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Precision medicine

Early development programs face many challenges, and portfolio valuation of a pipeline in which personalized medicines based on technologies such as cell, tissue, and gene therapies are under consideration adds even further potential layers of complexity. “Precision” or “personalized” medicine seeks to provide a bespoke therapy for the right patient – with the right treatment at the right time – by adjusting for the nuances within an individual’s genetic profile, health records, and lifestyle.

One particularly intriguing application of personalized medicine is the prospect of treating cancer, which is perhaps uniquely targetable by a precision approach because its detection is so often entwined with timely individualized screenings. The clear trend is towards an increase in the number of therapeutic products that have received regulatory approval along with a companion diagnostic (CDx) test. This interconnectedness, however, raises a number of possible challenges when it comes to regulatory approval of precision oncology therapies.

For example, a novel cancer therapy may require the assessment of one or more biomarkers to identify patients who have an underlying genetic defect and/or are more likely to respond to treatment. Especially where biomarker identification is essential to the safe and effective use of the therapeutic product, it is important for a prospective product developer to consider the impacts of having what becomes in practice a multi-pronged regulatory approach. CDx tests are regulated by the U.S. Food and Drug Administration (FDA) often as an in vitro diagnostic test (IVD) under FDA’s medical device framework. Therefore, a prospective developer or investor needs to consider this additional layer of regulatory approval to ensure that their therapies can ultimately reach patients as intended. No access to a CDx that FDA has deemed necessary for safe or effective use of a therapy means no access to the therapy itself, even if the therapy is otherwise FDA approved.

While many companies with early development programs have clinicians and other scientists involved in developing their early clinical trials, it is also important to think a few steps ahead and consider the regulatory approval and product labeling impacts of these early decisions. Waiting too long, or learning late in the process that an additional test or labeling requirement could mean further interactions with FDA and potentially significant further investment and resources, and significantly slow product rollout and increase time to market. In short, a successful value based product development strategy requires early, meaningful engagement with a regulatory professional to best guide your approach to FDA approval and commercialization strategies.

Bumps on the Fast Track

There is also a recent trend towards an increase in FDA approval via programs having accelerated clinical development timelines. While not limited to any particular use or targeted class, personalized medicine oncology therapies intended to address unmet medical needs may often avail of eligibility in programs such as Breakthrough Therapy (BT), Fast Track (FT), and Regenerative Medicine Advance Therapy (RMAT). Moreover, while most currently FDA-approved gene therapies are chimeric antigen receptor (CAR) T-cell based oncology therapies, the challenges faced in the approval routes of these therapies can provide guiding principles for early development programs across advanced therapies.

For example, marketing applications for products in accelerated programs still need to meet FDA’s exacting approval standards, including compliance with current good manufacturing practice (CGMP) requirements. Products with accelerated clinical development activities may face challenges in aligning expedited Chemistry, Manufacturing, and Controls (CMC) development activities. As a result of these accelerated review timeframes, CMC and CGMP issues are often rate-limiting factors that have resulted in Complete Response Letters and prevented FDA from approving drugs that treat cancer or other serious diseases and fulfill unmet medical needs.

These challenges have been particularly acute for complex biological products. While the Breakthrough and RMAT programs were intended to address these challenges, FDA and industry have recognized that additional interactions with, and assistance from, FDA are often necessary to accelerate resolution of CMC and CGMP issues earlier in the context of expedited program review cycles. Accordingly, starting in April 2023, FDA will be implementing a CMC Development and Readiness Pilot (CDRP) program to facilitate CMC readiness for selected Center for Biologics Evaluation and Research (CBER)- and Center for Drug Evaluation and Research (CDER)-regulated products with accelerated clinical development timelines. Under the CDRP, FDA will provide product-specific CMC advice early in product development so that the clinical benefits of earlier patient access to these products can be realized.

A related bottleneck for complex therapies is the need for high-quality manufacturing, even in the context of an early development program. Too often, products originating from a start-up or in-licensed from an academic institution face major challenges in scale-up or otherwise transferring materials beyond their initial lab- or pre-clinical scale usage demands. These issues can often be addressed, or even avoided, by focusing on scalability at an early stage. For example, developers can prioritize standardizing protocols, preparing for technology transfer to a contract manufacturer in keeping with CGMP compliance, as well as exploring advanced manufacturing technologies such as continuous processes at an early stage. This type of preparation is key to avoiding surprises during late stage development or that may come up in the FDA review process and cause delays. Just as waiting too far into a development program to properly address the complexities of access to a CDx can result in regulatory delays, so too can failure to have a clear path forward for compliant manufacturing processes at a commercial scale. 

The cutting-edge therapies of innovative early development programs require an integrated analysis of science, data, and the law. Companies and investors that neglect these early steps risk delays in approval and corresponding delays in commercialization. Proactive planning for the product development and approval process of an early development program along with a regulatory specialist can help to optimize limited time and resources by developing a strategic approach to regulatory approvals and help ensure these innovative medicines are made available to patients and their loved ones.

Schedule some time with Lowell while at the Biotech Showcase (click here) and let him talk you through any high level factors you might want to consider.

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Authored by Lowell Zeta.