Clinical Trials Arena: What are some of the biggest challenges you face in your role?

Pablo Lapuerta: I think there are inherent challenges in drug development. It is always full of surprises and you have to strive to ensure your drugs are not only safe but effective. The thing with drug development is that there are safety questions you have to address, questions on efficacy you have to address, and you really need to collect quality data. Our biggest challenge is the uncertainty of the science in drug development.

CTA: Would you say that is part of a wider industry problem, i.e. making sure the drugs developed are of the highest quality?

PL: Yes, and I’ll give you an example. We’re very fortunate that we had positive results in Phase III for a drug that’s intended to treat a rare type of cancer. There was a lot of uncertainty over our ability to enrol patients. We had to make decisions advancing to Phase III based on experience with only a few dozen patients. We were in a therapeutic area where showing benefit had been difficult and other companies had failed. We were very fortunate to have success, but it was a long road with a lot of obstacles to overcome.

CTA: How did you overcome the issues that this trial presented?

PL: For our orphan drug programmes, one of the fundamental things to do was to understand the data at the patient level. So even though we didn’t have a lot of patients, we had a solid understanding of how they were doing. That was an important guide that helped us make very appropriate choices in our development programme.

CTA: So even though you had a small pool of patients, did you ever have problems retaining them?

PL: No, fortunately our patients liked the investigational drug they were taking, and we’ve had some who’ve been on the drugs for as long as five years now.

CTA: What would you say is vital to retaining patients in orphan drug trials?

PL: I think one of the reasons why our patients are taking our drugs long term is because we’ve shown a lot of flexibility on how they take it. In our long term studies, we allow our patients to pursue other treatments. We don’t limit their options. So investigators must be flexible when dealing with patients, and I think that’s key in retaining them.

CTA: Did you ever encounter problems obtaining and managing clinical data?

PL: We never really had issues managing the data. We’ve worked with CRO partners that have been very effective in doing that. One of the things that made it easier for us was having a programme that included electronic diaries. Patients liked using them and they were very compliant with them, and I was really encouraged by the quality of the data we received.

CTA: What advice would you impart to smaller pharma companies who run orphan drug trials?

PL: I think you have to have a culture that’s patient focused. The patient has to be the priority. If the patient’s not the priority, you’re not going to be able to recruit. Sometimes the protocol becomes a priority or the imaging study becomes the priority – it really has to be the patient first.

You really need to learn your data at the individual patient level if you’re involved in an orphan drug. Otherwise, you just can’t rely on statistical tests when you’re handling small clinical trials with relatively few people.