Planning for CBER and EMA success with cell and gene therapy

Cori Gorman, Ph.D.

The fields of cell and gene therapy are enormously promising while at the same time difficult to navigate – both from a scientific and regulatory perspective. Developers need to plan for success from the outset, understand regulatory authority expectations and learn how to work with various agencies to facilitate development.

In both the US and the EU, the objective for the regulators is the same: to help innovative companies bring life-saving and life-saving therapies to patients. Regulatory authorities in both regions  strive to work with companies to make that happen.

Meeting with the agencies

Regardless of the market, companies are well-advised to seek advice from the health authorities early in the development process.

The Center for Biologics Evaluation and Research (CBER) made clear that it is eager to work with innovative companies to facilitate the development of their therapies.

The agency has put in place a number of initiatives to help to expedite approval. As an example, developers aren’t required to have clinical data to have a discussion with CBER about fast tracking a drug, since the agency will accept good animal data for a conversation.

For companies pursuing rare disease indications CBER director, Dr. Peter Marks, has noted that the agency recognizes the difficulty with patient recruitment and will work with developers to find alternative ways to expedite the process, as long as they present good clinical data.

In Europe, there are also a number of initiatives aimed at supporting innovation, including incentives and support for micro, small and medium-sized enterprises (SMEs). There is also good evidence that companies that do meet with the European Medicines Agency for scientific advice, and that follow that advice, have a high success rate during the approval process.

One well known way to expedite approval in Europe is the PRIME scheme, which gives developers of priority medicines enhanced interaction and dialogue. However, companies must present early clinical data. On the other hand, the UK’s Innovative Licensing and Access Pathway (ILAP) scheme for innovative medicines does not require developers to present clinical data.

Companies might also consider speaking with one of the national committees in Europe rather than the EMA, which often provide very helpful advice on starting clinical trials in specific countries. As an example, one company working in the field of rare diseases chose to meet with the Swedish National Committee, which provided some valuable information on starting first-in-human trials in Sweden.

Driving and enabling innovation

An exciting and progressive development from CBER is new guidance that allows developers to test multiple versions of their cell and gene therapy under an umbrella IND that would enable the developer to test multiple iterations of a product, either as part of one trial or a series of trials. In its guidance, released in November 2022, titled Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial, FDA has provided “recommendations for studies that evaluate multiple versions of a cellular or gene therapy product, including how to organize and structure the INDs, submit new information, and report adverse events”.

This approach can be especially helpful for developers of cell and gene therapy products, where a sponsor may not be sure which vector to use to administer the product, for example, or which exact transgene to use.

To further innovation, CBER has also redefined sameness for cell and gene therapy to allow for small differences so more than one company can move forward with the same indication. Under the guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, FDA notes that two gene therapy products that express different transgenes or use different vectors from different viral classes, even if they express the same transgene, will be regarded as different products.

These and other regulatory initiatives will be key to supporting the development of ground-breaking cell and gene therapies. For innovator companies, collaborating with the regulators and staying on top of regulatory developments that support and enable development will be key to successful outcomes.

Are you developing cell or gene therapy products? Are you struggling with scientific or regulatory hurdles? What challenges have you faced? Or what exciting breakthroughs have you been able to achieve, perhaps supported by advice from the health authorities? We would like to hear from you.

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