National Consumers League

Health

NCL Health Issues

NCL forum explores biologics pathway

attention open in a new window

Access to safe and affordable medications has long been a priority of NCL. It is especially important now as consumers increasingly rely on pharmaceutical and biotechnology products to enhance and prolong their lives. Due both to increased utilization and product costs, the amount consumers spend on these therapeutics has increased significantly in the last decade.

 

The generic pharmaceutical market provides some financial relief for consumers and health care payers by enabling the production and sale of functionally equivalent products once the term of the brand patent has expired. Generic drugs contain the same active ingredients as their branded counterparts, and provide cost-savings for consumers and health care payers. Unlike traditional pharmaceutical drugs that are made from chemicals, biologic drugs are made from more complex proteins (live, organic matter). Examples of biologic drugs include insulin and some drugs used in treatments for cancer and multiple sclerosis. At present, there is no similar mechanism within the Food and Drug Administration for reviewing and approving biosimilar biologics. Given the complexities of the issue, Congress and the public continue to debate whether and how a pathway should be established to create biosimilar biologics.

NCL convened a forum on September 18, 2007 to provide policy makers and other stakeholder organizations with the opportunity to engage in an open and candid discussion on this issue. Over 40 participants representing broad interests, including consumer and patient advocacy groups, health care providers, government, industry, and others, attended the half day forum. The gathering featured two panels, plus a legislative overview and concluded with a lunch speaker.

A National Consumers League Stakeholder Forum
Biologics: The Pathway to Biosimilar Products?

September 18, 2007
AFL-CIO Building
Presidents Room, First Floor
815 16th Street, NW
Washington, DC

Forum Report

Access to safe and affordable medications has long been a priority of the National Consumers League (NCL). It is especially important now as consumers increasingly rely on pharmaceutical and biotechnology products to enhance and prolong their lives. Due both to increased utilization and product costs, the amount consumers spend on these therapeutics has increased significantly in the last decade.

The generic pharmaceutical market provides some financial relief for consumers and health care payers by enabling the production and sale of functionally equivalent products once the term of the brand patent has expired. Generic drugs contain the same active ingredients as their branded counterparts, and provide cost-savings for consumers and health care payers. Unlike traditional pharmaceutical drugs that are made from chemicals, biologic drugs are made from more complex proteins (live, organic matter). Examples of biologic drugs include insulin and some drugs used in treatments for cancer and multiple sclerosis. At present, there is no similar mechanism within the Food and Drug Administration (FDA) for reviewing and approving biosimilar biologics. Given the complexities of the issue, Congress and the public continue to debate whether and how a pathway should be established to create biosimilar biologics.

NCL convened a forum on September 18, 2007 to provide policy makers and other stakeholder organizations with the opportunity to engage in an open and candid discussion on this issue. Over 40 participants representing broad interests, including consumer and patient advocacy groups, health care providers, government, industry, and others, attended the half day forum. The gathering featured two panels, plus a legislative overview and concluded with a lunch speaker.

 

Opening – Status of Legislation on Biosimilar Biologics

Linda Golodner, President of NCL, opened the forum by providing a brief background on the issue of biosimilar(also sometimes called generic or follow-on) biotechnology products. Two representatives from congressional offices then gave an overview of proposed legislation regarding biosimilar biologics:

Nick Shipley, Legislative Director, Office of Representative Jay Inslee (D-Wa)

Ann Witt, Health Counsel, Office of Representative Henry Waxman (D-Ca)

Shipley discussed his work on the development and introduction of H.R. 1956, the Patient Protection and Innovative Biologic Medicines Act of 2007. H.R. 1956 establishes a pathway for FDA approval of biosimilar biologics using an approval process based on the European Union (EU) model. The legislation calls for a guidance system involving FDA, provides incentives for innovation, mandates clinical trials and post-marketing follow-up, and requires unique names for biosimilar biologics from their branded counterparts. The bill allows for interchangeability, or the substitution of a biosimilar biologic product for its branded counterpart. In addition, the bill allows a 14-year window of exclusivity, or the length of time that the manufacturer of the biologic product has exclusive rights to that product in the market (no generic versions of that product can be sold during that period). This 14-year window of exclusivity would start after the patent had been issued.

Witt discussed Waxman’s bill, H.R. 1038, the Access to Life-Saving Medicine Act, and the importance of establishing a pathway for biosimilar biologics based on abbreviated clinical trials (shortened and less extensive trials compared to full-length trials) to prove bioequivalence between biosimilars and their branded counterparts. Bioequivalence means that the active ingredients of two products are the same, and the rate and extent of absorption into the body are the same. Waxman’s bill details when the abbreviated trials are appropriate and gives FDA the discretion to make such decisions. The bill does not mandate interchangeability, but it does not rule it out either. Interchangeability could be established for some simpler drugs, and it could be expanded over time to include other drugs as research evolves and progresses. The Waxman bill calls for five years of exclusivity and allows for extended approval past that period, but no more than 14 years total.

