clinical trials in biomedical engineering

1. TOPIC: CLINICAL TRIALS OF DRUGS AND MEDICAL
DEVICES FROM PHASE 1 – 4
Joseph Asamoah-Asare
10934524

2. OUTLINE
2
1. Introduction
2. Importance of Clinical Trials
3. Types of Clinical Trials
4. Phases of Clinical Trials
5. Classes of Medical Devices
6. Medical Devices vs. Drug Clinical Trials
7. Cost of Medical Device Clinical Trials
8. Conclusion
9. References

3. INTRODUCTION
● Demonstrating the effectiveness and safety of new medical products is a critical
part of the medical product development process.
● Clinical trials are fundamental to the development of innovative, investigational
products such as drugs or high-risk (and some medium-risk) medical devices.

4. INTRODUCTION cont.
● Clinical trials are research studies that are conducted in people (healthy
participants or patients with a specific health issue) in order to study and test new
medical treatments, such as drugs, vaccines, medical devices, medical procedures
and diagnostic tests.
● Clinical trials may also be conducted to study new combinations of treatments, or to
compare treatments, or to study an already available treatment for a new use
● The whole idea behind a clinical trial is for us to gain the knowledge to treat patients
better.

5. IMPORTANCE OF CLINICAL TRIALS
● Clinical trials are a critical part of evidence-based medicine, as they help test out
how new medical treatments, tests or vaccines will work.
● They are important for discovering new treatments for diseases, as well as new
ways to detect, diagnose, and reduce the chance of developing the disease.
● Clinical trials can show researchers what does and doesn’t work in humans that
cannot be learned in the laboratory or in animal studies.
● They also help doctors decide if the side effects of a new treatment are acceptable
when weighed against the potential benefits.

6. IMPORTANCE OF CLINICAL TRIALS cont.
● In addition to testing new drugs and devices, clinical trials provide a scientific basis
for advising and treating patients. Even when researchers do not obtain the
outcomes they predicted, trial results can help point scientists in the correct
direction.
● Although some clinical trials have had major setbacks, millions of people have
been helped because other people before them chose to participate in a trial that
resulted in a new, more effective treatment.

7. TYPES OF CLINICAL TRIALS
● Treatment Trials: test experimental treatments, new drugs, combinations of drugs,
medical devices, natural health products, or new approaches to surgery or therapy.
An example of an experimental treatment is an investigational pacemaker.
● Prevention Trials: test ways to prevent disease or prevent a disease from
returning. The approaches may include drugs, vitamins, minerals, natural health
products, educational programs, and lifestyle/behavioral change. Examples include
the use of a plant extract used to boost the immune system.

8. TYPES OF CLINICAL TRIALS cont.
● Quality of Life Trials: test or explore ways to improve comfort and the quality of
life for individuals with a chronic illness, disability, or condition. For example, trials
may look at better ways to manage people with end of life diseases.
● Diagnostic Trials: are designed to find better tests or procedures for diagnosing a
particular disease or condition. For example, a trial to determine if ultrasound is
more effective than mammography in detecting breast cancer.
● Screening Trials: are conducted to test the best way to detect certain diseases or
health conditions. Example, trial to test a new blood test that would detect markers
that indicate cancer may be present in a person’s body.

9. PHASES OF CLINICAL TRIALS
● Clinical research is broken up into a series of “phases”, each with a different distinct
purpose, beginning with (though not always conducted) pilot studies, then safety
testing, then efficacy testing, then clinical efficacy, and, if approved for market
release, post-market monitoring.
● New pharmaceuticals/drugs all undergo this clinical trial phase sequence.
● However for medical devices, the sequence is fairly similar, and some devices do
go through a clinical trial phase process, however, most medical devices will go
through clinical trial “stages”, instead of phases.

10. PHASES OF CLINICAL TRIALS cont.
Typically, clinical trials are conducted in four sequential phases with each phase involving a
larger sample of participants. However there is a Phase 0 which is not required by the FDA,
but is suggested particularly for drugs that aim to treat serious diseases.
PHASE 0: A relatively new step in the clinical trial process, this early stage is intended to
quickly weed out ineffective drugs, or establish that a drug will work as expected in the
body. This stage only enrolls a few volunteers, who are given just 1% of the dose of the
drug being researched. Phase 0 trials take no more than seven days. These short trials give
researchers a sense of how the drug will behave in the body.
PHASE I: Trials are conducted to assess safety. Usually the testing involves a small
number (about 10-100) of healthy participants.

11. PHASES OF CLINICAL TRIALS cont.
PHASE II: Trials assess effectiveness and further evaluate safety. This phase of trial
usually involves participants who have the disease or condition under investigation.
Normally participants are randomly assigned to groups or conditions, with one condition
being the treatment (program, drug, etc.) under consideration, and at least one comparison
condition. Studies involve a limited number (about 100-300) of closely monitored
participants.
PHASE III: The new drug or treatment is given to large groups of people (1,000–3,000) to
confirm its effectiveness, monitor side effects, compare it with standard or similar
treatments, and collect information that will allow the new drug or treatment to be used
safely.

