Industry has spent the past two days wrapped around the biosimilars hearing held at FDA headquarters in Rockville, MD, to discuss the implementation and regulation of a follow-on biologics pathway. The pathway was cleared via President Obama’s Biologics Price Competition and Innovation Act of 2009, which falls under the 2010 approved healthcare reform legislation. Trade associations PhRMA, BIO, and GPhA had quite a bit to say during the hearing about their thoughts on the pathway and how it should be pursued.

Marie A. Vodicka, PhD, associate vice-president of scientific and regulatory affairs for PhRMA, provided PhRMA’s perspective. After reviewing the association’s key principles meant to guide FDA in its implementation of the pathway, including a suggestion to use EMA’s approach (i.e., issuing industry guidance after extensive public consultation) as a model, Vodicka gave more specific views on biosimilarity; interchangeability; pharmacovigilance, naming and labeling to promote patient safety; and exclusivity. (See below for details on these topics from each industry group.)

Rasmus Rojkjaer, MD, PhD, vice-president and head of Global Biologics Research & Development for Mylan, represented GPhA at the hearing. His introduction noted that “it will take much more to create price competition for the $31.3 billion of blockbuster biologics that will lose data exclusivity on or before 2018” and that the “new pathway is full of ‘landmines’ that will hamper the true intent of the law.” He pointed out these three barriers, for example: 1) The data exclusivity period of the innovator biologic – which will block a biogeneric approval but not an alternative biologic filed as a BLA; 2) Uncertainty surrounding the information required for a biogeneric application; and 3) The expense of developing a biogeneric. Rojkjaer also briefly discussed GPhA’s support for biogeneric user fees and the use of foreign reference products.

Sara Radcliffe, executive vice-president for Health at BIO, spoke to the panel on behalf of her organization. In addition to the key topics discussed by all groups (see below), she presented BIO’s ideas for maintaining incentives for innovation such as user fees and restricting reference product comparisons to only publicly available information. Radcliffe also noted that BIO supports the issuance of guidance governing the review and approval of biosimilars.

Biosimilarity

PhRMA: “FDA should, at a minimum, consider the following factors before deciding to waive the clinical data requirement for any indication: molecular mechanism of action; disease mechanism of action; and disease state of the patients, such as, immunocompetent or immuno-suppressed.”

GPhA: Rojkjaer argued that “any clinical development required must be needs-based and data driven, taking into account the degree of similarity demonstrated earlier in development. If the biogeneric has been demonstrated to be highly similar to the reference product, the established safety, purity and potency profile of the reference product can be relied upon without mandating unnecessary duplicative testing.”

BIO: “Comparative analytical, nonclinical, and clinical studies are necessary to protect patient safety… The scope of clinical studies depends on factors including the findings and limitations of analytical and nonclinical studies, and the state of public knowledge.” Of note, both BIO and PhRMA noted support for a step-wise system for determinin biosimilarity.

Interchangeability
PhRMA: “Only FDA should make interchangeability decisions” including its affect on public health.

GPhA: “FDA must be exceedingly cautious in elevating the interchangeability standard to a level that is unattainable and thereby creating inconsistency with the interchangeability standard applied over the past 14 years to highly similar branded biological products.”

BIO: “Must demonstrate that alternating between the two products in an individual patient does not negatively impact efficacy or safety”

Naming and labeling
PhRMA: “Each biologic product should have a unique name. The simplest way to achieve this is to require unique non-proprietary names.” Vodicka also pointed out that each interchangeable biologic should have its own labeling.

GPhA: “Biogenerics should be given the same generic name – whether International Nonproprietary Name (INN) or United States Adopted Name (USAN) – as the reference product. Otherwise, a finding of interchangeability or biosimilarity will count for very little among healthcare providers…”

BIO: “Pharmacovigilance activities must be guided by an understanding of the unique nature of biologics/biosimilars.”

Exclusivity
PhRMA: “In determining which products are eligible for the 12-year data protection period, referred to as the exclusivity period in the Federal Register notice, FDA should adhere to the wording of the statute. Any difference in structure of the product accompanied by data demonstrating differences in safety, purity or potency from a previous product warrant a product receiving its own 12-year exclusivity period. We note that notwithstanding the phrasing of the question in the Federal Register notice, this is not a second period.”

BIO: “There is no “second” 12-year period of marketing exclusivity for any biologic, under any circumstances.”

The quotes contained herein are based on documents provided to Pharmaceutical Technology from the organizations. A full transcript of the hearing is supposed to be uploaded on this site once it becomes available.