At a TEDx event, TEDTalks video and live speakers combine to spark deep discussion and connection in a small group. These local, self-organized events are branded TEDx, where x = independently organized TED event. The TED Conference provides general guidance for the TEDx program, but individual TEDx events are self-organized.* (*Subject to certain rules and regulations)

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FDA Approved Drugs in 2012

FDA Approved Drugs in 2012

The following drugs were approved for sale in the United States.

Dermatology/Plastic Surgery
Erivedge (vismodegib); Genentech; For the treatment of basal cell carcinoma, Approved January 2012

Horizant (gabapentin enacarbil); GlaxoSmithKline; For the treatment of postherpetic neuralgia, Approved June 2012

Picato (ingenol mebutate) gel; LEO Pharma; For the treatment of actinic keratosis, Approved January 2012

Sklice (ivermectin) lotion; Sanofi Pasteur; For the treatment of head lice, Approved February 2012

Endocrinology
Belviq (lorcaserin hydrochloride); Arena Pharmaceuticals; For the chronic management of weight loss, Approved June 2012

Bio-T-Gel (testosterone gel); Teva Pharmaceuticals; For the treatment of hypogonadism, Approved February 2012

Elelyso (taliglucerase alfa); Pfizer Inc; For the treatment of Gaucher disease, Approved May 2012

Jentadueto (linagliptin plus metformin hydrochloride); Eli Lilly; For the treatment of type II diabetes, Approved February 2012

Korlym (mifepristone); Corcept Therapeutics; For the control of hyperglycemia in adults with endogenous Cushing’s syndrome, Approved February 2012

Ultresa (pancrelipase) delayed-release capsules; Aptalis Pharma; For the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions, Approved March 2012

Viokace (pancrelipase) tablets; Aptalis Pharma; For the treatment of exocrine pancreatic insufficiency due to chronic pancreatitis or pancreatectomy, Approved March 2012

Gastroenterology
Elelyso (taliglucerase alfa); Pfizer Inc; For the treatment of Gaucher disease, Approved May 2012

Ultresa (pancrelipase) delayed-release capsules; Aptalis Pharma; For the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions, Approved March 2012

Viokace (pancrelipase) tablets; Aptalis Pharma; For the treatment of exocrine pancreatic insufficiency due to chronic pancreatitis or pancreatectomy, Approved March 2012

Hematology
Elelyso (taliglucerase alfa); Pfizer Inc; For the treatment of Gaucher disease, Approved May 2012

Omontys (peginesatide); Affymax; For the treatment of anemia due to chronic kidney disease, Approved March 2012

Immunology/Infectious Diseases
Horizant (gabapentin enacarbil); GlaxoSmithKline; For the treatment of postherpetic neuralgia, Approved June 2012

Qnasl (beclomethasone dipropionate) nasal aerosol; Teva Pharmaceuticals; For the treatment of seasonal and perennial allergic rhinitis, Approved March 2012

Musculoskeletal
Elelyso (taliglucerase alfa); Pfizer Inc; For the treatment of Gaucher disease, Approved May 2012

Lyrica (pregabalin); Pfizer; For the treatment of neuropathic pain associated with spinal cord injury, Approved June 2012

Myrbetriq (mirabegron); Astellas Pharma US, Inc.; For the treatment of overactive bladder, Approved June 2012

Neupro (Rotigotine Transdermal System); UCB; For the treatment of Restless Legs Syndrome, Approved April 2012

Stendra (avanafil); Vivus; For the treatment of erectile dysfunction, Approved April 2012

Votrient (pazopanib); GlaxoSmithKline; For the treatment of soft tissue sarcoma, Approved April 2012

Nephrology/Urology
Afinitor (everolimus); Novartis Pharmaceuticals Corporation; For the treatment of renal angiomyolipoma associated with tuberous sclerosis complex, Approved April 2012

Inlyta (axitinib); Pfizer; For the treatment of advanced renal cell carcinoma, Approved January 2012

Myrbetriq (mirabegron); Astellas Pharma US, Inc.; For the treatment of overactive bladder, Approved June 2012

Omontys (peginesatide); Affymax; For the treatment of anemia due to chronic kidney disease, Approved March 2012

Stendra (avanafil); Vivus; For the treatment of erectile dysfunction, Approved April 2012

Voraxaze (glucarpidase); BTG International; For the treatment of toxic plasma methotrexate concentrations in patients with impaired renal function, Approved January 2012

