On day two of AAM’s October meeting, a panel including Jim Polli, Ph.D., Professor at the University of Maryland, Anna Scwenderman, Ph.D., Professor at the University of Michigan College of Pharmacy, and Aaron Josephson, Senior Director at Teva Pharmaceuticals, discussed the International Generic and Biosimilar Medicines Association’s (IGBA) project update on Single Global Development for Generic and Biosimilar Medicines. Given that efforts toward international harmonization are in high gear and the need for conserving resources at companies is becoming increasingly important, this could not have been a timelier update

Also mentioned during this session were motivations for a single global development program:

  • The high cost of the studies that need to support approval in multiple jurisdictions; and
  • The exceptionally high cost for obtaining sufficient quantity of a reference product necessary as the standard against which a biosimilar’s “highly similar” standard must be demonstrated.

The goal of single global development is predicated on the fact that if medicines developed in one country are based on good science, then there is no reason to duplicate studies and generate new data to support approval in another country. The group expressed the belief that, while this would benefit all patients, the development of orphan drugs would likely benefit most from global development harmonization. For orphan drugs and especially ultra orphan drugs, the development costs for country-by-country approval would likely preclude their development in many countries.

While it is hard to argue with principles of global development, there are regulatory requirements that must be overcome. For example, except in very unusual situations, the FDA does not permit the use of foreign-approved reference products for use as comparators for generic ANDA products or 505(b)(2) applications. The same is mostly true for biosimilar applications; however, there are some exceptions as follows:

  • The FDA may permit the use of foreign reference products under certain conditions.
  • The foreign product must be approved by a regulatory authority recognized by the FDA.
  • The applicant must demonstrate that the foreign product is highly similar to the proposed biosimilar.
  • Data from the foreign reference product can support the biosimilar application.
  • The FDA may require additional data to ensure safety and efficacy.

It is recommended that the FDA be engaged early in the development process for clarity on its stance regarding the use of a foreign reference product in a particular study or set of studies. Also, there may also be unique issues associated with patent protection or exclusivity that could come into play so be sure to consult appropriate regulatory counsel.

In another session on legislative initiatives, an august panel made up of Kimberlee Trzeciak, Senior Vice President at Capitol Hill Consulting Group, Kurt Karst, JD, Director at Hyman Phelps & McNamara, P.C., Brian McCormick, JD, Vice President, Chief Regulatory Counsel, and Head of Global Regulatory Policy at Teva Pharmaceuticals, and Eva Temkin, JD, Partner at Arnold & Porter, ran through a number of legislative developments and what to expect in potential year-end packages. They discussed a number of potential game-changing legislations including:

  • H.R.1843/S.1302, the Increasing Transparency in Generic Drug Applications Act, which is designed to, once and for all, fix the Q1-Q2 issue associated with generic drugs.
  • S.1954/H.R.5526, the Biosimilar Red Tape Elimination Act, which would amend the PHS Act to state that all biosimilars, upon licensure, shall be deemed interchangeable with their respective reference product. Among other things, it would revise the sections of the PHS Act and the BPCIA that would memorialize the FDA decision regarding interchangeability of biosimilars once approved.
  • S.1954/H.R.5526, the Biosimilar Red Tape Elimination Act – PREA requirements that would address the problem associated with the application of PREA to biosimilars. “Under FDC Act 505B(l), a biosimilar product that has not been determined to be interchangeable with the reference product is considered to have a “new active ingredient” for PREA purposes, and a pediatric assessment is generally required unless waived or deferred or inapplicable.” “In eliminating the distinction between highly similar biosimilars and interchangeable biosimilar biological products, the Biosimilar Red Tape Elimination Act also eliminates the need for companies to conduct expensive and time-consuming PREA studies.” (There is an exception here that applies in certain circumstances, however.)

There were other issues discussed but I don’t want to blow your mind. I will say that, based on the current length of the government shutdown, panelists were somewhat skeptical that many of the topics discussed would be included in any legislation to get the government back open, but there is a chance for passage of some of these legislative initiatives once things in Washington cool down after the government finally reopens and an appropriations package is passed. However, short term passage of these bipartisan bills may be more of a hope than a sure thing.