From Research to Reality: The 5 Steps in Drug Development

Drug Development2

It takes a long time to develop a new drug. Not to mention it can be very expensive. But why does it take so long and cost so much? In this article, we will be going through the five key steps in drug development to shed some light on this process.

Drug Development

Step 1: Discovery and Development

Before a treatment can be created, researchers need to understand how a disorder impacts the body. This step is sometimes called ‘basic research’ since it focuses on asking basic questions. In the case of Ataxia, some of the questions asked include:

  • What causes this type of Ataxia to happen?
  • What kinds of symptoms occur?
  • What does this type of Ataxia do inside people’s cells?

Once researchers have an idea of how an Ataxia type impacts the body, they can begin researching how to fix things. This discovery research takes trial and error, with thousands of potential treatments being tested. Most of this work is done using donated human tissue, cell lines, or animal models. Once they identify a potential treatment that has a measurable effect, researchers will conduct further tests to better understand how the treatment works. This step is called development.

Step 2: Preclinical Research

After several potential treatment options are identified through discovery and development, preclinical research will begin. This step is focused on safety. Researchers must check that these treatment options are safe for people to use.

Some treatments may be very effective when tested in a petri dish, but toxic when used in larger quantities. Researchers use various laboratory tools and animal models to identify unsafe or ineffective treatment options. They will also determine how long treatments stay in the human body. This is important information. It will help the researchers determine what dose of treatment to use in future tests.

On average, only 5 out of 5,000 treatments that enter pre-clinical testing move on to the next step of research.

Step 3: Clinical Research

After a treatment is shown to be safe through preclinical research, the next step is clinical research. This step involves human volunteers taking potential treatment. There are three phases of clinical research.

In Phase 1, the potential treatment is tested in a small number of healthy volunteers. This is yet another step to confirm the safety of the potential treatment. If the drug is safe, with minimal side effects, it will move to Phase 2.

In Phase 2, the potential treatment is tested in a small number of volunteers with Ataxia. This stage examines safety yet again, as well as monitors any potential side effects of the treatment.

Since Phase 2 is the first trial stage where people with Ataxia are taking the potential treatment, the researchers will also test how effective the treatment is. Different treatments have different goals, so what they measure to show effectiveness can vary.

Common measurements will include assessing balance and coordination. The researchers will monitor the increase or decrease of other Ataxia symptoms. Usually, researchers will test multiple doses of a potential treatment. Researchers do this to identify the dose with the most benefits and fewest side effects. If a treatment proves safe, with minimal side effects, and effectively reduces Ataxia symptoms, it advances to Phase 3.

In Phase 3, testing enlists a larger number of volunteers with Ataxia. The goal of Phase 3 is to confirm the results of the Phase 2 trial in a larger group of people. Researchers will again assess the safety and effectiveness of the potential treatment.

Additionally, researchers examine if the potential treatment is better than current treatment options if they exist. Many kinds of Ataxia lack approved treatment options for comparison with new treatments.

Step 4: Regulatory Review

If a potential treatment is safe and effective, it will make it through all three phases of clinical research. Then comes the next step, regulatory review. Depending on where you are in the world, different agencies are responsible for reviewing potential treatments:

Although each regulatory agency has slightly different procedures for approving new treatments, they all have the same goal. They want to confirm that they properly executed Steps 1-3 in the drug development process. To do this, the regulators will go through all the data provided by the developers of a potential treatment.  Their job is to confirm a drug is both safe and effective.

Step 5: Post-Market Safety Monitoring

If a regulatory agency approves a drug, doctors can prescribe it to patients. But the steps of drug development don’t stop there! Now that the drug is on the market, it enters Post-Market Safety Monitoring.

In this step, the regulatory agency will monitor the safety of approved treatments. They will keep track of the types and frequency of side effects associated with the treatment. If there is an unusual number of people having major side effects, the regulatory agency will investigate why this is happening. If necessary, the regulatory agency will remove the treatment from the market.

Want to Learn More About Drug Development?

You can learn more about the drug development process for rare diseases like ataxia through our PrepRARE Clinical Readiness Education series. Check out this talk from David Spotts on Drug Development from a Biotech perspective.

Rare Disease Drug Development: What Patients and Advocates Need to Know online course available through the National Organization for Rare Disorders (NORD). One of the case studies used in this course is the approval of SKYCLARYS™ (omaveloxolone) for Friedreich Ataxia and the advocacy work done by the Friedreich Ataxia Research Alliance.

We would like to thank PrepRare and NAF for sharing this educational article with us.  As always, we welcome your thoughts and value your feedback.  Let us know what you think by Dropping Us a Line or commenting below.

Join Our Community today and share your experience with Ataxia by getting in touch with us. A place where we empower you to build a healthy lifestyle and raise overdue Ataxia awareness.  Experience transformative storytelling and share your story to inspire positive change.


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