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  • Date posted:04/09/2023
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With Federico Goodsaid

The FDA has launched a new pilot program, opening the door for some incredible opportunities. To better understand what this means for the Oncology space, Katy Barber spoke to Ariana Pharma’s SVP of Regulatory Affairs, Federico Goodsaid.

When it comes to Regulatory Affairs, Federico is a leading expert. Having worked in the FDA for nearly a decade, Federico built up a wealth of knowledge, which he then put to use in industry. Since then, he has remained consistently focused on the Regulatory Affairs sector, even acting as an Adjunct Professor since 2007.

Federico has also been a consultant since 2015, helping his clients solve various product issues within therapeutics and in vitro diagnostics.

What is the FDA’s New Pilot Program?

In Federico’s words, “The Centre for Excellence is trying to do something very simple; address a long-term issue concerning the regulatory pathway for in vitro diagnostics”.

Currently, labs have to be individually certified by CLIA, meaning specifically authorised equipment and processes must be used. In turn, these labs can produce Laboratory Developed Tests (LDTs), which are used to support therapeutic development. The process, however, is lengthy and difficult to regulate.

In this pilot, the FDA will request from drug manufacturers performance information for tests used to enrol patients into clinical trials supporting drug approval. The FDA will review these data and post to the FDA website minimum performance characteristics recommended for similar tests that may be used to select patients for treatment with the approved drug. CLIA laboratories may use this information to guide their development of LDTs to identify specific biomarkers for the selection of patients who will benefit from cancer treatments.

What Problems Does It Solve?

When Federico was at Toma Biosciences, his team developed a 130-gene set of next-generation sequencing tests, creating one multiple companion diagnostic result.

The overall idea was to develop a single set of reagents that could give you an answer for 130 different genes at once. Initially, the process was very successful and the results were promising…

Unfortunately, the machines used to submit the data did not have 510 K approval. In response, Federico’s team were told that they would need to transfer the detection of the variance in those 130 genes to a different platform that had received 510 K approval – a nearly impossible endeavour.

The new pilot scheme could eliminate this problem forever.

Instead, individual labs could use their own set of equipment to carry out experiments. Different labs could even perform similar experiments on completely different machines without issue.

As long as each lab keeps track of what equipment was used to reach which analytical validation and shares this with the FDA, they would be able to continue their research unimpeded.

What Does the Future Hold?

If this pilot program succeeds, it could mean great things for the wider R&D space. Previous schemes (which attempted to regulate LDTs) haven’t worked, and even had to be recalled.

But Federico is optimistic about this latest scheme.

It gives individual laboratories the freedom to research and develop as they see fit, as long as all the data is passed to the FDA for analysis.

It’ll Change the Diagnostic Landscape

There is one immediate, unintended consequence of the FDA’s new pilot scheme. It will become less profitable for big companion diagnostic businesses to produce companion diagnostic tests.

Currently, diagnostic companies have difficulty competing with laboratory-developed tests. For instance, the price of FDA-approved companion diagnostics remains a crucial challenge. With this new pilot scheme, developing companion diagnostics that through the normal regulatory approval pathway may become less frequent.

Of course, the new program has a range of pros and cons, making it impossible to fully predict how exactly the landscape will change.

A More Collaborative Industry

Ideally, the FDA’s new pilot program should result in greater business collaboration. With how the scheme is designed, new findings will be shared between all the partaking businesses.

In turn, this should reduce individual R&D costs, as the development of the assays can be shared between different laboratories. Since each company is working towards the same goal, this scheme should foster better collaboration between laboratories which typically compete against each other.

Hopefully, others working in the oncology diagnostics field see this as an excellent opportunity to reach patients faster, and benefit the Life Sciences industry as a whole.

Expanding Outside of Oncology

While the program is currently exclusive to Oncology, nothing would prevent its application in a wider diagnostic space.

For instance, Neurology is an area which companion diagnostics development has been painfully limited. Neurology is particularly vulnerable to the development of biomarkers supported by noisy, and often equivocal data. The fear of repeating over and over again the development of imperfect biomarkers has become a high hurdle in Neurology. Since these imperfect biomarkers would be used to select patient subpopulations, they could prevent an accurate validation of these biomarkers in neurology.

The FDA’s scheme would make the process both cheaper and easier, potentially influencing a drastic improvement in future developments — especially since the therapeutic company would absorb much of the initial development costs. Individual CLIA labs would then decide whether, how and when they would like to participate in the development of their own assays for specific companion diagnostic tests.

Potential Worldwide Adoption

Initially this pilot scheme will impact labs in the US. Of course, that doesn’t mean it will stay that way.

Similar schemes could be rolled out across other major territories, such as Europe. In fact, with the relative complexity of companion diagnostics regulatory review pathways in the European Union, a similar scheme may have an even greater impact in the EU on how labs get CDx products approved and the results of these tests into the hands of the patients who need them.

There will be many watchful eyes on how this pilot program progresses. Its success could revolutionise the delivery and penetration of targeted therapies and personalized medicine.

Connect With Federico

Want to stay on top of all the latest developments in the Therapeutic and Regulatory spaces? Make sure to connect with Federico and ask for his thoughts.

As a top mind with a wealth of knowledge and experience behind him, Federico can offer unique and in-depth insights into the latest Regulatory developments and trends.

If you want to develop your knowledge of this area, Federico is an invaluable addition to your network. You can reach him at Ariana Pharma (f.goodsaid@arianapharma.com), where he is a consultant for Ariana on the integration of biomarker identification and prioritization using Formal Concept Analysis (FCA) through KEM into regulatory strategy and submissions. Ariana identifies and prioritizes biomarkers in small (10-20) patient population studies for patient selection, pharmacodynamic and surrogate applications.