Stem Cell Therapies and Clinical Trials

Clinical research involves studies in which patients or healthy people participate. Stem cell therapy developers need to understand how to conduct clinical trials in order to process FDA investigational new drug (IND) applications. Stem cell therapies are generally considered drugs – which means stem cell therapies require FDA approval.

Some of the goals of clinical research include:

• Developing new treatments or medications
• Identifying causes of illness
• Studying trends
• Evaluating ways in which genetics may be related to an illness.

Anyone concerned about FDA oversight of a particular stem cell therapy can look for the FDA-issued Investigational New Drug Application number to review the IND.

The first step in a clinical trial normally begins with new therapy research or procedures in a laboratory – and with animal studies. The research and procedures that show the most promise then advance to the clinical trial stage so researchers can learn more about the effectiveness of the treatments and the risks.

The National Institutes of Health have very strict rules for how clinical studies should be conducted. Some of the studies in clinical studies focus on directly helping the participants while others have a more intermediary benefit.

Confidentiality during clinical trials

Only researchers with proper authorization should have access to the information obtained during the clinical study. Only these researchers should know the personal identity and medical information of the participants. Individual patients should know their own medical information. The results of the clinical study should be presented in broad terms without identifying specific participants.

Clinical research versus medical treatment at a healthcare provider’s office

The key differences include the following:

• Intent. Clinical research aims to answer specific questions through various researchers. Medical treatment addresses the needs of a specific patient.
• The intended benefit. Clinic research aims to benefit future patients. Medical treatments aim to benefit a specific current patient.
• Funders. Normally, government agencies and drug developers fund clinical research. The patients and their health plans pay for medical treatments.
• Timeframe. Clinical research responses depend on research protocols. Medical treatment requires real-time responses.
• Consent. Clinical research requires a written informed consent by each participant. Medical treatments may/may not require informed consent.
• Assessment. Clinical research “involves periodic and systematic assessment of patient data. Medical treatment is based on the patient’s assessment.
• Safeguards. Clinical research is governed and regulated by government agencies, institutional review boards, professional standards, informed consent, and legal standards. State medical boards, “professional standards, peer review, informed consent, and legal standards” regulate medical treatments.
• Certainty. Clinical research “tests products and procedures of unproven benefit to the patient.” Medical treatments use “products and procedures accepted by the medical community as safe and effective.”
• Information access. Clinical research is considered confidential intellectual property Medical treatments are “available to the general public through product labeling.”
• Release of findings. Clinical study results are usually published in medical journals. Medical records are generally confidential and not available to the public.

Which people should consider participating in a clinical trial?

People participate in clinical trials for different reasons. Common reasons for participation include the fact that other treatments haven’t worked or the treatments cause intolerable side effects. Some people participate to help “contribute to the advancement of medical knowledge.”

Clinical trials do have eligibility criteria “based on such factors as age, sex, type and stage of disease, previous treatment history, and other medical conditions.” The eligibility criteria helps ensure that the results of the study will help researchers become more informed about the questions the scientists seek to answer. Many people who apply for a clinical study are not accepted.

Generally, drugs and medical devices should be tested in the people the drugs/devices are meant to help. Clinical studies need to include a broad range of different people to have a broader view of how different people may respond to the treatments. “FDA seeks to ensure that people of different ages, races, ethnic groups, and genders are included in clinical trials. Learn more about FDA’s efforts to increase diversity in clinical trials.”

Where are clinical trials conducted?

There are usually different sponsors for clinical trials. These may include:

• Pharmaceutical companies
• Federal agencies such as the National Institutes of Health or the U.S. Department of Veterans Affairs
• Physicians and other healthcare providers

Clinical studies are usually “conducted at universities, medical centers, clinics, hospitals, and other federally or industry-funded research sites”

The safety of clinical trials?

“FDA works to protect participants in clinical trials and to ensure that people have reliable information before deciding whether to join a clinical trial.” This is why there are federal regulations and guidelines for clinical research – so that participants are protected from unreasonable risks. Some risks may be unavoidable since the aim is to learn what medical treatments work and which ones do not work.

The government does require that researchers give “prospective participants complete and accurate information about what will happen during the trial.” Participants will receive an informed consent document that explains the study, the possible risks, and the rights of the participants. By signing the informed consent, a participant acknowledges that he/she “understands that the trial is research” and that the participant may leave at any time.

What should participants consider before joining a clinical trial?

