Towards the “Dossier In The Cloud” to Democratize Global Access to Medicines
A Conversation with Accumulus Synergy CEO Francisco Nogueira

n February 2024, the nonprofit industry association Accumulus Synergy announced the launch of its initial cloud-based information and data exchange platform through a regulatory reliance pilot project. This is in line with the overall mission of Accumulus to dramatically accelerate getting critical therapies to citizens of the world by using technology to simplify regulatory interactions and by bringing forward the much-anticipated concept of “a dossier in the cloud” to support real-time information exchange between those that develop medicines and those that review and approve them.

Led by Roche, one of the 12 Accumulus Synergy Sponsors, this particular pilot engages regulatory authorities across six continents and allows them to submit questions and receive sponsor answers on a post-approval change submission via the Accumulus platform and follows World Health Organization (WHO) principles of good regulatory reliance practices.

Accumulus Chief Executive Officer Francisco Nogueira discusses this pilot and its potential impact in the following Q&A with Global Forum Editor-in-Chief Alberto Grignolo.

DIA: Would you please share and explain as many goals and as many details of this reliance pilot as you can, understanding there may be confidentiality considerations that apply?

FN: You and I spoke about a year ago when we were in a conceptual phase and it’s really a pleasure for me to talk to you today now that we are in practice. We are “live.” We are working with multiple agencies around the world.

I want to first give thanks to Sanofi for having tried what hadn’t been tried about a year and a half ago and doing a similar pilot and testing it out. On this Roche-led pilot, we’re showing the art of the possible. When you have 30, 40, 50 agencies around the world collaborating differently through leveraging technology and looking at ways to unify how they do reviews, you’re starting to get to the future of regulatory interactions.

The world is going to get more complex. Studies are going to get more complex. The capacity for agencies around the world to review these are not going to get easier and they’re not going to get simpler. This is the beginning of what I look at as work-sharing, collaboration, and (at some point) even components of convergence. This is the beginning of showing the art of the possible.

DIA: What is the specific goal of this pilot?

FN: The defined goal of this Roche-led pilot and in collaboration with some of the WHO frameworks is to reduce timelines. As you may be aware, post-approval changes can take anywhere up to four years from the point of initiation between some of the faster health authorities and lagging health authorities. This is not a criticism of the lagging health authorities, but it’s a function of volume and their capacity to address volume. Whether you think of it as a two-and-a-half year pilot or a two-and-a-half-year turnaround, our goal is to take that three years, four years, which are averages now calculated by WHO, to somewhere around six months. You’re effectively taking two-plus years out of the system and that will be helpful, whether it’s drug shortage or supply chain efficiency or just making sure that there’s alignment between regions. It’s important for patients, for pharmaceutical companies, and for health authorities who can begin to collaborate and see each other’s questions and answers and minimize the amount of new work they have to produce.

DIA: Given Roche’s lead, what is Accumulus Synergy specifically contributing to this pilot?

FN: We’re contributing the technology. We now have technology in production based on security at its most basic. We are secure for our data in transit, we are secure for our data at rest, we meet the security requirements of the participants of the pilot, and we provide a platform where health authorities can log in and feel like it’s a secure environment and share this information dynamically and in as close to real time as possible, versus trying to leverage either paper mail or eMail, or other ways to share communications.

You can have what we call the “one to many”: Now we have a platform that is “one sponsor to many agencies” and allows those agencies to collaborate among themselves. It’s a really important opportunity and we’re excited to be part of this journey in parallel with Roche. It’s remarkable that their portfolio opportunity and our readiness became a match at the end of last year.

DIA: Can you share how many regulatory agencies are involved in receiving the post-approval change submission in this pilot?

FN: It’s more than 35 health authorities.

DIA: How long will the pilot last, and when will it be completed and assessed for lessons learned? Will the results be made public to any extent, or will they remain within the confines of Accumulus?

FN: We are working closely on two fronts on this. We are picking up lessons learned almost daily, and we collaborate with Roche almost daily because it is a “first” and so there’s always going to be lessons learned. The pilot will last through the six months of the review cycle. And I know both from our discussions with Roche and what we want to do internally that there will be presentations of lessons learned, including at DIA 2024. We want to make sure that we share lessons with others because we don’t want this to be a one-off. We already have a number of our Sponsors lining up to do new pilots in a very similar fashion already slated for 2024.

