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Exclusive Life Science Feature HOW TO APPLY FOR THE BREAKTHROUGH THERAPY DESIGNATION Keeping up with global regulatory changes can be a challenge for a small or virtual pharmaceutical/biotech. If you have a drug in development that might qualify for the breakthrough therapy designation, here is what you need to know. The request for the designation should be submitted concurrently with, or as an amendment to, an Investigational New Drug (IND) application with a cover letter and a completed form 1571 with the following information, which in most cases should be explained in approximately 10 to 20 pages: If the breakthrough therapy designation request is submitted to the sponsor's IND as an amendment, the cover letter should indicate the submission as a REQUEST FOR BREAKTHROUGH THERAPY DESIGNATION in bold, uppercase letters. If the request is submitted with an initial IND, the cover letter should indicate the submission as both an INITIAL INVESTIGATIONAL NEW DRUG SUBMISSION and a REQUEST FOR BREAKTHROUGH THERAPY DESIGNATION in bold, uppercase letters. • the name of the sponsor's contact person and the person's address, email address, telephone number, and fax number • if applicable, the IND application number • if available, for drug products, the proprietary name and active ingredient and, for biological products, the proper name and trade name • the division or office to which the IND is being submitted or in which it is active • the proposed indications • a concise summary of information that supports the sponsor's breakthrough therapy designation request for the indication being studied, including: • the basis for considering the drug as one intended to treat a serious condition • the preliminary clinical evidence that the drug may demonstrate substantial improvement over available therapies (A sponsor should describe the preliminary clinical evidence, including, for example, justification for the clinical study endpoint used and a brief description of statistical analyses.) • if applicable, a list of documents previously submitted to the IND considered relevant to the designation request, with reference to submission dates. Paper submissions can be resubmitted to FDA as appendices to the designation request. If you do everything properly, you will find out whether your application has been granted or denied within 60 days. For more information, you can visit the FDA website, which provides contact information for breakthrough therapy coordinators, frequently asked questions, and other useful information at http://goo.gl/Cl13M. *Information derived from FDA website under Fact Sheet: Breakthrough Therapies 20 LifeScienceLeader.com July 2013 generated by Remicade in 2012. Currently the drug represents nearly 25% of the company's pharmaceutical sales. Since its initial approval, Remicade has received FDA approvals for 15 additional indications, which have not only dramatically increased the commercial viability of the drug, but also more importantly, have benefitted far more patients than the initial single indication. Hait cautions against using potential commercial success as the only criterion for pursuing a drug for approval, pointing to the first new treatment for TB in 40 years. In December 2012, the FDA granted accelerated approval for Sirturo as part of a combination therapy to treat adults with pulmonary multidrug resistant TB (MDRTB). Presently there are approximately 100 cases of MDRTB annually in the United States and about 500,000 cases globally. If you thought the initial market for Remicade was small, Sirturo's market, by comparison, is downright microscopic. "If commercial success were the only criterion, you probably would not go after a drug for TB," says Hait. "You need to place some bets on drugs you think are going to have a major impact for doing good in the world. That's what ethical companies do." Consider this — globally, one in every three people already carries the germ (bacterium) that causes TB. Once TB becomes active, and if left untreated, as many as half of those afflicted will die. "It's a huge problem," attests Hait. According to Hait, it is important to balance your company's portfolio with commercially viable candidates against those, which on the surface, may have less commercial appeal, yet could prove to be hugely important to humankind and help the company maintain an innovative culture. Sirturo had been in development for nearly a decade. Yet Hait assures that the drug was neither sitting on a shelf in limbo nor a pet project for which researchers allocated a small percentage of their time. "It was a drug that had a very dedicated small team in our labs in Belgium, who were continuously gaining a fuller understanding of how it was going to work and be used," he explains. If the drug had been killed based on its small potential commercial market, it could have been a devastating blow to the morale of the R&D; team. "It's really important for the culture to keep those projects going — when they are working," reminds Hait. Perhaps Sirturo has the possibility of additional indications, or it could lead to other breakthroughs arising from the research. Only time will tell. Although Hait expresses the importance of keeping projects going as they can lead to other research and serve as morale builders, even a company the size of J&J; can't fund every project. Sometimes it is necessary to find other opportunities for lower priority projects — thus the development of the J&J; innovation incubator. To prioritize drug development, J&J; uses a model that started in Janssen's oncology R&D; unit and expanded across all therapeutic areas. "We define the highest-priority diseases within each therapeutic area," says Hait. "For example, in immunology, we define