Supporting People with Breast Cancer 2012

Annual Patient Conference

Clinical Trials – Exploring your Options

Deirdre McDonnellClinical Research Nurse

St. Vincent's University Hospital

Research TeamSt. Vincent's University Hospital

Options

•You will usually be invited to take part in a clinical trial because you are a patient who needs treatment. If your hospital has a clinical trial open that might be suitable for you, You may be offered this trial and will then have to consider your options.

•It is not an easy decision. You will have to weigh up all the pros and cons of either option and decide what’s important to you and what type of treatment your happy with.

Best licenced

treatment

No conventional treatment

Clinical trial participation

What are my options?

Who conducts trials

•Every clinical trial is led by a principle investigator who is often a medical doctor. The research team may include doctors, nurses, social workers and other health care professionals.

•Clinical studies can be sponsored, or funded by pharmaceutical companies, academic medical centres and other organizations in addition to government agencies.

Voluntary

Clinical trial participation is entirely voluntary and you cannot take part in a clinical trial unless you freely give your permission.

The rights, safety, and well-being of trial participants are the most important considerations and prevail over interests of science and society.

Clinical trials overview

Demystify Clinical Trials

Trials terminology

Phases

Benefits

Drawbacks

Costs

Informed consent

Protections

More information

What is a Clinical Trial?

•Cancer research studies are carried out to try and find new and better treatments for cancer. Trials which are carried out on patients are known as clinical trials.

•A clinical trial involves research using human volunteers (also called participants) that is intended to add to medical knowledge.

Why do Clinical Trials?

•The aim is to answer specific questions about new medical treatments.

•Clinical trials are used to determine whether new drugs are safe and effective.

•Clinical trials are conducted in phases and each phase has a different purpose.

Why are clinical trials important?

•By taking part in clinical trials, cancer patients can help test if new approaches to cancer prevention, diagnosis and treatment are safe and effective. This information will help society and future generations.

•Due to progress made through clinical trials, many people treated for cancer are now living longer and more cancers are being cured.

•Cancer has touched every family in Ireland and in most cases more than once. It is vital that we all work together to improve the future for Irish people with cancer.

Types of Research

•Epidemiological research looks for various risk factors for various cancers so that the disease may be prevented.

•Basic lab research tries to unravel the mystery of the cells and why some cells behave abnormally and become cancerous.

•Clinical research identifies the most effective therapy or combination of therapies to treat cancer and other diseases.

•Translational Research combines Lab and Clinical Research

1st Clinical Trial?

•Perhaps the first ever clinical trial was James Lind’s demonstration in 1753 that citrus fruits cured scurvy. He compared the effects of various different acidic substances ranging from vinegar to cider, on groups of sailors, and found that the group who were given oranges and lemons had largely recovered from scurvy after 6 days.

From idea to proven treatments?

Pre clinical testing

•Before scientists can conduct trials in human beings, they must conduct extensive laboratory research.

•Research usually involves years of experiments in animal and human cells.

Preclinical testing

•If preclinical testing of a drug of is successful, the sponsor then provides the data to the Regulatory Body requesting approval to being testing in humans.

•If approved by the Regulatory body testing in humans begins. This is done through a formally written and approved study protocol.

Phase 1

•First step in testing in humans.

•Establish how a new drug should be given (tablet or injection), how often, and what dose is safe.

•Researchers look for safety and potentially harmful side effects.

•Usually include only a small number of participants.

•This phase of testing usually takes several months.

Phase 2

•Once a drug has shown to be safe, then it must be tested for efficacy.

•This phase may last from several months to two years.

•Usually involves several hundred patients

•Most of these trials are randomized trials

•Only about 1/3 of studies successfully complete both phase I and phase 2 due to poor patient activity or toxic effects.

Phase 3

•Studies move into phase 3 only after a treatment has shown promise in phase I and II trials.

•Conducted in many centres and include large groups of patients (300-3000)

•Compare the results of patients on the experimental Arm to those patients having standard treatment.

•A participant will usually be assigned to the standard group or the new group at random.