Neurology
Horizant (gabapentin enacarbil); GlaxoSmithKline; For the treatment of postherpetic neuralgia, Approved June 2012

Lyrica (pregabalin); Pfizer; For the treatment of neuropathic pain associated with spinal cord injury, Approved June 2012

Neupro (Rotigotine Transdermal System); UCB; For the treatment of Restless Legs Syndrome, Approved April 2012

Subsys (fentanyl sublingual spray); Insys Therapeutics; For the treatment of breakthrough cancer pain, Approved January of 2012

Obstetrics/Gynecology
Natazia (estradiol valerate and estradiol valerate/dienogest); Bayer HealthCare; For the treatment of heavy menstrual bleeding, Approved March 2012

Perjeta (pertuzumab); Genentech; For the first-line treatment of HER2+ metastatic breast cancer, Approved June 2012

Oncology
Afinitor (everolimus); Novartis Pharmaceuticals Corporation; For the treatment of renal angiomyolipoma associated with tuberous sclerosis complex, Approved April 2012

Erivedge (vismodegib); Genentech; For the treatment of basal cell carcinoma, Approved January 2012

Inlyta (axitinib); Pfizer; For the treatment of advanced renal cell carcinoma, Approved January 2012

Perjeta (pertuzumab); Genentech; For the first-line treatment of HER2+ metastatic breast cancer, Approved June 2012

Picato (ingenol mebutate) gel; LEO Pharma; For the treatment of actinic keratosis, Approved January 2012

Subsys (fentanyl sublingual spray); Insys Therapeutics; For the treatment of breakthrough cancer pain, Approved January of 2012

Votrient (pazopanib); GlaxoSmithKline; For the treatment of soft tissue sarcoma, Approved April 2012

Ophthalmology
Zioptan (tafluprost ophthalmic solution); Merck; For the treatment of elevated intraocular pressure, Approved February 2012

Otolaryngology
Dymista (azelastine hydrochloride and fluticasone propionate); Meda Pharmaceuticals Inc.; For the relief of symptoms of seasonal allergic rhinitis, Approved May 2012

Qnasl (beclomethasone dipropionate) nasal aerosol; Teva Pharmaceuticals; For the treatment of seasonal and perennial allergic rhinitis, Approved March 2012

Pediatrics/Neonatology
Qnasl (beclomethasone dipropionate) nasal aerosol; Teva Pharmaceuticals; For the treatment of seasonal and perennial allergic rhinitis, Approved March 2012

Sklice (ivermectin) lotion; Sanofi Pasteur; For the treatment of head lice, Approved February 2012

Surfaxin (lucinactant); Discovery Laboratories; For the treatment of respiratory distress syndrome in premature infants, Approved March 2012

Pharmacology/Toxicology
Voraxaze (glucarpidase); BTG International; For the treatment of toxic plasma methotrexate concentrations in patients with impaired renal function, Approved January 2012

Pulmonary/Respiratory Diseases
Dymista (azelastine hydrochloride and fluticasone propionate); Meda Pharmaceuticals Inc.; For the relief of symptoms of seasonal allergic rhinitis, Approved May 2012

Kalydeco (ivacaftor); Vertex Pharmaceuticals; For the treatment of cystic fibrosis with the G551D mutation in the CFTR gene, Approved January of 2012

Qnasl (beclomethasone dipropionate) nasal aerosol; Teva Pharmaceuticals; For the treatment of seasonal and perennial allergic rhinitis, Approved March 2012

Surfaxin (lucinactant); Discovery Laboratories; For the treatment of respiratory distress syndrome in premature infants, Approved March 2012

Rheumatology
Stendra (avanafil); Vivus; For the treatment of erectile dysfunction, Approved April 2012

Trauma/Emergency Medicine
Lyrica (pregabalin); Pfizer; For the treatment of neuropathic pain associated with spinal cord injury, Approved June 2012

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Pharmacokinetic interaction study between quercetin and valsartan in rats and in vitro models.

