There is very often a them-and-us mentality when it comes to how biotech companies think about the regulators. New companies, in particular, tend to view what is said by the regulators as prescriptive and are afraid to speak to them or get the scientific advice that is available.
However, if they did take time to speak to the authorities early in the development process, they could learn what the regulatory difficulties with their planned dossier might be, rather than risk long delays to their marketing authorisation as they scramble to gather the necessary information later. The earlier companies speak to the regulators and get their advice, the better off they will be in terms of understanding weaknesses in the development strategy, gaps in information about the product, what data will be harder to source, and which aspects they should be making a priority.
For example, if a company has a first-in-class molecule and doesn’t yet know how to identify it, it’s possible they haven’t refined the crystallography. But it’s likely the regulatory authorities will have scientists who can offer advice or recommend an equivalent test that would provide the information needed. Reaching out to the regulators is an invaluable way for companies – particularly small innovator companies – to get the answers they need.
I have spoken to regulators who have gone so far as to provide a detailed template protocol to a company that didn’t know how best to design a study for their orphan drug, when they took a COMP advice procedure as part of the orphan drug incentive. That is a huge saving, since companies can spend thousands on KOL’s and medical writers for a protocol.
The fact is that the regulators want to help companies bring good products to market, especially orphan drugs. They have established programmes such as the SME (small and medium-sized enterprises) and Orphan Drug Designation to facilitate this and are there to guide companies through the regulatory process. They do not expect absolute agreement with the guidelines and regulations either and acknowledge and welcome the expertise of the company’s scientists. If companies feel a different approach is best for their product, they can debate it so long as they justify their argument with data and scientific evidence. Indeed, this is actively encouraged, especially for innovative products where no precedent is available in the guidelines.
Working with the regulators to bring a product to the market can be a wonderful, collaborative, process. It’s a shame to fear it and miss a golden opportunity to advance promising products.