•Many phase 3 trials are randomized and blinded.

Drug development

• For every 1,000 compounds that are identified by a company, only about 30 show promising results.

• And for every 30 compounds that show promise, three get past the first round of clinical trials and finally, only one hit the market.

•Can take 12 – 15 years from an idea to licenced treatment.

•It is estimated, according to various sources, that cost of developing a single new drug including commercialization varies from US $ 800 million to US $ 1.7 billion

Randomisation

•You are assigned to a treatment group by chance, similar to “flipping a coin”.

•Randomization helps avoid bias.

•The assigned groups are often referred to as “arms”.

Example: Patient #1 is assigned to Arm A of the trial, which involves the new treatment. Patient #2 is assigned to Arm B, which is the standard treatment.

Blinded studies

Blinding means that whoever is assessing the effects of treatment will not know if they are studying patients on the treatment or patients on placebo. This helps to prevent bias.

Double blinded studies are where neither the patient or the doctor know whether the patient is receiving the actual study drug or standard drug.

When no standard is available, some studies compare new drugs with placebo drugs.

Patients are “unblinded” only if it becomes medically necessary prior to the end of the study.

What is a placebo?

•A placebo is a treatment that does not contain any active substance.

• It allows investigators to test for the ‘placebo effect’. This is a psychological response where people feel better even though they do not take a medicine with an active ingredient.

• By comparing people’s responses to the placebo and to the treatment being tested, the benefit of the treatment can be described.

What is a protocol?

•A study plan on which all clinical trials are based.

•Carefully designed to protect the health of all participants.

•Describes what types of people may participate in the trial (inclusion and exclusion criteria)

•Gives detailed schedule of tests, procedures, medications, dosages, and length of the study.

Considering enrolling in a study?

•Your Doctor or Research nurse is there to discuss all aspects of the study with you. It is a good idea to have a family member or friend with you to help ask any questions or just for support.

•You will be given a Patient Information Leaflet and an Informed Consent Form to read and discuss.

•You should only agree to take part in a trial if you are entirely happy with what you are being asked to do.

Informed Consent Form

•Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate.

• If you sign this form, you confirm that you believe you have been given all the important facts about a trial, you understand them and that you have decided to take part in the trial of your own free will.

•An informed consent is not a contract and you may withdraw from the study at any time.

Informed Consent Form

•You will be asked to read the information carefully and encouraged to ask as many questions as you wish.

•It includes details about: the aim of the study, background information about the treatment, how the treatment will be given and for how long, the benefits and drawbacks, possible side effects and confidentiality.

Informed Consent Form

•If you wish to take part in the study you then sign the consent form.• It may be necessary for a witness to sign the consent form with you.

• You can withdraw your consent at any time if you wish to do so or your doctor may take you off the study if he/she feels it’s in your best interest.

•If you do not wish to take part you will receive the best treatment currently available for your condition.

Quality of life

•Over the last few years, researchers have developed detailed tests and questionnaires that allow them to measure people’s quality of life.

• These allow them to look not only at the effects of a new treatment on your cancer but also at its much wider effects on your life as a whole.

Eligibility Criteria

•All trials have guidelines about who can take part. These are called ‘eligibility criteria’, consisting of inclusion criteria (i.e. who is suitable to participate) and exclusion criteria.

• Not everyone who suffers from a particular disease can take part in a trial.

•The researchers may only be including a subset of people with a particular disease.

•There may be safety concerns which may exclude you from a study for example high blood pressure.

Inclusion Criteria

• Inclusion criteria help investigators decide who can join a trial.

• For example, some trials only include people of a certain age, or at a particular stage in their illness.

• You may have to have a medical examination, scans or special tests before enrolling to assess whether you are suitable to take part.

Potential Benefits

•You may be one of the first to benefit from the latest cancer treatments before it becomes widely available

•Your health care and response to treatment will be closely monitored during and after the trial.

•You have an opportunity to contribute to science and provide information which may be of benefit to you and others with the same health problem.

•Research has found that people receiving treatment within clinical trials tend to do better than similar patients treated outside trials.

