FDA Breakthrough Therapy Designation: How Does It Affect Medication Development?
Key takeaways:
The FDA is able to grant a breakthrough therapy designation for medications that treat serious medical conditions. These medications must offer advantages over existing treatments.
The designation allows pharmaceutical companies to speed up the development process. They also offer extra guidance and support.
A breakthrough therapy designation allows medications to be available to the public quicker.
The FDA’s Center for Drug Evaluation and Research (CDER) evaluates new medications before they become available to the public. This ensures that safe and effective medications are available for consumers.
When rare or serious medical conditions have few or no treatment options, the FDA can incentivize the development of new treatments. And when promising treatments are identified, there are ways to help make them available faster. One way of doing this is known as the breakthrough therapy designation.
In this article, we’ll review how breakthrough therapy designations help speed up medication development.
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What is the breakthrough therapy designation?
The FDA has different ways to help speed up the development of certain medications. These can be helpful for medications that treat rare or serious conditions. These FDA processes are called drug development designations.
Breakthrough therapy is an example of a drug development designation. The FDA grants breakthrough therapy to medications that treat rare or serious conditions. A medication qualifies as a breakthrough therapy if it offers new benefits over existing treatments.
For example, they may work better than available medications. And they must have meaningful effects for a condition.
Preliminary evidence should show the following:
The treatment met its surrogate endpoint(s) — markers that can predict a benefit.
The treatment had an effect on additional metrics, showing it would offer benefits for the condition it's treating.
The treatment is as effective as existing treatments but safer. For example, a medication has a lower risk of serious side effects than other medications.
A pharmaceutical manufacturer can request a breakthrough therapy designation during early clinical trials. The FDA can also suggest it if they think a treatment would be a good candidate for the program.
Examples of medications and devices that have received breakthrough therapy designation
From January to September 2021, 23 breakthrough therapy medications were FDA-approved. Many of them treat serious conditions, such as cancer.
Below are some notable examples of approved breakthrough therapy medications:
Ukoniq (umbralisib). This is an oral medication that treats forms of lymphoma (a type of cancer).
Lumakras (sotorasib). This is an oral medication that treats non-small cell lung cancer.
Keytruda (pembrolizumab). This is an intravenous medication that treats different types of cancer. It’s a breakthrough therapy for treating high-risk, early-stage triple negative breast cancer.
Farxiga (dapagliflozin). This is an oral medication that treats Type 2 diabetes. In 2021, the FDA approved Farxiga to treat chronic kidney disease as well. The medication received the designation for this use.
Livmarli (maralixbat). This oral medication treats itching caused by Alagille syndrome, a rare genetic condition. It’s the only medication available for this problem.
Does the breakthrough therapy designation speed up the FDA approval process?
Not directly. The breakthrough therapy designation helps speed up the medication’s research and development. But this means the FDA could review it sooner than it would have without the designation.
Breakthrough therapy provides manufacturers various benefits. These benefits may include:
More frequent meetings with the FDA to review the study’s data.
More frequent written communication with the FDA.
Eligibility for other drug development designations. These don’t always apply to all medications.
Rolling review. This means companies can provide research information to the FDA as it becomes available. Usually, companies only submit information to the FDA after finishing all clinical trials.
Extra FDA guidance and help from experienced FDA members.
These features can help manufacturers develop and study treatments as efficiently as possible. But in some cases, breakthrough therapies may be eligible for accelerated approval. This does directly speed up the approval process.
How does accelerated approval work?
FDA approval of certain medications can be sped up directly too. The accelerated approval program is one way to do this. It can be an important avenue if a medication treats a serious or life-threatening condition. Often, these medical conditions have limited or no treatment options.
Accelerated approval allows medications to be approved based on predicted benefit. This is through a surrogate endpoint. In other words, a clinical benefit hasn’t been confirmed before approval. But additional studies are required after it’s on the market to confirm a clinical benefit.
Sometimes, breakthrough therapy can be combined with the accelerated approval program. An example of this combination is the Keytruda approval listed above. When the FDA grants both of these, the development and approval processes can move even faster.
Does a breakthrough therapy designation always lead to FDA approval?
No. Earning the designation means the FDA provides guidance to help speed up the medication’s development. But this doesn’t mean that the medication will be approved.
The FDA may also deny, withdraw, or cancel a breakthrough therapy designation. This happens when medications don’t show meaningful benefit, as described earlier.
For example, in 2020 out of 125 breakthrough therapy requests, only 58 were granted. The FDA denied 53 requests and withdrew 14.
What are other examples of development designations?
Including breakthrough therapy, there are three types of development designations. A new medication may receive more than one designation based on their benefits. They can also be combined with the accelerated approval program.
Fast track
Fast track helps new medications that treat serious conditions to become available quicker. This designation helps speed up both medication development and FDA review. Fast track is often used for treatments that are the first of their kind, such as a new class of medications.
Medications must meet certain criteria to receive fast track designation. For example, they’re the first medication to be able to treat a health condition. Fast track can also be used if a treatment is going to address a public health emergency.
Priority review
Priority review shortens the amount of time it takes for the FDA to review a new medication. This designation doesn’t affect the length of time spent developing the medication. Priority review allows the FDA to decide on medication approval sooner than usual.
The FDA grants priority review when medications offer significant improvement over other treatments. For instance, the medication is effective for a new group of people (e.g., a new age group). A priority review means the FDA should decide on approval within 6 months. This is faster than the traditional timeframe of 10 months.
The bottom line
The FDA’s breakthrough therapy designation allows for faster medication development. These medications are usually for serious medical conditions. Breakthrough therapy medications must provide more benefits compared to available medications.
A breakthrough therapy designation doesn’t mean that a medication will be FDA-approved. But it can help make medications available to the public faster.
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References
Food and Drug Administration. (2014). Guidance for industry expedited programs for serious conditions – drugs and biologics.
Food and Drug Administration. (2017). Frequently asked questions about the FDA drug approval process.
Food and Drug Administration. (2018). Accelerated approval.
Food and Drug Administration. (2018). Breakthrough therapy.
Food and Drug Administration. (2018). Fact Sheet: Breakthrough Therapies.
Food and Drug Administration. (2018). Fast track.
Food and Drug Administration. (2018). Priority review.
Food and Drug Administration. (2019). Development & approval process.
Food and Drug Administration. (2020). CDER breakthrough therapy designation requests received by fiscal year.
Food and Drug Administration. (2020). Center for drug evaluation and research | CDER.
Food and Drug Administration. (2021). CY 2021 CDER breakthrough therapy calendar year approvals.
Food and Drug Administration. (2021). FDA approves pembrolizumab for high-risk early-stage triple-negative breast cancer.
Food and Drug Administration. (2021). Frequently asked questions: Breakthrough therapy.
Kepplinger E. E. (2015). FDA's expedited approval mechanisms for new drug products. Biotechnology law report.
Mirum Pharmaceuticals, Inc. (2021). U.S. FDA approves LIVMARLI (maralixibat) as the first and only approved medication for the treatment of cholestatic pruritus in patients with Alagille Syndrome one year of age and older.
National Cancer Institute. (2021). Definition of endpoint.
National Cancer Institute. (2021). Lymphoma — patient version.
National Organization for Rare Disorders. (2021). Alagille syndrome.
Yao, J. C., et al. (2013). Accelerated approval and breakthrough therapy designation: oncology drug development on speed? Clinical Cancer Research.
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