When the Food and Drug Administration announced its approval of Atripla'the first single-tablet triple-drug combination therapy'a year ago it ushered in a new era of simplified regimens. The efforts of thousands are to thank for helping to push the evolution of HIV care toward this important milestone. A vital factor in this fight has been HIVers who have participated in clinical trials since the 1980s and helped to bring the different classes of antiretrovirals to reality.
What Is a Clinical Trial?
Before gaining approval from the FDA, any medication, lab test, or medical procedure used to treat HIV'or any other disease'must undergo rigorous testing to ensure both its safety and efficacy. Human drug trials are divided into four phases.
Phase I, which can last up to a few months, is used to establish preliminary safety data, how the drug is metabolized, and how well it targets HIV. Lasting roughly a year, Phase II better establishes the safety and efficacy of the drug and also studies potential side effects. Phase III weighs the risks and benefits of taking the drug and further studies side effects, safety, and efficacy. If this phase is successful, the drug goes before the FDA to seek approval. This step can take two to three years. Phase IV allows for ongoing research regarding an approved drug's safety and efficacy.
There is also an expanded-access phase. During Phase III, if the drug company deems it safe to do so, the FDA allows patients outside the study to take the drug before it is approved.
There are roughly 30 new antiretrovirals currently in clinical trials, with many more in the preclinical pipeline. If you're interested in getting involved, there are pros and cons to consider.
What Is the Benefit?Finding a potentially effective drug that isn't available yet. This is a particular draw for multidrug-resistant patients who are seeking new drug classes. One such person is activist Nelson Vergel. Last year the 23-year veteran of HIV disease entered a Phase III trial of Merck's MK-518, an integrase inhibitor, along with Prezista, a protease inhibitor available in expanded access that has since been approved by the FDA. He achieved an undetectable viral load for the first time, and his CD4 count jumped from 180 to 440. 'I am so excited,' he says, almost gushing.
Keeping an effective therapy in your regimen. If the investigational drug proves to be a useful therapy for you, there is a strong possibility you will be able to remain on it indefinitely. If not, the research team will help you find a new drug regimen. 'We don't let anyone float in the wind,' says Michelle Simek, community education coordinator for the AIDS Research Alliance. 'We will definitely help someone find additional medical care.'
Getting access to free medical care and medications. In addition to prescribing meds at no cost, the research team will provide many basic checkup services, such as monitoring blood pressure, weight, CD4 count, and viral load. If you have a primary care physician, it's like adding a layer of care. If you don't have one, the team will try to help set you up with one or even take on that role.
Getting access to top-of-the-line tests. Trials often provide regular resistance tests, which are expensive and covered only once a year by most insurers. Pharmacokinetic tests, which analyze how the body is processing meds, are also possible.
Receiving one-on-one care from experts. The research team is likely composed of HIV specialists, and they often have the luxury to spend focused time with you'in contrast to the hurried office visits typical of primary care docs.
Contributing to the greater good. By participating in a clinical trial, you are participating in the larger effort to improve HIV care for everyone. To provide the best possible safety data for different ethnicities, for example, researchers need diversity in their study populations.
Getting a financial incentive. Some studies pay the participants who complete them.
What Is the Downside?Facing unknown side effects and safety issues. There is a chance of experiencing unpleasant or even dangerous side effects from an investigational drug. Before consenting to participate, ask what kind of safety nets are in place in the event of adverse reactions.
Receiving an ineffective therapy. A drug might not work for you.
Taking on a strict schedule. You might have to spend a considerable amount of time visiting researchers' offices.
Potentially losing out financially. Ask what expenses'such as travel, parking, and child care'the study will reimburse. New Jerseyan Annette Lizzul paid her way to Boston once a week to participate in an ultimately unsuccessful stem cell research project.
Receiving a placebo. In placebo-controlled studies a percentage of participants will receive an inactive sugar pill instead of the drug that is under investigation. Studies should have a provision to move participants on to the active agent if their health begins to decline.
Ultimately, after weighing both the pros and the cons, many HIVers who are eager or desperate for new treatments find that they can benefit from participating in a clinical trial.