TweetChat Recap 4/14/22: Clinical trials basics

This blog is adapted from the inaugural #CRCTrialsChat session that was on April 14, 2022 and covered Clinical Trial Basics. 

The blog follows the format of the TweetChat question & answer, where the questions asked are numbered Q1, Q2, Q3.. etc and the answers are numbered A1, A2, A3.. etc.

Here are the 11 questions covered in the hour-long session:

Q1. What are clinical trials?

A1-1. Cancer clinical trials are research studies where doctors investigate new treatments, procedures, or medical devices, to learn how safe & effective they may be in treating cancer.
The goal: Show they are more safe & effective than currently existing treatments. Here’s a figure showing the phases of clinical trials:

A1-2. Clinical trials are the last stage in the process of treatment development, and usually comes after years of preclinical lab research.

Research studies include the study of novel combinations of treatments to see:

  • If they work better
  • How they interact with each other
  • If they are safe together

A 1-3. Clinical Trials:

  • Offer patients a way to access tx based on the latest research
  • Are especially helpful for patients who have progressed through standard-of-care (SOC) treatment &/or those for whom SOC treatment yields poor outcomes

Well-conducted trials help advance understanding of cancers.

A1-4. The NCCN and ASCO guidelines recommend patient participation in clinical trials at all stages of treatment! Click on the images below to check them out.

A1-5. This @FightCRC blogpost is a great place to start reading more about the basics of clinical trials:

Q2. Why are cancer clinical trials important?

A2-1. Cancer clinical trials help doctors to find new & better ways to prevent, diagnose, & treat cancer.

CRC has only a few effective approved lines of therapy.

Clinical trials help doctors develop the next generation of therapy so patients can have better outcomes.

A2-2. Clinical trials offer patients a way to access treatments based on the latest research developments.

These may potentially be better than existing options.

Trials help advance research.

Outcomes are better for patients who participate in trials compared to those who don’t!

A2-3. The treatments your doctor is recommending to patients today were initially developed in clinical trials.

Those trial participants helped pave a better path for everyone who came after.

NCCN & ASCO recommend trial participation at every stage of treatment.

Q3. Clinical trials have eligibility criteria that govern which patients can enroll. What are eligibility criteria?

A3-1. Eligibility criteria are the set of rules that govern participation in #clinicaltrials.

Common eligibility criteria are disease type (CRC as an example), stage of disease, specific health parameters, & previous treatments received.

A3-2. Inclusion criteria: Must-have things to enroll in the trial
eg. the patient must have MSS colon adenocarcinoma.

Exclusion criteria: Things the patient MUST NOT have to enroll in the trial
eg. the patient must not have brain mets.

A3-3. It can be difficult to know whether you qualify for a clinical trial.

Best to discuss your case with the trial coordinators to see if you are eligible now.

Not eligible for one trial? Try a different one. Keep looking… Soon you may find one you qualify for!

Q4. Why do trials have eligibility criteria?

A4-1. Eligibility criteria group similar patients in a clinical trial, so scientific conclusions drawn are more likely to be accurate.

An effective drug for KRAS wild-type (WT) mCRC patients may not work in KRAS mutant mCRC.

Similar groups help find the true efficacy of a treatment.

A4-2. Eligibility criteria can allow doctors to test treatments in specific CRC patient populations in pursuit of the promise of Precision Medicine — where CRC patients are given treatments based on the particular biomarkers of their disease.

A4-3. Eligibility criteria are important safety mechanisms to ensure that patients are not in trials that could put them at increased risk.

BRAF-mutated CRC can cause aggressive disease & a trial with a surgical procedure & long recovery time may not be the best.

Q5. How is patient safety monitored during a clinical trial?

A5-1. There are two different aspects to safety in a clinical trial:

  • Safety of the participants first and foremost
  • Safety of the trial

A clinical trial must pass many safety hurdles before the trial researchers can start enrolling participants.

A5-2. Most trials must be reviewed by BOTH a Scientific Review Committee and Institutional Review Board (IRB) made up of people knowledgeable about trial conduct but with no conflicts for a particular trial.

A5-3. Principal Investigators (PIs) follow strict scientific guidelines & ethical principles to protect participants.

A5-4. Patients are protected in a clinical trial in more ways: by Institutional Review Boards (IRBs), Data Monitoring Committees and FDA inspections.

A5-5. The IRB reviews & monitors medical research involving people to make sure there is minimal possible risk to participants & that the risks are reasonable in relation to the expected benefits.

A5-6. There are also data monitoring committees and USFDA inspections of records, clinics, and research sites.

A5-7. Participant safety is monitored by the research team who follow strict scientific guidelines & ethical principles to ensure research is done safely. Each trial comes with a protocol which also lets the research team know what types of side effects to look for.

A5-8. Trial participants receive high-quality, individualized care and are closely monitored for treatment response, health and any side effects.

A5-9. Safety in clinical trials is evaluated by:

  1. Treating oncologist
  2. Study coordinators, nurses & data managers
  3. Study sponsors
  4. Data safety monitoring committees
  5. A local research ethics board at each institution participating in a trial
  6. The FDA/ Health Canada or other regulatory organizations

In summary a lot of 👀 👀 on safety

A5-10. PIs, sponsors & regulatory orgs have a role to protect volunteers to ensure studies are done properly.

