4 Drug Development Strategies To Accelerate Commercialization

By Mark Bleackley, Ph.D., chief scientific officer, Incannex Healthcare

In the clinical trial universe, pushing the envelope has become an essential piece of the puzzle. Given the extenuated clinical trial timelines, every chance to shave off time, save money, and still obtain regulatory approval is one worth taking. Herein, discover four ways to accelerate your therapy’s path to commercialization.

1. Pursue An FDA Expedited Approval Pathway

Navigating the FDA's complex requirements for expedited approval pathways can be a challenge, especially for smaller biotech companies. As such, a clear understanding of these pathways and how to choose the right one for every drug candidate is crucial for success in today's pharmacological environment. The fact that 65% of the drugs approved by the FDA in 2023 received their approvals under one of the four expedited pathways illustrates just how important this understanding has become.

The FDA offers four different pathways for expedited review and approval of drugs that treat serious and life-threatening diseases with unmet medical needs. The agency is especially seeking treatments that are the first ones available for a particular condition or those that offer significant advantages over existing therapies.

Of course, all four of these pathways enable companies to develop their drugs more quickly and reach the commercialization stage much faster than is possible under the traditional development process. However, each pathway has its own specific advantages stemming from a combination of the data required, the indication focused on, and the strategy of where that drug candidate fits into the other available treatment options, if there are any.

The four pathways are:

• Breakthrough Therapy – This requires preliminary clinical data showing substantial improvement in clinically significant endpoints over available treatments; advantages include more frequent FDA meetings and communications with rolling reviews, intensive guidance on the development program, and involvement of senior FDA managers.
• Fast Track – This requires preliminary nonclinical, mechanistic, or clinical data demonstrating the potential to address an unmet medical need in a serious condition; advantages include more frequent meetings and communications with the FDA, rolling reviews, and eligibility for Accelerated Approval or Priority Review.
• Priority Review – This requires data contained in the final NDA submission showing significant improvement in safety or effectiveness, prevention, or diagnosis of a serious condition; the key advantage is FDA review of the new drug application within six months of submission.
• Accelerated Approval – This requires justification of an alternate endpoint using scientific support showing a meaningful advantage over available treatments and effects on a surrogate endpoint that's reasonably likely to predict clinical benefit or a clinical endpoint that can be measured earlier than irreversible mortality or morbidity; the key advantage is approval based on the surrogate endpoint or intermediate clinical endpoint.

Digging Deeper Into The Breakthrough Therapy Designation

Incannex's primary strategy is to target unmet medical needs, which makes all of its programs potentially eligible for one or more of the FDA's expedited commercialization pathways. In particular, we’ve targeted the Breakthrough Therapy designation, which requires preliminary clinical data. As a result, we and other companies planning to use this particular accelerated commercialization pathway need to plan ahead, starting in the very earliest stages of development. This planning includes considering the data requirements for the pathways when designing preclinical experiments and early-stage clinical trials.

As indicated above, one of the benefits of the Breakthrough Therapy pathway is constant guidance from the FDA throughout the development process. Frequent discussions with the regulator are especially important in today's drug-development environment, as the drugmaker can ensure it's addressing all the regulatory concerns and improve its likelihood of success. We have found FDA engagement to be particularly helpful with development of our treatment for sleep apnea, specifically around the selection of endpoints, refinement of the patient population, and duration of the treatment period. There are no other pharmacotherapies approved for that indication, so there is no roadmap for getting the drug approved.

While we’ve seen significant benefits from the Breakthrough Therapy pathway for other drugs, we've also been considering the Fast Track designation, Accelerated Approval pathway, and Priority Review. The consideration is ongoing and includes dialogue with the FDA on eligibility and appropriateness of each of the pathways as the development of drug products progress.

Choosing the right expedited program for a particular drug depends largely on the type of data in hand and the FDA's response to that data, as well as the stage of development. The expedited pathways function at different stages of drug development, so it is possible to utilize more than one of the pathways through the life cycle of a single drug.

However, the outcomes of the pathways are different, and so are the qualifying criteria, as outlined above. Additionally, the eligibility for the different pathways is assessed at different times in development, and the benefits occur at different stages of review. The Fast Track and Breakthrough Therapy pathways generally come earlier in development and facilitate increased engagement with the agency, while Accelerated Approval and Priority Review are focused on the approval process.

2. Seek Commercialization In International Markets

In addition to leveraging expedited pathways, another way to speed up commercialization of a treatment is by exploring other markets. For example, we've found that Australia can be an excellent place for biotechs and small pharma to set up not only their early-stage trials but also their late-stage trials and all the way through commercialization. Incannex is continually evaluating the potential opportunities that may exist in other markets.