Valsartan is a potent, orally active non-peptide tetrazole derivative and selectively inhibits angiotensin II receptor type 1 which causes reduction in blood pressure and is used in treatment of hypertension. The risk of heart disease mortality decreased significantly as flavonoid intake increased. Interestingly, the flavonoid-containing foodscontain a high amount of Quercetin. The objective of this study was to evaluate the influence of quercetin on the pharmacokinetics of valsartan. In vivo studies were performed on rats. Rats were treated with quercetin (10 mg/kg) and valsartan (10 mg/kg), blood samples were collected at 1, 1.5, 2, 2.5, 3, 3.5, 4, 6 and 8 h. Plasma concentration of valsartan was estimated by Reverse Phase (RP-HPLC). Quercetin significantly increases the plasma concentration of valsartan and peak concentration (70.45 µg/mL) was achieved at 3.5 h. In vitro studies were performed on rat intestinal everted sacs. The transport of valsartan from serosal side to mucosal side decreased from 53.12 ± 1.27 to 40.15 ± 0.45 µg/mL in the presence of quercetin and from 53.12 ± 1.27 to 28.68 ± 0.31 µg/mL in the presence of verapamil (standard P-glycoprotein (P-gp) inhibitor) at 120 min. Verapamil is a potent P-gp and CYP3A4 inhibitor. Quercetin is a P-gp inhibitor and may be an inhibitor of CYP3A4. The simultaneous administration of quercetin significantly increases the intestinal absorption and decreases the efflux of valsartan. The observed effect may be beneficial to develop oral valsartan dosage forms using safe P-gp inhibitor (quercetin) to improve its oral bioavailability.

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10.Links to Other Information About Clinical Trials

NHLBI Resources

Non-NHLBI Resources

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9.Finding Clinical Trials

If you’re interested in learning more about, or taking part in, clinical trials, talk with your doctor. He or she may know about studies on your disease or condition and whether they’re in your area.

You also can visit the following Web sites to learn more about clinical research and to search for clinical trials:

For more information about clinical trials for children, visit the NHLBI’s Children and Clinical Studies Web page.

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8.How Do Clinical Trials Protect Participants?

Protecting the safety of people who take part in clinical trials is a high priority for clinical researchers. Each trial has scientific oversight, and patients also have rights that help protect them.

Scientific Oversight

Institutional Review Board

Institutional review boards (IRBs) help provide scientific oversight for clinical trials. An IRB is an independent committee created by the institution that sponsors a clinical trial. IRB members are doctors, statisticians, and community members.

The IRB’s purpose is to ensure that clinical trials are ethical and that the participants’ rights are protected. The IRB reviews the trial’s protocol before the study begins.

An IRB will only approve research that deals with medically important questions in a scientific and responsible way. The IRB also checks on results during the trial. All U.S. clinical trials are required to have an IRB.

Data Safety Monitoring Board

Every National Institutes of Health (NIH) phase III clinical trial is required to have a Data and Safety Monitoring Board (DSMB). This board consists of a group of research and study topic experts.

The NIH also requires DSMBs for large trials comparing alternative strategies for diagnosis or treatment. In addition, the NIH requires DSMBs for some earlier phase trials that involve high-risk procedures (such as gene therapy) or vulnerable patients (such as children).

A DSMB’s role is to review the data from a clinical trial for safety problems or differences in results among different groups. The DSMB also reviews research results from other relevant studies. These results may reveal unknown patient risks, or they may even answer the NIH study’s research question.

Scientific oversight informs decisions about a trial while it’s under way. For example, some trials are stopped early if benefits from a strategy or treatment are obvious. That way, wider access to the new strategy can occur sooner.

Sponsors also may stop a trial, or part of a trial, early if the strategy or treatment is having harmful effects.

Patient Rights

Informed Consent

Informed consent is the process of giving clinical trial participants all of the facts about a trial. This happens before they agree to take part and during the course of the trial. Informed consent includes details about the treatments and tests you may receive, and the benefits and risks they may have.

Before you decide whether to enroll in a clinical trial, a doctor or nurse will give you an informed consent form that presents the key facts. If you agree to take part in the trial, you’ll be asked to sign the form.

You can and should ask questions about the trial to make sure you understand what’s involved. Here are some questions to ask before enrolling in a clinical trial:

  • What is the purpose of the study?
  • Who is sponsoring the study, and who has reviewed and approved it?
  • What kinds of tests, medicines, surgery, or devices are involved? Are any procedures painful?
  • What are the possible risks, side effects, and benefits of taking part in the study?
  • How might this trial affect my daily life? Will I have to be in the hospital?
  • How long will the trial last?
  • Who will pay for the tests and treatments I receive?
  • Will I be reimbursed for other expenses (for example, travel and child care)?
  • Who will be in charge of my care?
  • What will happen after the trial?

