How did you get started in your career in Regulatory Policy & Intelligence?
I got started at the FDA, and I’ve worked in both regulated industries and in government for over 20 years. I worked for the FDA, HHS, and within the US House of Representatives as an oversight investigator for FDA and HHS issues. I’ve also led the offices in Washington for three pharmaceutical companies, as well as one food & drug company. Currently, I’m on my second stint of consulting (I have been a consultant in the past), and I’ve worked for patient groups, academic medical centers, and large and small pharmaceutical and healthcare companies.
Why do you feel Regulatory Policy & Intelligence is so important to Life Science organizations?
If you look back at the industry in the last 20-30 years, the role of regulation has done nothing but increase, and that trajectory is going to continue. There’s also an increasing number of players that are getting involved, such as patient groups who have legitimate concerns about the development in their disease area due to regulatory or scientific barriers that are awaiting a breakthrough.
Organizations like the FDA want to make sure the end points agreed upon with the sponsor are scientifically valid for purposes of efficacy and safety. We’re getting into more research and development of therapies in areas where we just don’t have that kind of information yet. We don’t have a lot of information on certain rare diseases, such as the natural history of the disease; we don’t have a good feel for end points, and sometimes the patients have a lot more information than their physicians. In the end, we’re going to have to get creative in regulatory policy. We all want the same thing, which is more therapies for more diseases, but it’s going to be harder to get there if we aren’t thinking more creatively moving forward.
Regulatory intelligence is going to be even more important than it’s ever been. The fact of the matter is this: more is expected of companies now in terms of stepping up to the plate and being part of the solution, establishing mechanisms to address lack of information, and breaking new ground in regulatory areas and new therapeutic options. You really can’t step back and say, “it’s not my job.” This is not an add-on to traditional regulatory affairs, they have their responsibilities. This is really something that’s complimentary that needs to be a part of any regulatory affairs department.
How can Regulatory Policy & Intelligence benefit different groups, such as product development?
As a Regulatory Policy & Intelligence professional, it’s key to have a good understanding of what the needs are of your organization. For example, there are a lot of development projects that are dropped early in the stage for a variety of reasons. In many cases, it’s because whoever is making those decisions doesn’t see a clear regulatory path. If there’s a willingness to explore and try to find a regulatory pathway, and specifically looking at what the hurdles are – such as being blocked because there isn’t a good understanding of applicable endpoints – then the issue can be resolved. It’s resolved every day in negotiations with the FDA, often on rare disease products and unmet medical needs projects. That’s an example of how product development would benefit, and that really takes somebody who understands the process and who understands that the client is the product development department in the company. They have to figure if there is enough there for the team to explore whether or not they can provide a solution, either with patient groups or internally.
As we’re moving ahead with Oncology, there’s a push towards basket studies. There are new models for drug development that are supposed to be quicker, cheaper, etc., so somebody has to be pushing those kinds of products into the applicable kinds of trials. Regulatory Intelligence has a significant role in trying to ensure that we can maximize the company’s research and its outcomes. This needs to be done without sacrificing some of the potential benefits of those therapies by ensuring the team can find a way to resolve issues in the Regulatory Policy area moving forward.
Sometimes there are issues in reimbursement, and I have seen situations where reimbursement fears have chilled product development. One of the roles here for Regulatory Intelligence is also working with groups like CMS. Years ago, I was working with a client who had a product that required a surgical implementation, and there was finally enough evidence where they could implant this in two places in the same organ, but CMS only allowed for one implementation, even though, medically, there was no question that this product was effective. One of the things we had to do was resolve that issue with CMS before moving forward with any further development.
How do you normally go about Regulatory Policy & Intelligence? What are some learnings or best practices you can share from your work in this field?
I think it’s extremely important to have relationships with Regulatory Policy Makers at the NIH and FDA in areas that are of therapeutic importance to your company. Having those relationships, being able to keep in touch with people, see them at conferences, and be helpful – maybe NIH will ask you to serve on a committee – are key. Here are a couple examples:
I was asked to serve on an NIH committee for an international project (in partnership with the administrative health organization of the other country) working on some cooperative ventures. They asked representatives of the company to attend this meeting in this foreign country. This meeting was very beneficial to the head of the country’s office for my company; all of the important government officials had a seat at this meeting and could network. Having this cooperative meeting helps everyone, and the NIH needs people to network to move forward with a collaborative mindset into the project.