Clinical participants shouldn’t enter into a trial without reviewing the various risks, reviewing their concerns, and getting answers to their questions from the healthcare team conducting the trial. If a participant is being treated for a medical disorder, the FDA recommends that participants discuss with their healthcare provider whether or not the trial is a good option for their current treatment.

Participants should understand:

• “What happens during the trial
• The type of health care you will receive
• Any related costs once you are enrolled in the trial
• The benefits and risks associated with participating.”

Does the FDA conduct its own clinical trials?

No. The FDA does not conduct its own clinical trials. The agency oversees the clinical trials that are taking place. The agency meets with the researchers, conducts inspections, of the clinical trial study sites to protect the rights of patients, and verifies the quality and integrity of the results

How can people learn about clinical trials?

The first place to start is to speak with your doctor. Your physician may even recommend you for a clinical trial. Other sources of information include:

• “FDA Clinical Trials Search. Search a database of Federally and privately supported studies available through clinicaltrials.gov. Learn about each trial’s purpose, who can participate, locations, and who to contact for more information.
• Clinicaltrials.gov. Conduct more advanced searches
• National Cancer Institute or call 1–800–4–CANCER (1–800–422–6237). Learn about clinical trials for people with cancer.”

What are placebos?

“A placebo is a pill, liquid, or powder that has no treatment value. It is often called a sugar pill.” Clinical trials often use placebos to compare a treatment’s effectiveness to a treatment that should have no effect. Placebos are commonly used in clinical trials. Usually, neither the patient nor their doctors know who is receiving the real treatment and who is receiving the placebo.

“Many cancer clinical trials, as well as trials for other serious and life-threatening conditions, do not include placebo control groups because of the need to use treatments that might reasonably work. In these trials, all participants receive the experimental drug.”

You should review the possibility you may receive a placebo with the trial coordinator and your physician.

How do I find out what phase a drug is in as part of the clinical trial?

You can speak with the clinical trial coordinator to learn which phase the clinical trial is in. Clinical trial phases during the Investigational New Drug (IND) Process normally include a few phases with each subsequent phase having more participants.

Most preclinical (animal) research drugs never get to the human testing phase.

What are the different types of clinical research?

The different types of clinical research include:

• Treatment Research. This type of research usually “involves an intervention such as medication, psychotherapy, new devices, or new approaches to surgery or radiation therapy.”
• Prevention Research. This type of research “looks for better ways to prevent disorders from developing or returning. Different kinds of prevention research may study medicines, vitamins, vaccines, minerals, or lifestyle changes. “
• Diagnostic Research. This type of research “refers to the practice of looking for better ways to identify a particular disorder or condition.”
• Screening Research. This research study “aims to find the best ways to detect certain disorders or health conditions.”
• Quality of Life Research “explores ways to improve comfort and the quality of life for individuals with a chronic illness. “
• Genetic studies “aim to improve the prediction of disorders by identifying and understanding how genes and illnesses may be related. Research in this area may explore ways in which a person’s genes make him or her more or less likely to develop a disorder. This may lead to the development of tailor-made treatments based on a patient’s genetic make-up.”
• Epidemiological studies examine “the patterns, causes, and control of disorders in groups of people.”

Outpatient research means a participant does not stay overnight at the hospital. Inpatient research means the participant does stay at least one night in the hospital or research center.

What are the different phases of a clinical trial?

Clinical research that is used to evaluate medications and devices generally involves the following phases:

The standard phases of a clinical trial to review medications (such as stem cell therapies) and medical devices generally include:

• Phase I trials. This trial normally involves a small group of people, to “evaluate the treatment’s safety, determine a safe dosage range, and identify side effects.”
• Phase II trials. This phase involves a larger group to examine if the drug/device is effective and to further evaluate its safety.
• Phase III trials. “The experimental study drug or treatment is given to large groups of people. Researchers confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.”
• Phase IV trials. This phase involves “post-marketing studies, which are conducted after a treatment is approved for use by the FDA, provide additional information including the treatment or drug’s risks, benefits, and best use.”

Additional FDA clinical research trial issues

As discussed above, there are additional rules for informed consent and diversity in Clinical Trial Participation, issues involving Sexual and Gender Minorities, and other issues.

Approval of stem cell therapies as drugs normally requires clinical studies. There are specific legal and regulatory compliance issues along with scientific compliance issues for clinical studies that seek FDA approval.

Stem cell developers and manufacturers should contact Cohen Healthcare Law Group, PC to discuss FDA’s stem cell and clinical trial rules. Our experienced healthcare attorneys advise healthcare companies and medical practices about healthcare compliance laws and regulations.