With today marking the ninth International Rare Disease Day, CTA recently spoke to Mark Milberg from Ultragenyx Pharmaceutical Inc., a company that develops treatments for rare diseases. In this compelling interview, Mark reveals the pressing need for specialised rare disease vendors.
Clinical Trials Arena: Explain some of the challenges you face when outsourcing clinical trials for rare diseases?
Mark Milberg: In this space, the crux of the matter, to cite an example, comes if you only have one site in one country then how do you deal with the fact that you're only assigned someone at .05 FTE (Full-time Equivalent)? We're expecting to have very skilled people who have the right type of mentality, people who ensure that every patient gets through the conduct of the trial. We may have lots to follow up and it can be difficult to find these patients, so rather than thinking in terms of .05 of an FTE, I'm thinking that perhaps we can bring on people on a time and materials contract (fee for service). Resourcing is really the issue for me; you want a very skilled person yet I don't want to have them at full-time equivalency if we're only going to be using them for one site in one country. So the issue boils down to cost-effective resourcing - we need people who truly believe in working with the types of rare and ultra-rare patients we work with.
CTA: So how do you go about mitigating that and find the right kind of outsourcing model?
MM: We try to harness a hybrid model where we'll supplement resourcing internally if it's a Phase I or Phase II trial. If it's a Phase III study, we'll go outside the CRO we're contracting with and find a staffing model from an independent monitoring organization. Another way is to ensure the roles and responsibilities are clearly stated in the Statement of Work, so these are some of the ways to mitigate these problems. However, we're yet to come across the perfect solution.
CTA: Is it a case of over time companies will find an effective way of resourcing to meet their needs?
MM: There was a meeting I attended recently where a CEO of a large CRO talked about getting to the critical mass of need in order for CROs to provide a dedicated unit of their personnel to serve rare and ultra-rare disease Sponsors. Unfortunately, for smaller companies, we don't they have the luxury to plan that far ahead, so we will wait until they arrive and search around for what we think is the closest to that model. For us at Ultragenyx, we are open to sharing our needs and our clinical program information to find the right fit. Maybe it is a matter of time to get to the critical mass where companies like mine will grow and we get to a point where the industry needs to be. Personally, I don't feel we're going to get there until we have units within CROs that are dedicated to rare diseases.
CTA: Is there a pressing need for vendors that specialise in rare and ultra-rare diseases?
MM: I think there is. The complexities of the protocol are much more intricate than a standard research trial. We're trying to do our best in really getting people trained to understand these protocols and the nuances of these kinds of studies. They need to have the clinical skill set to be able to know what to look for and to help us because there are certain things we think we do really well, and other things we need expert help with. Part of the issue I've found over the years is the CROs that may even have the ability to get us to where we need to be might not be as forthright about that path; they're waiting for us to ask. I would rather be told this is the path because we don't have all the answers, we're looking for that help.
CTA: How do you determine your vendor has the requisite regulatory knowledge?
MM: A lot of it is done upfront before we do a bid defence. We ask prospective vendors to send us their per country regulatory knowledge, so you can gauge their acumen. Furthermore, most CROs these days will have leads within that country that do regulatory as well as the study start-up and the clinical conduct, and I think it's sometimes an opportunity to educate our internal staff because that expertise is present most of the time. So whether it is at the initial capabilities presentation before we send out an RFP, we always look to learn how qualified and suited they are to the trial.
CTA: How do you see things evolving over the next few years?
MM: I definitely want to see dedicated units for rare diseases because what we do is so unique and what we need is so unique. I'm intrigued to know how we're going to get to that level of critical mass. I think overall with the largest sponsors doing more innovative things I think that bodes well for the rest of the industry, and we need to feed off that. Technology is getting better and better; the individualised patient tools and clinical endpoints will only benefit us in the rare disease space in the long run. I'm excited - it's a great time to be involved in clinical research.
* Mark Milberg is the Director of Clinical Procurement and Outsourcing at Ultragenyx Pharmaceutical Inc.