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New Treatments – How Much Longer?

After months - maybe even years - of feeling like a hamster on its exercise wheel chasing a solution to your child’s health issues, you are devastated to finally learn that your loved one has a rare disease that is so novel, a cure has yet to be found. To manage the disease and provide your child with a shred of quality of life, you have your child cycle through several different medications, some of which may alleviate a few of their symptoms, but almost none that alleviate all of the symptoms or even the major burden of the disease. Why is that the case? How difficult can it be to find that one medication that could be the ultimate remedy? What does it take to find a new medication to help these children? Why does it take so long for a drug to get approved?

I’m sure these are some of the questions that have been occupying your mind as you navigate the rare disease journey. I am hoping, through this blog, to answer these questions and many others you may have pertaining to the clinical trial process.

How difficult can it be to find new medications?

The clinical trial research path is long, expensive and arduous for drug developers, clinicians and patients. The estimated cost to develop a new medicine that gains marketing approval is $2.6 billion according to a study published by the Tufts Center for the Study of Drug Development (Journal of Health Economics, May 2019, Where does a drug development company get this type of funding to develop a new drug? If the company doesn’t have a product on the market (commonly referred to as a research and development company or R&D company), it is not generating revenues and therefore, has very limited financial resources to sustain its research. It is necessary for these R&D companies to rely on the investment community for funding to support their research. Such funding typically comes in waves and is usually aligned with achievement of milestones. Obtaining adequate and timely funding is essential for an R&D company to conduct clinical trials in an efficient manner. Without appropriate funding many R&D companies need to halt or delay their clinical trials.

Funding is only piece of the puzzle. The regimented process to advance an investigational drug through clinical development is highly regulated. As noted in Figure 1 below, it can take 12-15 years to move an investigational drug through clinical trial research to approval (the drug development and approval process).

With limited funding and multiple years necessary to execute trials, the process to find new medications is very difficult.

Why does it take so long for a new drug to go through the drug development process to approval?

First, a new, or investigational, drug must undergo rigorous testing in laboratory animals to identify toxicities. Basically, is it ‘safe.’ If the drug isn’t toxic, it can move to a Phase 1 clinical trial. In Phase 1, approximately 20-100 healthy volunteers (these are people who do not have the disease being studied) are given the investigational drug and studied over several months to identify a safe dose to advance. Once that safe dose is identified, the next step in the process is a Phase 2 trial. A Phase 2 trial can enroll several hundred patients who have the designated disease with the goal of determining if the drug has an effect on the disease and if there are any side-effects. Upon the successful completion of a phase 2 study, the company can then advance to phase 3, an adequate and well-controlled study with the goal of achieving a safe, well-tolerated and effective treatment of the disease with the investigational drug. A phase 3 study can enroll up to thousands of patients and is typically conducted as a placebo-controlled study. The use of placebo’s in clinical trials is covered in the next section below.

There are several hurdles at each stage of development that must be met before a drug can advance to the next phase. Any clinical trial phase can be elongated, enroll more patients or re-conducted until the desirable effects are achieved. The overall probability of clinical success is estimated to be less than 12%.The many steps to the drug development process are shown above in Figure 1 and clearly identified and defined on the FDA website. This entire approach involves hundreds of patients, takes many years, and is highly regulated by the U.S. FDA before any investigational drug can be brought to the market and made available to improve the health of the people in the United States.

What does it take to get a drug approved?

The outcome/data from the phase 3 study will serve as the basis of the new drug application (NDA) for commercial approval with the FDA. The FDA reviews the NDA to ensure the company provided adequate data on the drug’s effects and has shown that the benefits provided by the drug to the patients suffering from the designated disease outweigh the potential risks. If the NDA filing is complete and doesn’t require any additional or supplemental information and the manufacturing facility passes inspection, the drug could receive FDA approval for commercial use within 12 months from filing. Once a drug has been approved by the FDA, a company can make the drug available for commercial use to physicians and patients. Keep in mind, approval does not mean that the drug will become immediately commercially available. It may take a couple of months post-approval before you can gain access to the drug for your child.

Why do clinical trials contain placebo’s?

Imagine you are considering participating in a clinical trial. As you review the protocol for the study, you learn that your child will be randomized to receive either the investigational drug or a placebo (inactive drug). You pause and have second thoughts. Why would you enter a clinical trial and endure the regimented commitment if there were a chance that you wouldn’t receive the investigation drug? While this is a very common and logical question, the answer is quite simple – to advance the development of the drug with the goal of finding a cure or treatment for your child’s disease.

Placebo’s are a standard tool used in most clinical trials to assess the efficacy on treatment of the investigational drug. A placebo accounts for the effects from treatment that aren’t actually caused by the treatment (ie, expectation from the drug, attention from healthcare professionals, etc.). Recall in the earlier section that in order for the FDA to approve a drug, there needs to be adequate data on the drug’s effect. To achieve this, a company needs to show that the effect seen with their drug is greater than the effect seen with placebo. This information cannot be obtained from a single-arm clinical trial (one where there is no control or placebo to compare to the drug).

Most R&D companies recognize the importance of providing a potential treatment with their investigative drug to patients, and therefore, have designed creative studies which allow all study participants to receive the investigational drug at some point during the clinical trial process. While your child may not receive the investigational drug initially, if they persevere through the study, they may have the option to receive drug at a later point.

Let’s do this together!

With a basic understanding of the clinical trial process, your journey through clinical trials should be more informative and less stressful. Conducting clinical trials requires a great commitment by patients, caregivers, medical practitioners and R&D companies. To ensure the integrity and success of the trial, all stakeholders need to collaborate for one common goal – to identify new treatment options for patients in need. The R&D companies are committed to this cause, are you ready to do your part?

Lisa Caperelli is an accomplished senior executive in the life sciences industry with extensive demonstrated experience in the strategic planning and implementation of successful investor relations, corporate communications and patient advocacy programs. Lisa has successfully cultivated strategic partnerships with patient advocacy groups and key stakeholders based on her credibility, knowledge, reliability and responsiveness. She is a dynamic and energetic collaborator with exceptional leadership skills and an unwavering passion for patients.

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