My Insights Into the Regulatory World

Dan Leonard | March 13, 2014

It takes a great deal of knowledge and skill to be a truly successful patient advocate. With so much to master, one area that can sometimes get overlooked is the role of Regulatory Affairs (RA) within industry. It is important to understand what RA does and how the process works, because new therapies do not get approved without a long journey through the regulatory process. For patient advocates both in and out of industry, it’s also important to understand how RA professionals can be critical allies in making therapies available to patients. Having worked on the Regulatory Affairs end of biotech for many years, I thought I would share some insights I’ve gleaned over the years.

Broadly speaking, Regulatory Affairs is the department that exists within pharmaceutical and biotechnology companies that interacts and works with the regulatory authority within each government. Of course these regulatory authorities or agencies have control and oversight of the products companies are developing or manufacturing. Examples would be the Food and Drug Administration (FDA) in the U.S. and European Medicines Agency (EMA) in Europe.

So what does RA do?  It may be easier to start with what it doesn’t do. It doesn’t actually run pre-clinical experiments or generate data from manufacturing studies. But it does need to have an in-depth understanding of both. RA advises scientists to ensure they are following regulations. It deciphers and summarizes complex scientific data for submissions to authorities like the FDA. RA is responsible for transmitting vast amounts of data to FDA and similar agencies in other countries to review and approve, which can be an enormous undertaking.

All of this goes on for years, throughout the drug development process, and leads up to the big moment – the submission of a marketing application (in the U.S., a New Drug Application or NDA). In the old days, when applications were actually printed on paper, they were so massive that they actually needed to be driven from biotech or pharmaceutical company headquarters to the FDA in Bethesda, MD in an 18-wheel truck. Nowadays they are sent on CDs or electronically.

But the work of regulatory affairs doesn’t end there. In the U.S., for example, the FDA will have hundreds -- if not thousands -- of questions on all aspects of a NDA to ensure that the product is both safe and effective. This process can take years if there is an issue with the application or the FDA believes the data is not sufficient. Hopefully it ends with an approval, but some products will not withstand the rigor of this review and are not approved. The work of RA doesn’t stop even if a drug is approved. There are regular reports that need to be sent to regulatory authorities on any continuing clinical work; safety updates; and even seemingly insignificant changes to the manufacturing process need to be communicated for the life of the product.

Now, I have primarily been using the FDA as an example. Take this and multiply it by the world! Every developed country and most developing countries have their own regulatory agency, and their requirements and processes are unique. A new drug submission for the EU, will have different requirements than that of the US, or China, or Colombia, or Egypt or Australia. There have been efforts to “harmonize” requirements by the International Conference on Harmonization (ICH) but even for countries that have signed on, differences persist. For the EU and countries like Brazil or Japan, filing the new drug submission can happen in parallel with the US, but many other countries require an FDA or EU approval before they will even consider accepting an application. The result is often a “drug lag” which can be frustrating to patients who know there is a safe and effective therapy available, but not yet in their country.

However, patient advocates can have an impact in the regulatory world. When I was working on a team that was trying to get an enzyme replacement therapy approved in Japan, we were stymied by the Japanese regulatory authority’s slow progress. Local patient advocates worked with a popular Japanese pop band to urge the government to move faster. The product was approved in nine months, which was a record at the time.

It is often difficult for busy advocates to gain an in-depth understanding of regulatory affairs due to lack of time and resources. But organizations like the Regulatory Affairs Professional Society (RAPS) offer courses in the EU, US, and Japan to help advocates educate themselves.— Dan Leonard, Patient Advocacy

Patients can get involved in working with the U.S. FDA by joining the FDA Patient Representative Program. Advocates can educate themselves by going to an organization like the Regulatory Affairs Professional Society (RAPS) that has courses in EU, US, and Japanese regulations. I have even talked to amazing parent advocates who have basically learned Regulatory Affairs the hard way – learning on their own by authoring and submitting periodic reports to the FDA for the trials they are collaborating with researchers on.

By learning how the regulatory world works, you can be a more effective and powerful advocate. And as your influence grows, agencies around the world will listen to you.

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