DNA editing biosimilar CRISPR researchCRISPR (Clustered Regularly Inter-Spaced Palindromic Repeats), is a set of processes for correcting genetic damage or dealing with specific genetic disorders by editing parts of the DNA or RNA sequence. It’s at the cutting edge of healthcare R&D, making the establishment of proper regulations a significant challenge. A lack of proper or timely regulation could be the determining factor in which country becomes the worldwide leader and patent holder of this treatment methodology. CRISPR need only look to the recent regulatory history of biosimilars to learn the importance that defining regulation will play in its future.

Biosimilars are to biologics as generic drugs are to their original, patent-holding innovatives. Biologics (insulin for example) aren’t created by a chemical industrial process—they need to be grown. This fact results in different challenges when trying to reproduce that process. After a biologic comes off patent, a potential manufacturer won’t have access to cloned cells from the original process. So, whatever they produce is going to have a similar effect to the innovative treatment, but with a different core molecule; a copy but not an exact one.

There are noted concerns about whether these biosimilars can be directly interchangeable or easily substitutable for original biologics. The same question continues to be asked around the world: How will biosimilars be regulated and controlled?

Biosimilar Regulation

Being first to market with a drug presents a great value due to the years of exclusivity that a patent provides. That, in turn, significantly impacts who’s going to be the world leader in production.

The United States, for example, lagged behind worldwide competitors in developing clear guidelines and regulations for their biosimilars. The EU was the real driver and continues to have the most comprehensive technical regulations for biosimilars. The EU set the standard for how biosimilars enter the market and how they’re classified, approved, and prescribed. The end result is that European biosimilars are about 30% cheaper than what the U.S. has to offer.

China and Taiwan are the other big players in the biosimilar industry, and they too have been playing regulatory catch-up. They’ve loosely adopted a similar model to the EU, but they face some regulations that work against them. In most countries, biosimilars—like generics—are usually approved faster than the original, innovative drug. For China, both biologics and biosimilars are approved under the same schedule, so the approval process takes the same amount of time.

In Europe and the United States, the innovative drug doesn’t necessarily have to be approved in said country for an off-patent biosimilar to be produced. So the U.S. could create a biosimilar based on the innovative drug that is only approved in the EU. Conversely, in China, a biologic must be approved there first before a biosimilar can be created, further increasing the approval time.

Another looming issue is that nearly $100B worth of biologics will be going off patent in 2021, which will potentially result in a significant battle over biosimilar production. Is the U.S. market ready to take on that fight or will biosimilars continue to come from zones that already have a robust production system like the EU? This is an area where the sophistication and development of regulation will have a massive impact on the growth of an industry to the end market.

CRISPR Regulation

The same questions and concerns raised by biosimilars will need to be addressed with CRISPR. What makes CRISPR so attractive is that it’s economical compared to other methods of genetic editing and has been one of the most successful methods thus far for widespread therapeutic use in humans. The areas seeing the most immediate CRISPR use in the end market are cancer therapies, antibiotic resistance, and viral infections.

Like any new, cutting-edge technology the predicted applications are endless, which leads into a more controversial and contentious area: editing embryos, possibly in utero, to deal with genetic disorders. There hasn’t been much disagreement about treating ailments in living humans with CRISPR, but the idea of germline editing (changing the code of every cell in an embryo) poses a bevy of potential regulatory concerns.

Complicating matters is the fact that the Chinese have been pushing a number of firsts with CRISPR editing, including implementing germline editing in terminal patients and the successful germline edit of a viable human embryo this past summer.

When it comes to editing genes, no one entirely knows what the full outcome of CRISPR will be. The germline editing that was performed on the human embryos in China worked correctly in 16 out of 18 attempts to replace one specific defective gene. The other two embryos incurred additional edits that weren’t supposed to happen. In the editing process, one illness could potentially be traded for another.

There is a real ethical concern about moving too quickly into human trials. While the Chinese have demonstrated proof of concept to edit specific genetic disorders and inheritable diseases in the womb, there’s already speculation that “designer babies” won’t be far behind.

Who Will Win the Regulation Race?

While this certainly raises concern about China leading or winning the race, being the first to a trial treatment isn’t the same as securing a patented end product. Regulation will determine not only what parts of the world become drivers for this market but how and where these treatments are used and who’s going to be able to produce them. Like biosimilars, the regulations for approval and the patenting of intellectual property for CRISPR are still not fully established.

What CRISPR can learn from the journey of biosimilars through the regulatory process is to be proactive and ahead of the curve in terms of laying out not just a basic regulatory system, but technical systems for evaluation and recommendations to health care providers for their usage.

Regulation will not cause a halt in interest in CRISPR research or innovation, but it will affect the timeline and types of implementation. CRISPR may not be at the trial level for non-terminal patients yet, but if proper and specific regulations are laid out early, it can help guide and structure how it enters into the healthcare system. That’s going to be a crucial issue for the country with designs on being the next worldwide leader.

To discuss your next healthcare project contact John.Lorinskas@martecgroup.com.

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