Taking part in a clinical trial is your decision. Talk with your doctor about all of your treatment options. Together, you can make the best choice for you.

Other Rights

The informed consent document is not a contract. You have the right to withdraw from a study at any time, for any reason. Also, during the trial, you have the right to learn about new risks or findings that emerge. If researchers learn that a treatment harms you, you’ll be removed from the study.

Rights and Protection for Children

Children (aged 18 and younger) get special protection as research subjects. Almost always, parents must give legal consent for their child to take part in a clinical trial.

When researchers think that a trial’s potential risks are greater than minimal, both parents must give permission for their child to enroll. Also, children aged 7 and older often must agree (assent) to take part in clinical trials.

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7.What Are the Possible Benefits and Risks of Clinical Trials?

Possible Benefits

Taking part in a clinical trial can have many benefits. For example, you may gain access to new treatments before they’re widely available. If a new treatment is proven to work and you’re in the group getting it, you may be among the first to benefit.

If you’re in a clinical trial and don’t get the new strategy being tested, you may receive the current standard care for your condition. This treatment may be as good as, or better than, the new approach. You also will have the support of a team of health care providers, who will likely monitor your health closely.

In late-phase clinical trials, possible benefits or risks of a treatment can be identified earlier than they would be in general medical practice. This is because late-phase trials have large groups of similar patients taking the same treatment the same way. These patients are closely watched by Data and Safety Monitoring Boards.

Even if you don’t directly benefit from the results of the clinical trial you take part in, the information gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many people volunteer because they want to help others.

Possible Risks

Clinical trials do have risks and some down sides. For example:

  • The new strategies and treatments being studied aren’t always better than current standard care.
  • Even if a new approach benefits some participants, it may not work for you.
  • A new treatment may have side effects or risks that doctors don’t know about or expect. This is especially true during phase I and phase II clinical trials. The risk of side effects may be even greater for trials with cutting-edge approaches, such as gene therapy or new biological treatments.
  • Health insurance and health care providers don’t always cover all patient care costs for clinical trials. If you’re thinking about taking part in a clinical trial, find out ahead of time about costs and coverage.

You should learn about the risks and benefits of any clinical trial before you agree to take part in the trial. Talk with your doctor about specific trials you’re interested in. For a list of questions to ask your doctor and the research staff, go to “How Do Clinical Trials Protect Participants?”

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6.What To Expect During a Clinical Trial

During a clinical trial, doctors, nurses, social workers, and other health care providers may be part of your treatment team. They will monitor your health closely. You may have more tests and medical exams than you would if you were not taking part in a clinical trial.

Your treatment team also may ask you to do other tasks. For example, you may have to keep a log about your health or fill out forms about how you feel.

Some people will need to travel or stay in hospitals to take part in clinical trials. For example, the National Institutes of Health Clinical Center in Bethesda, Maryland, runs clinical trials. Many other clinical trials take place in medical centers and doctors’ offices around the country.

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5.Who Can Participate in Clinical Trials?

Each clinical trial defines who is eligible to take part in the study. Each trial must include only people who fit the patient traits for that study (the eligibility criteria). For more information about eligibility criteria, go to “How Do Clinical Trials Work?”

Some trials enroll people who have a specific disease or condition. Others enroll healthy people to test new approaches to prevention, diagnosis, or screening.

In the past, clinical trial participants often were White men. Researchers assumed that trial results were valid for other populations as well.

Researchers now realize that women and people in different ethnic groups sometimes respond differently than White men to the same medical approach. As a result, the National Institutes of Health and the National Heart, Lung, and Blood Institute (NHLBI) are committed to sponsoring clinical trials that include women and are ethnically diverse.

Children also need clinical trials that focus on them, as medical treatments and approaches often differ for children. For example, children may need a lower dose of a medicine or a smaller medical device. Their stage of development also can affect how safe a treatment is or how well it works.

Children (aged 18 and younger) get special protection as research subjects. Almost always, parents must give legal consent for their child to take part in a clinical trial.

When researchers think that a trial’s potential risks are greater than minimal, both parents must give permission for their child to enroll. Also, children aged 7 and older often must agree (assent) to take part in clinical trials.

Clinical trials for children have the same scientific safeguards as clinical trials for adults. For more information, go to “How Do Clinical Trials Protect Participants?”

For more information about clinical trials for children, go to the NHLBI’s Children and Clinical Studies Web page.