I also served on the Pediatric Formulations Policy Group for a couple of years. It was basically a group that the NIH was coordinating at the time to try and push pediatric formulations. You had people from a variety of areas, including scientists and policy people who had worked on this issue in the past. Those were all important, helpful contributions that Regulatory Policy & Intelligence can make.
Another example is when I worked for a company – we were researching a very rare disease of which no therapies were currently available. I happened to be speaking with one of the Regulatory policy makers in the Federal Government, within HHS, about another matter, and they mentioned that they were holding an exploratory conference the very next day, and it happened to be on this disease, the exact one we were researching. They had no idea we were researching it, but there was opportunity there. They said, “we didn’t know anyone was doing any work in this area,” as some companies can be a little closed-mouth. We were able to get somebody into the conference, and someone from our regulatory intelligence department was very thankful - it was very helpful. So, it is very important to know what’s going on; the only way you know that is if you have those conversations and those relationships. Having those relationships is the first step to a successful career in Regulatory Policy & Intelligence.
Also, it is extremely important to read the mail that comes from the trade associations/regulatory committees because they’re always asking for comments, or for people to look at something. If you don’t look at something, and your company is subsequently affected by an issue, you’ve only got yourself to blame. Just like the phrase, “Be the change you want to see in the world.” That applies to Regulatory Intelligence too – you have to step up! Companies have to provide their expertise and shouldn’t be shy about doing so.
Does Regulatory Policy & Intelligence apply to only the U.S./FDA or does it have global impact? And if so, how does global impact effect the approach?
Yes, it applies globally, and it’s not just FDA. Usually, companies based in the U.S. launch to America first, but some products are launched in more than one market at the same time or in close proximity to one another, particularly for those products that meet a previously unmet medical need. In this sense, it’s extremely important to have various international strategies, including in the EU, Japan, China, and Russia, if your company ever plans to market to those countries/regions. I’ve worked for companies that have planned to market throughout the world; Regulatory Intelligence can cross national borders, and it is very specific to the needs of the company and the product they are providing.
The trade associations’ Regulatory Committees’ work is often a global issue. Some of the larger companies have people who are dedicated to making sure the messages and the instructions given to trade associations on issues of impact to the company are consistent across national borders. Of course, not every company can do that, but that is a critical role. You don’t want to tell Pharma one policy, and then endorse something else for SMI. Consistency is important, and internal coordination on goals, messages, and company positions to deliver externally is equally important. The last thing you want to do is put your company in a position of planning for a marketing strategy, then finding policy barriers that prevent your team from doing so.
Do you think Regulatory Policy & Intelligence could be beneficial to small companies as well as large organizations? If yes, why?
Not only is it beneficial to small companies, but it may be even more beneficial to smaller organizations. If you’re marketing something, and you’ve got one or two products and fail to recognize early in the process that there’s a regulatory policy barrier, you’re out of luck; you’ve got one product and you can’t get it to market.
Larger companies have more problems, just because they tend to have more products, but often times smaller companies have bigger problems. The impact is so much greater. It is critical that smaller companies take the time to look at policy issues as they come out and as the requests for comments are made, particularly when they are working cutting-edge scientific/therapeutic areas, since the consequences can be catastrophic if they miss something.
I’ve been in coalition with companies that found themselves in Regulatory Policy predicaments. Sorting something like that out afterwards is always much more difficult – an ounce of prevention in Regulatory Policy is a pound of cure.
About the Author: Anne Marie Finley, Managing Expert, YourEncore Regulatory Center of Excellence, and former Vice President, Government Relations & Public Policy at Celgene, is a subject matter expert in regulatory strategy for pharmaceutical and biotech organizations. As a former executive of Regulatory Policy and current President of Biotech Policy Group, Anne Marie has developed robust regulatory solutions and provided holistic perspectives for the entirety of her career. She is currently on the Board of Directors of the FDA Alumni Association and was named to the list of "Top 50 Global Thought-Leaders in Orphan Drugs and Rare Diseases" by the World Orphan Drug Congress as "an expert with an expert with access to different national regulators. Anne Marie has a unique holistic perspective on the challenges surrounding orphan drug development across multiple markets."