Of course, we work in tandem and in collaboration with Roche. Some of it is confidential to the way they work. But our intent is: As much as we can share, we will. We’re an industry association, nonprofit organization, and we want this to be of value to the world. I will also share that we are working with WHO and IFPMA to do a webinar about what we believe is the right framework to move forward. While this reliance has gone through one country, there are many other reference countries raising their hand to say, “We can be one of those as well.” We want to place this in a way that we–as a nonprofit industry association but also as a technology enabler–can help make this a scalable way of working.

DIA: How is the work of Accumulus and its mission informed by transparency, including informing the broader ecosystem about how these pilots are working out?

FN: Transparency, trust, and responsible innovation are at the core of what we do. We do our best to say what we’re going to do, and we’re going to do what we say. We want to be transparent retrospectively on what’s taken place through lessons learned, and prospectively about what we’re seeing in the broader ecosystem and how organizations can take advantage of that.

DIA: Even though it was launched just recently, is this pilot generating any surprising or perhaps even sobering outcomes or lessons learned?

FN: What’s surprising in a positive way is how many countries raised their hand to try something new. By countries, I mean regulatory agencies, whether they’re a very small organization in perhaps a lagging market or a larger organization in one of the leading markets. I was surprised at the heterogeneity of those agencies and levels of maturity. But they all raised their hand and said that we want to try this. We want to give access to people in our team, to get in and look around and be part of a solution that I suspect has been years in coming in their minds, and certainly welcome.

DIA: If there are more than three dozen regulatory agencies on six continents, is it fair to say that there’s no angle of the world not represented in this pilot?

FN: That is absolutely fair. In a small organization like ours, we set out with the ambitious goal to dramatically accelerate critical therapies to citizens of the world. We remind ourselves of that every day. But when we started, a lot of folks said, “Come on–’the world’? Really?” It makes us proud that our very first one is across multiple continents. It is about democratization of access. So, yes, it touches everywhere. And everyone.

DIA: How does this pilot reinforce existing efforts in the ecosystem at large to promote regulatory reliance and regulatory convergence? How might this promote true reliance and convergence in practice, on a global scale?

FN: There are often antibodies to change if they don’t see the change happening. We could say a lot conceptually, but you need to show the art of the possible. I’m excited about this because we’ve delivered the first opportunity and the first pilot, but it’s really what this pilot symbolizes for the future. You could begin to say: How do we do this in a place that is sustainable beyond just post-approval changes? How do we move this to the next level? How do we continue to support large-scale global work sharing opportunities, and how do we show that those opportunities can take us one step closer to convergence? I know we’re not going to get there overnight. And I know that there’s a lot of legal constraints and perhaps even political constraints around convergence. But every step in that direction is a step in the right direction.

DIA: There are some 200 countries around the world, each with its own national regulatory authority. Some geographies have highly developed regulatory agencies, others have less developed, and sometimes even emerging, regulatory agencies. How does this kind of pilot or indeed the work of Accumulus as a whole address the disparity in just sheer capacity without implying any value judgment?

FN: We learned a lot during COVID. You can’t only address a certain percentage of the population and assume that the rest of the population will be fine. We learned that in a dramatic way where we didn’t have a choice. But we also learned during COVID that less-developed countries struggle. They are still receiving paper today as a form of information sharing. Their infrastructure is not set up for even components of eCTD, one could argue, because after 25 years, I believe only 25 major markets accept eCTD today. Our intent, and you’re starting to see this with the Roche-led PAC reliance submission, is: We have low-income countries with small agencies having as many users as a large, developed nation with a more well-funded set of health authorities and a set of staff at their health authority. We always knew that the tool had to be of light touch: You don’t have to have fiberoptic internet to access Accumulus. We designed it so it would be easy to access with low burden of usage. Training is a couple of minutes on a digital module. You can get up and running very quickly.

Now, can we solve for the expertise in the region? No. Can we solve for how the future of gene and cell therapy get understood and adapted within a region? No. But we can help democratize the sharing of information. What we’ve seen with organizations like EMA, who’ve been very good around reliance and being a reference country (I think 70% of all reliance opportunities have EMA as a reference country), they’re beginning to be supportive to other regions of the world that may not have that opportunity. There’s multiple players trying to democratize access to medicine.

DIA: EMA has also been very supportive of the emerging African Medicines Agency (AMA) which is in the process of being established this year and next. The AMA is going to become one of those agencies that presumably would be participating in future pilots or activities connected with the work of Accumulus. Have you given any thought to the African Medicines Agency?

FN: Yes, and it’s a response with a smile, saying “stay tuned,” because we are involved for sure. But in the meantime, until that construct comes into reality and matures a little bit, we are having opportunities to do some pilots in the not-too-distant future in the South Africa region as well.

