The vision of Life Science Leader is to be an essential business tool for life science executives. Our content is designed to not only inform readers of best practices, but motivate them to implement those best practices in their own businesses.
Issue link: https://lifescienceleadermag.epubxp.com/i/267232
insights LIFESCIENCELEADER.COM MARCH 2014 50 CLINICAL TRIALS How Rare Disease Know-how Can Shape Big Pharma Clinical Trials N I C K T A Y L O R Contributing Editor When Andy Lee joined Genzyme from Pfizer, the differences between the companies were staring him in the face. Walking the corridors around his new office, Lee passed artwork by rare disease patients. This was a different sort of drug development organization. Even the building was patient-centric. L By N. Taylor HOW RARE DISEASE KNOW-HOW CAN SHAPE BIG PHARMA CLINICAL TRIALS ee, now senior VP and deputy head of clinical sciences and operations at Sanofi, recounts this tale while talking about the experience of going from Big Pharma to biotech and back again. While Lee has nothing but good things to say about Pfizer, it is clear the 18 months he spent at Genzyme before its acquisition by Sanofi reshaped his thinking, particu- larly regarding how the industry runs clinical trials. "The classic clinical trial asks 'what does the sponsor want and need?'" says Lee. Everything is focused on deliver- ing the data the sponsor needs, with little consideration given to the two stakeholders intimately involved with generating the results — investigators and patients. This attitude manifests in many ways, such as with electronic data capture (EDC) tools designed more for the sponsor than the user, clinical study nurses, and doctors. Of course, the irony is that the sponsor- centric approach has done little to ben- efit Biopharma companies, which are paying more and more to run ever-larger trials that nonetheless still fall short of expectations. Activating a trial site costs up to $35,000, yet many never recruit a single patient. Equally, activating the trial sites and building other aspects of a clinical infrastructure takes time, push- ing back drug approval dates. Tufts Center for the Study of Drug Development calculated that between 1999 and 2005, the average length of a clinical trial increased by 70 percent and the burden on study staff rose by 67 percent. At the same time, enrollment fell 21 percent and patient dropout rates grew 30 percent. Lee saw the model was unsustainable for sites, patients, and pharma, and set about adopting lessons learned at Genzyme to change the para- digm at Sanofi. LEARNING LESSONS FROM RARE DISEASE TRIALS The different approach taken at Genzyme was borne out of necessity. Biotechs, even those that grow as big as Genzyme, are used to running trials on a tight budget. Splurging $350,000 on 10 clinical trial sites that never recruit a patient is an unacceptable use of limited resources. The nature of the drugs developed by Genzyme, which target rare diseases, increases the likelihood this scattergun approach will fail, too. Around 6,000 people in the United States have Type 1 Gaucher disease, the genetic disorder targeted by Genzyme's blockbuster biologic Cerezyme. There are 4,300 times as many patients with diabetes. When designing a clinical trial for an orphan drug — the term for treat- ments targeting rare diseases — compa- nies must think how this massive differ- ence in prevalence affects their patient recruitment strategies. "Finding a rare disease patient is like predicting the next lottery winner," says Lee. Instead of simply activating sites in metropolitan areas and waiting for patients to arrive — the flawed "if you build it, they will come" strategy — rare disease drug developers must take a more nuanced approach. It is no use activating trial sites in the 10 biggest cit- ies if the only patients you can find are in rural areas of Brazil or Montana. Consequently, Lee and Genzyme design studies that build support, capacity, and infrastructure around the patient long before the trial begins. This reverses the traditional model and takes the trial to the patient wherever possible. Instead of telling a patient to travel to a trial site, a nurse visits the person's home to do the infusion. When travel is unavoidable, the sponsor helps with logistics. "It's not rocket science. It's just about making things easier for the patient and site," says Lee. The process starts upstream in the study designs, where the input and engagement of patients and advocacy groups is essential. Technology can facilitate this approach. Lee is particularly effusive about the potential for telemetry innovations to 0 3 1 4 _ C l i n i c a l _ T r i a l _ G e n z y m e . i n d d 1 0314_Clinical_Trial_Genzyme.indd 1 2 / 1 9 / 2 0 1 4 2 : 4 2 : 5 3 P M 2/19/2014 2:42:53 PM