At its annual meeting in June, the Biotechnology Industry Organization (“BIO”) discussed a wide range of suggested policy changes to effectuate change within FDA. BIO’s proposals focus on encouraging biotechnology innovation and bringing biologic drugs to patients on an expedited basis. One such proposal was for FDA to use a progressive approach to FDA drug and biologic approvals. This progressive approach would allow new treatments to reach narrow patient populations, such as those with illnesses for which no adequate therapy exists, prior to final approval. Then, as efficacy and safety are demonstrated by clinical trials, the approved population could be gradually expanded. BIO’s proposed progressive approach would apply only “to innovative products for unmet medical needs, significant advances to standard of care, targeted therapies, or those that have been approved by the European Medicines Agency or other mature regulatory agencies.” According to BIO’s executive vice president of health, Sara Radcliffe, this proposed progressive approach would combine many of the best characteristics of several drug approval pathways already used by the FDA and drugmakers.
In addition to the progressive approval approach, BIO’s proposals also include:
Empowering FDA to use a “weight-of evidence” approach to establish therapeutic effectiveness;
- Leveraging electronic health records to facilitate clinical research; and
- Create an “experimental space” within FDA to pilot promising new scientific and regulatory approached to therapy development.
While a few legislators, notably Representative Fred Upton (R-Michigan) and Senator Richard Burr (R-North Carolina), have voiced support for many elements of BIO’s proposals, BIO’s CEO Jim Greenwood stated that many of the proposals could be implemented without legislative action, yet legislation would expedite the implementation of the proposals.
PhRMA is said to be in the process of drafting proposals that include very similar elements. BIO suggests that the two organizations may collaborate on such proposals in the future.
While FDA currently has an investigational new drug application process that provides an avenue for an unapproved drug, or an approved drug for a new indication or a new patient population, to be further investigated or available in an emergency situation prior to approval, the existing process is by no means as broad and potentially far reaching as BIO’s proposed progressive approach. Will the agency and legislators be open to consider BIO’s proposal to expedite drug development approval process, even for a narrow population with no adequate therapy available? While a progressive drug approval process may stimulate and encourage innovation, would it have a substantial positive or negative impact on the safety and effectiveness of approved drugs?