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Oct 15, 2011 (Vol. 31, No. 18)

Can the FDA Be a Catalyst for Innovation?

New Commissioner Realigning Programs to Better Support Core Functions and Responsibilities

  • Chief Innovation Officer

    Although FDA reviewers typically are trained in scientific research, applying that experience to the regulatory environment is learned on the job. “That’s a significant deficit for the agency,” Dr. Rodell said. “There’s no process to train the kinds of people the FDA needs.” Consequently, “it takes one to two years for new reviewers to get up and running.”

    Meanwhile, the FDA, in its determination to do no harm, risks being so cautious that it withholds live-saving therapies. Admittedly, it’s a delicate balance. “Innovation occurs so quickly that FDA staff can’t keep up with new technology,” observed Holli Riebel, president and CEO of Colorado BioScience.

    “Patients are waiting and—sometimes—dying, while amazing drugs aren’t released because of fears about potentially adverse reactions. Science isn’t perfect. We can’t push forward without some risks.”

    BIO is working with the FDA to reduce the risks of timidity by triaging new technologies by their potential contributions to science and healthcare (such as their ability to reduce false positives and false negatives), and providing the information necessary to help reviewers make informed decisions when they encounter these technologies.

    For the future, Greenwood suggested creating the position of “chief innovation officer”. This is different from the chief science officer already in place, Emmett said. “The chief science officer is tasked with enhancing the internal science infrastructure,” he explained.

    A chief innovation officer, in contrast, would work with external consortia and public/private/academic partnerships to coordinate and integrate their advances into the FDA as pilot programs. “For example, as new clinical trial designs or new biomarkers or new ways to develop or use electronic medical records are developed, the chief innovation officer would work with their developers to see that they are validated and tested in the FDA’s centers, to increase the FDA’s comfort level with these innovations.”

    Despite the significant challenges the FDA is facing, Dr. Rodell said there also are significant opportunities to design an efficient, 21st century agency. “Many of the recommended changes require legislation,” Emmett admitted, “but we want to elevate the FDA’s role to focus on innovation.

    “BIO advocates reinvigorating the Regan Udall Foundation as a place for public/private partnerships.” That foundation was created by the Food and Drug Administration Amendments Act of 2007 to support the FDA’s regulatory science priorities, which aim to clarify issues at the intersection of science and regulation.

    BIO also supports a progressive approval pathway so that even before final trials are completed, promising therapeutics could be released and monitored. Dr. Rodell suggested that could be accomplished safely and effectively by designing electronic medical records systems to accommodate retrospective, anonymous analysis of drug safety in real time. Therefore, drug developers could track adverse events associated with the commercial release of particular therapies more accurately than under the current system, which depends upon harried physicians taking the time to voluntarily report adverse events. Dr. Rodell added that the FDA is exploring this already with its pilot Sentinel initiative.

    That approach can also be used to generate comparative effectiveness data. As Hrusovsky elaborated, “Databases have 10 years of retrospective samples, in which you know the therapies, outcomes, expression levels, etc.” Leveraging that data could yield practice guidelines that address specific details rather than broad generalities, as well as reimbursement guidelines.

  • Getting Biosimilars on the Market

    In an article published in the August issue of the New England Journal of Medicine, Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research (CDER), was one of four agency officials who discussed the challenges associated with reviewing biosimilars. They concluded that science has evolved to the point where the agency can conclude that some biosimilars are similar to the reference product. But that conclusion, the co-authors added, must vary from drug to drug to reflect differences in the composition of those biologic drugs.

    “Given the complex nature of biologics, it’s unlikely that a ‘one size fits all’ systematic assessment of biosimilarity can be developed. Instead, FDA scientists will need to integrate various types of information to provide an overall assessment that a biologic is similar to an approved reference product,” the FDA officials wrote.

    That “totality of the evidence” approach, as the authors label it, reflects the reality that biosimilars differ by size as well as composition of molecule. They can also differ not just by attribute but by combinations of attributes.

    “It’s very hard to fashion rules that govern all biologics because they range from compounds like insulin, which are relatively on the smaller side although still much bigger than traditional small molecule drugs, all the way up to factor 8, which is a very large product, a very sensitive product,” Sara Radcliffe, evp for health at BIO, told GEN. “Fashioning rules that govern all of them is complicated. At the same time, as a regulator they need to provide guidance for biosimilar sponsors so that they can understand what needs to be submitted to the agency.”

    In the NEJM article FDA said that their strategies may include a fingerprint-like identification of very similar patterns in a number of different products. Fingerprint studies could reduce the extent of testing required for protein biosimilars, according to FDA. They add, though, that both animal and human testing “will generally be needed for protein biosimilars for the foreseeable future.” But for those studies to be most effective, FDA’s eventual guidance should better define fingerprint tests, both in terms of qualities to be studied and the degree of detail needed. —Alex Philippidis

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