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For a half century, lupus patients – most of them women – had few treatment options and no signs of hope in the world of drug development. But in 2011, when the first new drug approved for systemic lupus erythematosus in more than 50 years entered the market, a new wave of confidence swept across the drug development industry.

Today numerous treatments are in development to address this chronic inflammatory disease. Yet, challenges remain for biopharma companies striving to create new drugs for this uncommon disease.

A group of experts from Quintiles’ Rheumatology Center of Excellence recently conducted a novel analysis of lupus clinical studies registered in between 2004 and 2013 and discovered that there was a three-fold increase in the number of patients required for lupus clinical trials between 2010 and 2013 compared to the previous three years. The results of the analysis were presented at the European League Against Rheumatism (EULAR) 2014 congress in Paris. The results underscore a growing interest and investment in treatments for this disease as well as complicating factors in the development of drugs for a small patient population.

Good and bad

One of the major barriers to developing new therapeutics for any disease continues to be patient enrollment. This barrier is heightened for lupus due to the limited patient populations, the heterogeneous nature of the disease, the inexperience of some specialists in evaluating and caring for these patients, and the need to test a new drug in patients when they have flare-ups which are infrequent for some patients. Compounding those challenges are the number of participants now required to demonstrate safety and efficacy in new lupus drugs.

This requirement for more clinical trial participants — within a program and across programs — is driving demand for more trial sites globally, which is adding time and cost to the research process. As the number of clinical sites engaged in lupus research is limited and competition for patients increases, drug development timelines are lengthening. This has direct impact on trial operators who must manage stakeholder expectations while navigating this increasingly complicated clinical trial space.

These pressures can be alleviated by expanding the global network of investigators who are interested in lupus studies. One of the key outcomes of our analysis is that we must add more clinical trial sites, as it can help us as an industry recruit more effectively, deliver trials faster, and get the outcomes known more quickly. Increasing use of novel approaches, such as adaptive trial designs and personalized medicine will further help facilitate ongoing drug development in lupus and ensure provision of new therapies.

Unless additional sites are deployed and novel approaches employed, the cost and time required to develop a drug for lupus could become prohibitive, especially as the number of drugs in development grows. Ensuring the need for new treatment options is sufficiently met will require partnership across the biopharma industry, investigators, clinical trial sites and regulators. But together, we can work to improve outcomes around this important research.