Early BTD projects offer lessons for drug companies, says Tufts CSDD

Flexibility and a willingness to learn from each other are key factors that enable drug companies and regulators to engage successfully in development projects that have been accorded Breakthrough Therapy designation, or BTD, according to R&D executives participating in a roundtable recently hosted by the USA’s Tufts Center for the Study of Drug Development.

Since its launch by the US Food and Drug Administration in 2012, the BTD program has been associated with lower total approval and development times, compared to other expedited drug development programs, according to Tufts CSDD.

"BTD can save developers time and effort, but it requires a company-wide commitment to work with senior FDA managers, who can provide intensive development guidance, sometimes as early as Phase I clinical trials. It also means that the sponsor must have the capacity to speed up manufacturing and other operations," said Christopher-Paul Milne, associate professor and director of research at Tufts CSDD.

Participants’ observations

Observations from participants in the Tufts CSDD Executive Forum, which included representatives from pharmaceutical and biotech companies, contract research organizations, and the FDA's Office of New Drug Products, summarized in the April Tufts CSDD R&D Management Report, released today, included the following:

• Evaluating potential BTD candidates generates internal debate in sponsor companies over what constitutes "a serious condition," whether existing therapies adequately address the condition, and if a clinical need that the new drug would address is currently unmet - all required of BTD candidates.
• The FDA seeks open, ongoing discussions with sponsors through the entire process so both parties can avoid surprises and jointly resolve issues. This includes discussing with BTD applicants their new drug application submission strategy even before BTD status is decided.
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• Pursuing BTD is "emphatically worth the effort" if sponsors fully understand the FDA guidance and bases on which breakthrough designation can be granted, build review and evaluation points into the development plan, and prepare a development strategy and study design before FDA discussions begin.

According to Dr Milne, BTD programs to date have addressed a broad range of disease areas: cancer (43% of all BTD awards); infectious diseases (16%); rare inherited disorders (14%); cardiovascular diseases (5%); and all other (22%).