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Exclusive Life Science Feature cular dystrophy (DMD). In the opinand others, would allow companies ion of its supporters, the drug caused to learn from past errors and sucsignificant improvement in patients' cesses, according to Woodcock. walking ability along with boostGHOST OF THE SEQUESTER ing the levels of dystrophin, a putaThe ability of the FDA and CDER to tive biomarker for DMD symptoms. conduct industry-friendly policies When the FDA asked the company and services ultimately rests on the for more information from its dataset federal budget. Although the endon eteplirsen to support its request of-year budget deal restored some for accelerated approval, the head of level of stability to the process, the the main DMD patient group sent a same political powers that forced long, petulant email to Woodcock in sequestration and shutdown on the response. U.S. government continue to promNo one expects Woodcock to comise more confrontation than comproment specifically on the eteplirsmise. Uncertain funding in the future en case, but she has this to say: can be more intimidating than sure "Sometimes people do pitch their scarcity in the present. case to me, and they're just so con"We would like to receive a budget vinced they have a breakthrough. each year at the beginning of the fisThey may come in with incredible cal year that we can execute with conclinical data, but we say, 'Well, just fidence," says Woodcock. "That hasn't repeat it, open label; take another 30 happened in a very long time. When people and show you get the same we do get the money, we don't know results, and then we'll talk about how much of it we will get because of accelerated review. It depends on the various cutbacks, sequestration, what the results are and how conand so on. All this uncertainty makes vincing the data are." it very difficult to manage a program Woodcock says she identifies with as complicated as a drug regulatory the patient-community advocates program. So, the situation is very and could even see herself joining suboptimal. But we just do the best them if she faced a disease such as "The science now can't be any better for we can because we have an important ALS or AIDS. "HIV was the instigation developing new medicines and for improving mission." for accelerated approval; we said we Framing the budget as just another were going to approve drugs based manufacturing. We are hopefully on the verge of problem, among many to be solved, on a surrogate marker if it was rearevolutions in both those areas." seems like a sensible solution for an sonably likely to predict clinical benJanet Woodcock, director of the Center for Drug Evaluation and Research (CDER) at the FDA absurd predicament. For example, efit. And we've done a large number Woodcock says the funds from the of accelerated approvals since then, PDUFA programs were sequestered last year, "So we didn't get many of them for orphan or rare diseases but also for cancer and the increase that we negotiated very carefully with the industry so forth. But the data, even if it's Phase 2, must be convincing." for additional services we would provide. Now we can't provide Data quality in general needs industry's attention, Woodcock services at the level we promised." says. Academic data is especially problematic, with some estiAs always, however, and in all our conversations with Woodcock mates that up to 50 percent of it cannot be reproduced. She through the years, she remains not only optimistic but also sincerely believes pharma companies are much more careful about validatexcited about her job and the agency she has helped shape in that ing the research data they generate, considering the money at time. "The science now can't be any better for developing new stake in selecting development candidates. medicines and for improving manufacturing. We are hopefully on Yet, she says, strange as it may seem, companies still routinely the verge of revolutions in both those areas, which will create great fall short with clinical research data, often because of poor pracbenefits for the public, and the industry you see today will not be tices by individual investigators. But poor trial design, rather the industry you see in 10 years." It is a safe bet that, during most than poorly run trials, may compromise trial results as well, of the changes she foresees, Janet Woodcock will be there to help she believes. A solution? More data transparency, ideally shared them along. through a "trusted third party" or custodian as proposed by GSK 24 LifeScienceLeader.com February 2014