Developing A Treatment

New therapies spend a lot of time being tested in labs and animals in preclinical studies before making it to patients. By the time the therapy enters human clinical trials, it is better understood and ready for further development. Learn more information about the process of developing a treatment.


FDA Efforts

Between preclinical study and clinical trials, the process can take many years to conclude. Clinical trials alone sometimes can take eight years or more. There are many variables that go into that duration including study planning, authorization to run a trial, ethics review, funding, research materials such as patient information, consent forms and monitoring systems. However, the U.S. Food and Drug Administration (FDA) is putting an effort into various expedited pathways to accelerate the process while still maintaining safety.

Sometimes the typically described three phases of a clinical trial are combined to make the evaluation process more efficient. For serious and rare diseases where there is a clear unmet medical need and very few patients, the treatment may be made available more quickly.

It’s worth noting that there are challenges associated with streamlining the clinical trial process. Researchers need to ensure that they avoid exposing multiple patients to suboptimal or toxic doses. Or, they need enough time to properly interpret the outcomes of a trial. It all goes back to making sure that the treatment is well understood, safe and effective enough to be approved for market.

Speeding up the availability of drugs that treat serious diseases is in everyone’s interest. That’s why the FDA has created five different approaches to accelerating the development of treatment, they're called "expidited pathways." Some of these approaches can be combined to make the development of treatment even more efficient. However, the names of these different approaches all sound, well, fast. So what is the difference between them?

Expedited Pathways

Accelerated Approval

Accelerated Approval helps drugs for serious conditions with unmet medical needs get approved. It does this based on a surrogate endpoint. A surrogate endpoint means a result that is thought to predict a clinical benefit, which is different than the outcomes typically required to show a therapy works. Some true clinical benefits of a therapy can take years to find. A measure such as the surrogate endpoint that can reasonably predict a valuable benefit—that is also safe and well-controlled—can be accelerated for approval.

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Breakthrough Therapy

Breakthrough Therapy is designed to accelerate the development and review of drugs that demonstrate a substantial improvement over therapies that already exist. It also includes intensive and early FDA feedback on efficient drug development. Those are key differences from the Fast Track process. Developers of a therapy request this designation at the time of its Investigational New Drug application (IND) before starting clinical trials.

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Fast Track

Fast Track designation speeds up the review of drugs that treat serious conditions and fill unmet medical needs. In other words, conditions of which existing treatment or diagnosis is not adequate. Fast Track allows rolling review, which means that a drug company can submit completed sections of its New Drug Application (NDA) accuracy check for review by FDA, rather than waiting until every section of the application is completed before it can be submitted and reviewed.

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RMAT Designation

RMAT Designation (regenerative medicine advanced therapy) is available for drugs that are intended to treat, modify, reverse or cure a serious condition. Preliminary clinical evidence also needs to indicate that the drug has the potential to address unmet medical needs for a disease or condition.

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Priority Review

Priority Review is a designation that means after clinical trials, the FDA’s goal is to decide whether to approve the biologics license application (BLA) within six months. Priority review is given to applications for drugs that—if approved—would offer significant improvements in the safety or effectiveness of treatment or prevention for certain diseases.

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Not all research transpires in the United States, so there are global approval bodies similar to the FDA. National governments are responsible for establishing strong national medicines regulatory authorities, also known as MRAs. You may also see the phases of clinical trial referred to as early, late and pivotal stages of development.

Patient Access

Once a treatment successfully passes through clinical trials and is approved by the FDA, then what happens? How can patients access the treatments? There were three gene therapies approved in 2017, so we’ll use those as our starting point - Luxturna, Yescarta and Kymriah. These therapies are offered at a limited number of sites, currently only at specialized academic medical centers.

Although patients can reach out directly to a treatment center that offers the therapy, it’s important to inform their primary medical practitioner of the decision. Most times a clinician that knows the individual best will help in determining if the treatment is applicable and then may set up the referral. This primary clinician can also be involved in post-procedure care and answering questions along the way.

The currently available gene therapies are covered by many health insurance options, including Medicare. Interested patients should contact their health insurance provider to determine its coverage, as well as what to expect as far as out-of-pocket costs. Manufacturers of the gene therapies often offer patient support services to assist in navigating the process.

One of the most important components to moving along research and development of these therapies are the many volunteers that participate in clinical trials every year. Thanks to the volunteers, more people are living longer, healthier lives. Participating in clinical trials is a way to receive an investigational treatment at no cost, while benefiting the medical community, and others who have the same disease or condition. According to a study from CISCRP, 94 percent of people who have participated in a clinical trial of some type would be willing to participate again or would recommend others to participate.

Finding a Clinical Trial

Patients can access gene or cell therapy treatment by participating in a clinical trial. Not only can you potentially receive treatment for a disease, you can also help the greater good by assisting researchers in their studies.

  • Talk to your Doctor: even if you find a clinical trial on your own, it is still important to speak with your doctor about it.
  • Visit ClinicalTrials.gov: this clinical trial navigation service is a national database of treatment trials from different sponsors.
  • Sign Up for a Patient Registry: patient registries are a collection of information about patients who share a condition or experience. Their goal is to help medical researchers better understand how diseases develop and progress over time. Some registries invite people to sign up to be contacted about participating in clinical research. Here’s a helpful list of patient registries from the National Institutes of Health (NIH). 
  • Patient Advocacy Organizations: these organizations are hard at work to support rare genetic diseases and can be a great way to get involved and advocate for a disease. It is also a valuable source for emotional support, advice, and to learn others’ experiences with clinical trials.

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Different Approaches

Gene therapy, cell therapy and gene editing are fields with a similar goal: To treat disease by changing our bodies at a microscopic level. What do these approaches have in common, and what makes them different?

Gene Therapy Basics

Gene therapy has been studied for more than 40 years and can help stop or slow the effects of disease on the most basic level of the human body—our genes. And to understand how it works, we’ll start at the basics.

2019
22nd Annual Meeting
April 29 – May 2 | Washington D.C.
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