Potential Drawbacks

•You may not always receive the new treatment, or the new treatment may not prove to be any better than treatments currently available.

•The new treatment may be effective but may not work for you.•New treatments may cause side effects not previously known to doctors. •You may have to visit the clinic or hospital more often because you are being monitored more closely•You may have to undergo more tests (for example blood tests) then you would usually have in routine care

Costs?

•You do not have to pay for treatments received as part of a study.

•You may have to pay for treatments which are considered standard of care.

•Some clinical trial sponsors will reimburse travel expenses for extra visits.

•Clinical trials are usually funded by pharmaceutical companies.•Your Study Doctor/Hospital will receive payments for conducting clinical trials.

What regulations are in place?

The Nuremberg Code is a set of research ethics principles for human experimentation set as a result of the Subsequent Nuremberg Trials at the end of the Second World War.

The “Declaration of Helsinki” was developed by the World Medical Association 1964. This sets the ethical standards for research involving human beings, human material or identifiable data.

What regulations are in place?

•Since 2004, clinical trials in Ireland involving medicinal products for human use have been covered by the European Communities (Clinical Trials on Medicinal Products for Human Use) Regulations.

•ICH -GCP International conference for the harmonization of Good Clinical Practice.

•All trials are also closely regulated by the Irish Medicines Board (IMB).

How are you protected?

•A clinical trial has to be approved by the Irish Medicines Board.

•This government department decides if a clinical trial can take place in Ireland.

•It ensures that the trial will be carried out in line with current Irish, EU and International standards for clinical research.

How are you protected?

•A Clinical trial has to be approved by a national Ethics Committee

•An Ethics Committee must have at least 5 members and is composed of health care professionals and investigators as well as members of the public.

•You must be thoroughly informed about a study’s treatments and tests before deciding whether or not to participate in the study and sign an informed consent form.

Questions to ask……

•What is the purpose of the trial?• What is the trial called?• What treatment will I be receiving?• What is the difference between the trial treatment and standard treatment?• What are the side effects of the trial treatment?• What are the benefits and risks of taking part in the trial?• How many patients are taking part in this trial?• Are there any extra hospital visits required?• Will the trial cost me money?• What treatment will I have if I do not want to take part in the trial?• What will happen if I want to discontinue the trial treatment?• When will the trial results be available?

Exploring your options

www.icorg.ie

• All Ireland Cooperative Oncology Research Group.You can log on and find out what clinical trials are currently open in Ireland for different types of cancer and what hospitals are participating in the studies.

www.cancer.ie

• The Irish Cancer website has information on Current research in Ireland.

ICORG Breast cancer studies

•SUPREMO \ ICORG 04-11 •CADY \ ICORG 08-01• NSABP B-39 \ ICORG 08-06•TRIO 018 \ ICORG 09-01• Proteomic Study of Breast Cancer \ ICORG 09-07•Lap/Epi Phase II \ ICORG 09-13•MYOCET \ ICORG 10-03•TCHL \ ICORG 10-05 •TROG/BIG DCIS \ ICORG 10-06•Denosumab D-CARE Amgen \ ICORG 10-08•Circulating miRNAs \ ICORG 10-11•Sorafenib MBC \ ICORG 11-06•GSK EGF114299 \ ICORG 11-08•TH v THL \ ICORG 11-10•NSABP B-47 \ ICORG 11-24 •SWOG S1007 \ ICORG 12-01•

Exploring your options

www.clinicaltrialsregister.eu

Since March 2011 the European Medicines Agency has made a database of drug clinical trials publically accessible

www.clinicaltrials.gov

In the US, the authorities have created a website where trials conducted around the world can be found.

IPPOSI is a unique partnership of Patient Organisations, Science and Industry. This patient led group focuses on policy, legislation and regulation around the development of new medicines.

How to Explore your options

Is there a trial for that?

I wish to pay tribute to all the brave women and men who have participated in trials of the past to provide us with the treatments of today….and to the brave women and men participating in trials today providing us with the treatments of tomorrow.

Thank you Deirdre McDonnell