While the U.S. is one of the biggest pharmaceutical markets, many people around the world have the conditions Incannex is targeting, including sleep apnea, arthritis, and generalized anxiety disorder. And so, it’s important to consider getting drugs approved in different markets.

Having been founded in Australia, Incannex considers that a primary market. We've certainly seen positive regulatory viewpoints there, particularly in terms of the change in scheduling of CBD. That change could allow some CBD-only products to be approved as over-the-counter treatments. Although it hasn't happened yet, Australia’s Therapeutic Goods Administration has opened up that pathway. The recent shift in attitudes toward psychedelics in Australia again opens up the conversation and the opportunity to start developing those drug products for the Australian market.

Incannex’s upcoming RePOSA study, a Phase 2/3 clinical trial assessing IHL-42X in patients with obstructive sleep apnea, will include sites in Europe. As a result, we've been considering not only what the European landscape looks like for clinical trials but also for drug approvals.

The clinical trial application processes differ between the U.S. and Europe, and different documentation is required. Advantages of conducting clinical trials in Europe include access to world-leading clinical researchers and facilities, expanded access to patients, and recent changes to streamline the application and review process via the CTR and CTIS.

The EMA has multiple facilitated review pathways that are similar to the expedited FDA pathways. The main difference in drug approvals is that with the U.S., there is one submission pathway. In the EU, there are four submission procedures that stem from the EMA covering multiple countries. The submission procedures range from central submissions to individual national authorities.

Of course, every market is different, but there is always a need to demonstrate the safety and efficacy of drug products, no matter where a company might go. However, a critical difference from one market to another is the type and amount of data they require, which can vary widely.

3. Tap Into Drug Products With Established Scientific Data

One other Incannex strategy is to combine cannabinoids with generic medications that already have well-established safety data. This strategy greatly reduces the company's burden, with respect to both time and money, as well as risk in generating its own safety data. Additionally, understanding how regulators in different markets view that data is an important part of assessing the opportunities for commercialization in global markets.

In addition to using generic medications that have well-established safety and effectiveness data, Incannex uses cannabinoids that have been approved for other indications. Combining two drug products with established safety profiles can reduce the amount of time spent on preclinical studies and clinical trials — also saving money in the process. This strategy allows Incannex to pursue approval via the FDA505(b)2 pathway, in which the FDA relies on data not developed by the applicant during review and approval of an NDA.

As a result, Incannex has taken its IHL-42X, IHL-675A and PsiGAD drug products from concept to the Phase 2 or Phase 2/3 clinical trial stage in under four years. There is considerable time savings compared to new molecular entities, which often take five years or longer to go from concept to Phase 1 clinical trials.

4. Develop Solid Patent Strategies

Another critical piece of any successful biotech firm's commercialization strategy is establishing robust patent strategies for each drug product. For example, the early news around Incannex's patents and IP portfolio focused on the patents that covered the drug combinations for the indications that we're working on. 

However, Incannex also has been looking to expand that patent portfolio by filing the same patents not only in the U.S. but also in Europe, Australia, and other countries. The company also has filed patents on other aspects of the drug products, including the proprietary formulations. This robust strategy ensures the necessary protections are in place to secure the exclusivity needed when commercialization begins.

Final Thoughts

Ultimately, biotech companies push the envelope by using unconventional clinical trial designs and strategies that have greater chances of accelerating the commercialization of their products and improving patients' quality of life in the process.

In summary, here's a brief list of all the strategies that can help biotechs accelerate the commercialization of their products:

• Focus on unmet medical needs.
• In the U.S., utilize one of the FDA's four expedited pathways.
• Work as closely with the FDA as possible, utilizing all available FDA resources.
• Adopt a robust patent strategy that crosses international borders.
• Seek additional opportunities outside the primary target market, possibly by running parallel clinical trials in those other markets or engaging with the relevant regulatory authorities.
• Tap into one or more drugs with established safety and efficacy data, if possible.
• Gather all the scientific data required for the selected commercialization pathway.

While accelerating the commercialization process is good for the company, it also serves the patients. Biotech companies with promising treatments for indications with a substantial patient population that is not receiving adequate treatment should do everything possible to bring those treatments to market, allowing patients to access the product with the potential to increase their quality of life.

About The Author:

Mark Bleackley, Ph.D., is the chief scientific officer at Incannex Healthcare Inc. He oversees all drug development activities at the company from proof-of-concept through to clinical trials. The R&D team at Incannex, under Bleackley’s guidance, works across formulation development, manufacturing, regulatory, and clinical operations to bring the company’s drug products closer toward commercialization. Bleackley has a Ph.D. in genetics from the University of British Columbia, Vancouver, Canada, and completed postdoctoral training at La Trobe University, Melbourne, Australia. He has experience working in commercial drug development across a range of indications and stages from drug identification and mechanism of action through to clinical trials.