They require participants go through a process called informed consent (IC)

IC gives participants complete info about the study before agreeing to take part

A5-11. The research team explains details of the study & provides an informed consent doc that has details such as:

  • Purpose & length
  • Any tests or procedures that will be done as part of the research
  • The risks & potential benefits and
  • Who to contact for more info

A5-12. As a volunteer participant you will be asked to read & sign an Informed Consent Form, & you will be given a copy to keep with your medical records.
This form must be written in a way that is easily understood when you sign it.
It is not a contract.

A5-13. Participants may leave the study at any time & for any reason.
In addition, informed consent is an ongoing process.

You have the right to be given all pertinent information & have questions answered at any time during the clinical trial.

A5-14. Patient safety is very important in clinical trials.

Every study must go through a rigorous review process before approval.

The informed consent process informs of possible risks, & patients are given the chance to ask questions about the trial.

A5-15. During a clinical trial, patients are monitored on a regular basis for any concerning side effects from the treatment. Clinical trials are also overseen by a large group of people and agencies, including researchers, doctors, and review boards.

Q6. What questions should patients ask before joining a trial?

A6-1. Patients should ask about the potential benefits & risks associated with the trial.

Particularly, if there is ANY previous data on the treatments being studied. Pre-clinical data? Previous trials? Case reports? What do we know so far?

A6-2. Why do the researchers believe the new treatment may be effective? Would they recommend this trial to a family member?

Are there any biomarkers associated with the trial? Is there evidence this biomarker is useful or predictive?

Q7. Do clinical trials use placebos?

A7-1. Patients worried they will not get treatment while on a trial is a common misconception.

Most trials do not have a placebo-only arm.

In early phase trials, patients will generally always receive the study treatments.

A7-2. Typically in later phase trials, new drugs are compared to SOC treatment so that in most cases, patients receive active treatment for CRC.

In 3 or 4th line setting, the control arm may be the best supportive care, effective 3 or 4L drugs for CRC aren’t available.

Q8. Who pays for the costs of a clinical trial?

A8-1. Generally 2 direct sets of costs for participating in a trial.

1st set:

  • Research costs that include the treatment being studied
  • Additional tests or procedures required for the trial

These are often covered by the sponsor or institution running the trial

A8-2. Clinical trials direct costs.

The 2nd set of direct costs is standard medical care costs:

  • Treatments, tests, or procedures that patients would normally receive even if they were not participating in a trial

Insurance will often cover these costs.

A8-3. Clinical Trials direct costs.

There may be additional costs. Often these are covered by the trial sponsor, but it’s important to clearly know the costs your insurance & the study sponsors will not cover.

In many cases, there are no additional direct costs!

A8-4. Clinical trials ancillary costs.

Such as travel & lodging costs if the trial is not nearby. Some trial sponsors & institutions cover these, & many amazing organizations can help patients with these additional participation costs. 

A8-5. Clinical trials costs.

The National Institutes of Health (NIH), in Bethesda Maryland, runs 100s of trials & have many great programs that cover costs including travel & lodging expenses.

Participating in a trial at the NIH is almost always entirely free.

Q9. What happens to patients when a trial ends?

A9-1. When a trial ends there may be patients who are on the study treatment & still benefitting.

In these circumstances, it is common for the treatment to continue to be given to the patient for a set amount of time, usually predetermined in the trial outline.

A9-2. Sometimes patients who are benefitting from a clinical trial are allowed to continue on the treatment indefinitely until it is no longer helping them. Not always the case though.
Important to clarify how long the study drug may be available if it’s working.

A9-3. On occasion, the data generated from the trial will suggest the treatment is not beneficial for patients & the trial will come to an end.

In these instances, the treatment is usually stopped for all patients.

Q10. What are some common clinical trial myths?

Q10-1 Myth or fact?: “I might get a placebo”

A10-1. Myth!

Most trials: no placebo-only arm.

Early phase trials: patients will mostly always get study treatments.

Later phase: new drugs are compared to a SOC treatment, majority of cases, patients get active treatment for CRC.

Q10.2 Myth or fact?: “I am just a Guineapig being tested on”

A10-2. Myth!

Treatments in trials go through years of testing in the lab & in animals before getting tested in humans.

This preclinical research provides valuable info that can help ensure patient safety in trials.

Clinical trials also follow strict safety protocols and patients often find they receive extra attention and personal care in a trial from the specialized trial team, though this may not always be the case. Always good to ask questions and learn more.

Q10-3 Myth or fact?: “Clinical trials are a last resort”

A10-3. Myth BUSTED!

Clinical trials are available to patients throughout every part of their cancer journey even including cancer prevention studies!

Clinical trials often include the standard treatment you would normally get from your local oncologist, paired with promising new therapeutics that researchers hope will work better than the standard treatment alone.

Patients who enroll in clinical trials have better outcomes than those who don’t.

NCCN & ASCO recommend trial participation at every stage of treatment.

Q10-4 Myth or fact?: “If I enroll in a trial, I’m not allowed to quit”

A10-4. Myth!

Patients always have full autonomy in a trial and can decide to stop the trial at any time, for any reason.

Q10-5 Myth or fact?: “Clinical trials are dangerous”

A10-5. Myth!

Doctors have become very skilled at clinical trial design that optimizes patient safety. Strict safety protocols ensure that patient safety is the number one priority for everyone involved.

Q11. How can CRC patients find clinical trials that might work for them?

A11-1. This is a vitally important question is covered in the next #CRCTrialsChat.
Meanwhile, visit CTU Clinical Trials Learning Center!!

If you want to see the tweets, you can also check them out at the Wakelet here, without going on Twitter.

A blog post covering the session of clinical trial finders will be available shortly.