DIA: Accumulus have committed yourselves to the “dossier in the cloud.” How does this pilot fit into that notion of establishing the dossier in the cloud as the future standard for regulatory submissions?

FN: We see this as a three-step approach. First step is to get everybody to collaborate in a common place. That’s what we’re doing now and will continue to do. Second: Once we have that collaboration environment set up, to then bring in–“ingest”–the dossier for broad distribution. That dossier will still be somewhat based on the current construct of lots of PDFs in a document-based paradigm. We want to spend some time there, but not more than we need to, to begin to shift to structured content and data. Third: Everybody’s in this kind of common place where you can share information, share data. Many agencies and sponsors of new medicines are at different levels of maturity but put them all in the same virtual place and you start allowing for the ability to upload, ingest, and share a dossier in the cloud in the traditional way. And then module by module, you start shifting into data and structured content. We think that opportunity’s not five or ten years away; we see that opportunity within 12 to 18 months–the full gamut.

DIA: So how essential or even critical is the Accumulus platform for this pilot or initiatives like it? Could a sponsor have done this pilot with three dozen regulatory agencies without your platform?

FN: We did a lot of things in COVID that we would say can’t be done, and so if I said it would absolutely not be able to be done, I think that would be a nontrue statement. But what effort and at what value and what level of complexity and error-prone opportunities would we have been introducing? And it wouldn’t be sustainable, right? You have 36 countries mailing, emailing, through different mechanisms, questions at different times. How would you share that back? It gets quite complex, because part of it is not just providing the opportunity to see each other’s work; it’s contributing your work and seeing everyone else’s in as close to real time as possible. We believe we bring scalability, we bring efficiency, and we bring a level of quality and compliance that wouldn’t be as evident otherwise.

DIA: Are speed and convenience two elements of advantage by using the platform?

FN: Absolutely. It’s speed of information sharing, and convenience and practicality of what you’re sharing as well. Again, this is the first step. But if you start getting into a “dossier in the cloud,” it gets even more complex. Again, we’re building the environment to be able to scale, to be able to evolve, and to be able then to innovate.

DIA: Is there any competition relative to the work that Accumulus is doing? Is any other organization doing more or less the same thing, attempting to achieve the same goal: the “dossier in the cloud”?

FN: As an industry association, as a nonprofit, we want the ecosystem to evolve. There’s a lot of great work being done in the cloud space. There’s a lot of great work being done within organizations as they establish their data lakes, as they prepare to move to the next generation of how we do global dossiers. And we see a lot of energy at agency levels with their own independent goals. How it all comes together in the next three to five years is still to be determined. But we see ourselves as an innovator that operates without real constraints around returns on investment to their shareholders. We want to be price sensitive to the market so there’s no barrier of entry, whether you’re a large pharma, a small biotech, or an academic center, and we pride ourselves in staying true to our mission.

DIA: Does Generative AI have any role to play in your platform? Or does it have a role with sponsors as they prepare submissions to be fed through to regulatory authorities through the platform?

FN: Yes to all of it. We are looking deeply at Generative AI through the lens of responsible innovation. AI right now is a great sort of buzzword, and everybody throws AI around, but we have to be very thoughtful in how we do this. We see an opportunity to play with AI later in 2024 in a development environment, things like questions asked, questions answered. How do you start to predict what’s coming? How do you query the information you have? And over time, could you start to query your own new drug application? Could the agency query your application in a different way than they do today? So there’s lots and lots of applications for AI. It’s how thoughtful do we want to implement it in a responsible way and make sure that we remove as much variability or bias from what we build.

DIA: Do you have talent within your organization that focuses on AI applications?

FN: We have expertise in all aspects of the engineering work we do from cloud to AI. We have this model of crawl, walk, run. We want to be able to crawl before we walk and walk before we run. Jumping too quickly to AI also becomes sort of a distraction, even though I know these days people don’t see it that way. But you really have to get the fundamentals in place before you start accelerating AI.

DIA: Finally, where do this pilot and its outcomes go from here?

FN: We have an opportunity, and we have a responsibility to patients around the world. We have the technology and we proved that you can have information flowing from point of collection at the patient level all the way through decision makers in a much more formal and fast fashion than we do today. That information needs to remain nontrapped in PDF. We think that information will lead to, at times, failing faster by developers of new drugs because the information is collected more quickly and shared more quickly, but then also the ability to get to approvals worldwide and get to post-approval changes worldwide much more quickly and much more dynamically than we do today.

Listen to Francisco’s complete closing message about this pilot and its outcomes below.

Read the preceding article for specific details of the Roche pilot project.