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4.How Do Clinical Trials Work?

If you take part in a clinical trial, you may get tests or treatments in a hospital, clinic, and/or doctor’s office.

In some ways, taking part in a clinical trial is different from having regular care from your own doctor. For example, you may have more tests and medical exams than you would otherwise.

The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results.

Clinical Trial Protocol

Each clinical trial has a master plan called a protocol (PRO-to-kol). This plan explains how the trial will work. The trial is led by a principal investigator (PI), who often is a doctor. The PI prepares the protocol for the clinical trial.

The protocol outlines what will be done during the clinical trial and why. Each medical center that does the study uses the same protocol.

Key information in a protocol includes:

  • How many patients will take part in the clinical trial
  • Who is eligible to take part in the clinical trial
  • What tests patients will get and how often they will get them
  • What type of data will be collected during the clinical trial
  • Detailed information about the treatment plan

Eligibility Criteria

A clinical trial’s protocol describes what types of patients are able to take part in the research—that is, who is eligible. Each trial must include only people who fit the patient traits for that study (the eligibility criteria).

Eligibility criteria differ from trial to trial. They include factors such as a patient’s age and gender, the type and stage of disease, and whether the patient has had certain treatments or has other health problems.

Eligibility criteria ensure that new approaches are tested on similar groups of people. This makes it clear to whom a clinical trial’s results apply. These criteria also are a safety measure. They ensure a trial excludes any people for whom the protocol has known risks that outweigh any possible benefits.

Clinical Trial Phases

Clinical trials of new medicines or medical devices are done in phases. These phases have different purposes and help researchers answer different questions.

For example, phase I clinical trials test new treatments in small groups of people for safety and side effects. Phase II clinical trials look at how well treatments work and further review these treatments for safety.

Phase III clinical trials use larger groups of people to confirm how well treatments work, further examine side effects, and compare new treatments with other available treatments.

Steps To Avoid Bias

The researchers doing clinical trials take steps to avoid bias. “Bias” means that human choices or other factors not related to the protocol affect the trial’s results.

Comparison Groups

In most clinical trials, researchers use comparison groups. This means that the patients taking part in a trial are assigned to one of two or more similar groups. Each group will receive different medical strategies.

For example, one group may get the current standard treatment for a condition, while another group gets a new treatment. Researchers can then compare the results to see whether one group has better outcomes than the other.

Using comparison groups also ensures that no one in a study is left without treatment for the sake of research.

Sometimes, when no treatment exists for a condition, people in one group may receive a placebo (plah-SE-bo). This is an inactive product that looks like the test product. You’ll be told if a placebo will be used in a study before you agree to take part.

Randomization

Most clinical trials that have comparison groups use randomization. This involves assigning patients to different comparison groups by chance, rather than choice.

This method helps ensure that any differences observed during a trial are due to the different strategies being used, not to preexisting differences between the patients. Usually, a computer program makes the group assignments.

Masking

The term “masking” refers to not telling the clinical trial participants which treatment they’re getting. Masking, or “blinding,” helps avoid bias. For this reason, researchers also may not be told which treatments the patients are getting. However, trial records can quickly show this information if safety issues arise.

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3.Why Are Clinical Trials Important?

Clinical trials are a key research tool for advancing medical knowledge and patient care. Clinical research is done only if doctors don’t know:

  • Whether a new approach works well in people and is safe
  • Which treatments or strategies work best for certain illnesses or groups of people

Some clinical trials show a positive result. For example, the National Heart, Lung, and Blood Institute (NHLBI) sponsored a trial of two different combinations ofasthma treatments. The trial found that one of the combinations worked much better than the other for moderate persistent asthma. The results provided important treatment information for doctors and patients.

The results from other clinical trials show what doesn’t work or may cause harm. For example, the NHLBI Women’s Health Initiative tested whether hormone therapy (HT) reduced the risk of heart disease in postmenopausal women. (When the trial began, HT was already in common use for the treatment of menopausal symptoms.)

The study found that, surprisingly, the risk of stroke, blood clots, and even breast cancer increased in women who took HT. As a result, the U.S. Food and Drug Administration now recommends never using HT to prevent heart disease. When HT is used for menopausal symptoms, it should be taken only at the smallest dose and for the shortest time possible.

Clinical trials, like the two described above, help improve and advance medical care. They also can help health care decisionmakers direct resources to the strategies and treatments that work best.

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