Who Can Join Clinical Trials to Access New Medicines? What Is the Situation in Our Country?

Research conducted in humans, concerning a clinical event and prospectively examining an intervention’s effect and value against a control group, comparing these outcomes in forward-looking (prospective) investigations, is called a clinical trial .

Interventions can include drugs, surgery, medical devices and prostheses, diet, physical therapy, diagnostic procedures or education.

Participating in a Clinical Trial

A clinical trial follows a research plan called a protocol . Designed to answer study-specific questions and protect participants’ health, it contains:

• The reason for the study
• Who may participate (eligibility / inclusion criteria)
• Required participant number
• Schedules and doses of tests, procedures or medicines
• Study duration
• What participant information will be collected

Who Can Participate in Clinical Trials?

Trials have standards determining who may participate. These are called eligibility criteria and listed in the protocol. Some studies seek participants with the condition being investigated, others healthy volunteers, while others are limited to a predefined group researchers wish to enroll.

Eligibility: Factors permitting participation are called inclusion criteria , while factors disqualifying participants are called exclusion criteria . They are based on age, sex, disease type and stage, prior treatment and other medical conditions.

How Are Participants Protected, and What Are Their Rights?

Informed consentis the process researchers use to explain a trial to potential and enrolled participants. Information helps people decide whether to enroll or continue. The process protects participants and must provide enough information to understand risks, potential benefits and alternatives.

Generally, before joining, participants sign an informed consent document indicating they received and understood information about risks, benefits and alternatives. Signing and consenting is not a contract. Participants may withdraw at any time, even before the study ends.

Authorized national and international review bodies protect participants’ health and well-being. In our country, these are Ethics Committees, while internationally, particularly the United States Food and Drug Administration (FDA), European Medicines Agency (EMA) and Office for Human Research Protections can determine whether sponsors adequately protect participants.

How Do I Access Other Healthcare If I Join a Trial?

Participants typically continue usual healthcare while enrolled. Under our country’s laws, the study operator covers all research interventions, procedures and medicines regardless of public or private insurance. Requiring sponsors to cover healthcare challenges clinical research in our country. However, a regulatory amendment in 2014allows continued use of social insurance in public-interest studies approved by the Social Security Institution (SGK), upon researchers’ application.

Participation Conditions

Participation contributes to medical knowledge. Results may improve future care by explaining benefits and risks of therapeutic, preventive or diagnostic products and interventions.

Clinical trials underpin development and marketing of drugs, biological products and medical devices. Experimental safety and efficacy may not be fully known at the time. Some trials may offer direct medical benefit, others not. Many require additional protocol-based procedures, tests and assessments, explained in consent documents. Potential participants should discuss these with the research team and usual healthcare professional.

Questions to Ask

Anyone considering participation should learn as much as possible and feel comfortable asking about the study, procedures and costs. The following questions may help. Some answers appear in consent documents. Many questions apply specifically to trials, but some also to observational studies.

• What are you investigating?
• Why do researchers believe the intervention may work? Why might it not? Has it been tested before?
• What interventions might I receive?
• How do risks, adverse effects and benefits compare with my current treatment?
• Which tests and procedures are involved?
• How often must I visit the hospital or clinic?
• How long will the study last?
• Who pays for my participation?
• If I benefit, may I continue the intervention after the study ends?
• Will I receive the results?
• Who manages my medical care during participation?
• What are my options if my condition worsens?

Clinical Research in Our Country

Approving new trials and protecting participants’ rights, safety and well-being is the responsibility of authorized independent Ethics Committees .

Under Additional Article 10 of the Basic Health Services Law, human research requires Ministry of Health or affiliated institution approval and approval from the relevant Ethics Committee. Trials therefore undergo rigorous approval and evaluation.

Medicines developed to benefit humanity and treat disease take years from laboratory discovery through successful clinical testing to routine use.

New medicines reach use only after decades of research and substantial effort. Bureaucratic approvals after trials also take time. At the 2016 European Cancer Congress (ESMO 2016), an important topic was drug approval times.

Delayed approvals are a major problem. Because development usually involves United States trials, new drugs generally receive FDA approval first, then EMA approval, and usually approval in our country later still…

Dr. Jean Charles Soria of France stated that time from positive trials to routine use is growing, a major loss for patients because longer access delays leave more untreated. EMA approval may take approximately 6-8 months after FDA approval. Each country then reassesses the medicine. European patients ultimately access drugs available to American patients only 1 year later.

Clinical research is among the most valuable investments in a country’s health and future. Developed countries prioritize and fund it for good reason. The best way to address scarcity and access difficulties in our country is educating the public to overcome the prejudice that“experiments are being done on people”, directing eligible patients to trials or compassionate early access programs. This is a major step toward offering appropriate treatments simultaneously with the rest of the world.