12. PHASES OF CLINICAL TRIALS cont.
PHASE IV: Trials may explore specific effects, or be designed to determine effects on
morbidity and mortality or on specific populations. For drugs, natural health products, and
medical devices, Phase IV trials are conducted after the drug, medical device, or natural
health product is on the market. After a drug is approved by the FDA and made available to
the public, researchers track its safety in the general population, seeking more information
about a drug or treatment’s benefits, and optimal use.

13. PHASES OF CLINICAL TRIALS cont.

14. CLASSES OF MEDICAL DEVICES
● A medical device is any “article, instrument, apparatus or machine that is used in
the prevention, diagnosis or treatment of illness or disease, or for detecting,
measuring, restoring, correcting or modifying the structure or function of the body
for some health purpose” (World Health Organisation).
● This includes something as simple as an adhesive bandage, artificial body parts
(prostheses), any surgical, diagnostic or monitoring equipment/devices, as well as
advanced, implanted devices such as pacemakers.
● They are classified based on their intended use, invasiveness, duration of use, and
the risks and potential harms associated with their use.

15. CLASSES OF MEDICAL DEVICES cont.

16. MEDICAL DEVICES VS. DRUGS CLINICAL TRIALS
● Unlike drugs, medical devices can gain a CE mark through a literature/clinical
evaluation route, which used to be considered the norm.
● However, this is becoming less and less common because more clinical evidence is
being demanded.
● Medical devices just like drugs must be approved for use, but go through a somewhat
different process.
● Devices are not tested on people without the condition, instead, they are tested first for
safety and performance in what is called a "pilot" or feasibility study with a small group
of patients, typically 10 to 30.

17. MEDICAL DEVICES VS. DRUGS CLINICAL TRIALS
cont.
● Next, a "pivotal" trial enrolls a larger population of patients, from 150 to 300
participants, to further test safety and effectiveness.
● The gold standard of study design for clinical trials is a randomized controlled trial,
preferably with blinding and a placebo as the control, however unlike drug trials,
medical device trials are not able to use a placebo and it is typically impossible to do
blinding. Instead, new medical devices are typically measured against the existing
standard of care.
● Post-approval studies collect long-term information, like post-marketing studies for drug
trials do.

18. MEDICAL DEVICES VS. DRUGS CLINICAL TRIALS
cont.
● Not all new medical devices need to go through clinical trials. Some are quite simple
and considered low-risk, such as new bandages or tongue depressors, which are both
considered medical devices.
● The table below compares study design elements between drug & medical device trials.

19. COST OF MEDICAL DEVICE CLINICAL TRIALS
● There is no denying that clinical trials are expensive propositions.
● There is little economy of scale; by and large and big pivotal trials cost proportionally
more than small studies.
● The best thing one can do is design the study well to collect only the most relevant
data, size it properly based on solid statistical grounds, select investigative sites
judiciously to provide sufficient numbers of subjects, and manage the trial efficiently.
● These actions will not make a study inexpensive, but emphasis on consistency and
paying strict attention to all the details will help minimize the cost.
● The cost drivers for clinical studies are numerous, vary between studies, and are
certainly subject to change as time goes by.

20. COST OF MEDICAL DEVICE CLINICAL TRIALS
cont.
● The table below shows the expenses per enrollee in a medical device clinical trial.

21. CONCLUSION
● Clinical trials are exciting, exhausting, and expensive.
● As an innovator of a new medical device, you need to decide what role you want to
play in the life cycle of your device (concept to commercialization).
● Do you want to give it up for adoption (sell the IP early), or do you want to try to raise it
to adulthood (obtain full regulatory approval to market), or do you want to participate in
something in between?
● That answer will help you decide if you need to conduct a clinical study on your device
and if so, the type of study.

22. REFERENCES
1. Kaplan, AV, Baim, DS, Smith JJ, et al. “Medical Device Development: From Prototype to Regulatory
Approval”. Circulation 2004;109(25):3068-3072.
2. Van Norman, GA. “Drugs, Devices, and the FDA: Part 2: An Overview of Approval Processes: FDA
Approval of Medical Devices”. JACC: Basic to Translational Science 2016;1(4):277-287.
3. Chittester B. Medical device clinical trials—how do they compare with drug trials? 2014.
https://www.mastercontrol.com/gxp-lifeline/medical-device-clinical-trials-how-do-they-compare-with-drug-
trials. Accessed August 31, 2022.
4. Government of Canada. Health Canada. Drug and medical device approval overview.
https://www.canada.ca/en/services/health/drug-health-products/drug-medical-device-highlights-
2017/approval-overview.html Accessed September 16, 2022.
5. World Health Organisation: “Medical devices”: http://www.who.int/medical_devices/full_deffinition/en/

23. THE END
THANK YOU