The congressional speakers sparked debate on several issues, including exclusivity and safety. Highlights of the discussion included the following:

• Exclusivity – Some argued that allowing for only five years of exclusivity under the Waxman bill would not give innovator drug companies time to recuperate from the costly process of developing a drug. However, Witt noted that she has not heard of any innovator drug companies saying that five years was inadequate. Witt believes that the innovator society would continue to thrive after the legislation is passed.

• Safety issues – Witt warned against excessive procedural protections as they could get lengthy and time-consuming, potentially causing the biosimilar biologics to be less technically advanced by the time they finally enter the market. However, others countered that extensive procedural protections are needed given the level of uncertainty that comes with biosimliar biologics, and the fact that these products are only similar and not identical, to their branded counterparts.

Panel 1 – Issues around Developing a Biosimilar Biologics Market

Presentations and discussion in Panel 1 focused on the issues around developing a biosimilar biologics market. Speakers were:

Roger Williams, Executive Vice President and Chief Executive Officer, United States Pharmacopeia (USP)

William Schultz, Partner, Zuckerman Spaeder LLP

Erika Lietzan, Partner, Covington & Burling LLP

Williams described the qualifications under the monograph system that indicate bioequivalence between two pharmaceutical drug products and how that system could be utilized in the development of a biosimilar biologics market. Schultz discussed four issues concerning possible legislation on biolsimilar biologics: (1) what the legislation would entail for establishing a pathway, (2) interchangeability, (3) exclusivity, and (4) the interrelationship between exclusivity and interchangeability. Lietzan discussed the biosimilar biologics market in the EU, including lessons learned from the development of the market and what characteristics of the EU system may and may not successfully translate to an American system.

Discussion:

The discussion following the panel presentations focused on the following questions:

1) Are full clinical trials needed to ensure that biosimilar biologics are equivalent to their branded counterparts, or can abbreviated trials be used?

Some forum participants supported the use of abbreviated clinical trials for biosimilar biologics, while others felt that such trials would be inadequate for the approval of biosimilar biologics. Those who supported abbreviated trials felt that the approval of biosimilar biologics should be handled similarly to that of traditional pharmaceutical biologics. FDA could be given the discretion to determine on a case-by-case basis whether each individual biosimilar biologic should be allowed to undergo abbreviated trials based on its level of comparability and sameness (questions of equivalence) to its branded counterpart. Others felt that biosimilar biologics cannot be treated in the same fashion as traditional pharmaceutical generics because of their complex nature. They argued that abbreviated trials would not ensure or prove the safety of a product but would only demonstrate an ability or inability to detect anything during a clinical trial.

 

2) Should biosimilars have names that are unique and distinct from their branded counterparts to allow differentiation between the two classes of drugs?

Some noted that the ability to detect differences between biosimilars and their branded counterparts supported the need for unique names. Several agreed that health care consumers deserve the right to know if they are taking the branded version or the biosimilar version of a drug. It was suggested that FDA should be involved in the labeling of biosimilar products so that the labels indicate to what degree individual biosimilar products are interchangeable with their branded counterparts. If biosimilars are partly being developed to provide competition and cost-savings, health care consumers should be allowed the choice between taking a biosimilar product or its branded counterpart. However, having a choice will most likely be out of reach for most health care consumers as many will only be able to afford the biosimilar product. It was noted that certain traditional pharmaceutical drugs are deemed interchangeable, but the patient has a role in deciding which drug to take. Therefore, it may be appropriate to handle biologics in the same manner.

3) How should post-market surveillance of biosimilars be handled?

In regard to what can be learned from the EU in terms of post-market surveillance, Lietzan noted that it is likely that states will respond to legislation with their own plans for monitoring post-market surveillance. The EU is ahead of the United States in risk management. The burden of risk management and post-market surveillance is dependent upon each individual drug and what types of risks are present for each.

Panel 2 – Economic and Practical Implications of Biosimilar Biologics

Presentations and discussion in Panel 2 focused on exploring the economic and practical implications of biosimilar biologics for health care payers, consumers, health care providers, and society. Speakers were:

David Borenstein, George Washington University Medical Center, American College of Rheumatology

Steven Miller, Chief Medical Officer, Express Scripts

Michael Brodeur, Head of Formulary Development & Pharmacy Clinical Policies, Aetna

Henry Grabowski, Professor of Economics, Duke University

Borenstein discussed how the development of biosimilar biologics could affect health care providers and consumers - greater choice and availability of biologic drugs could lead to better health outcomes, as well as cost-savings from decreased need for medical attention. However, with the development of this new biosimilar biologics market it would be crucial for health care providers to fully inform patients about the meaning of bioequivalence and comparability, as well as disclose any potential risks associated with the new drugs to ensure the best and most appropriate utilization of the new drugs.

Miller and Grabowski discussed the economic implications of developing a biosimilar biologics market. Miller explained that as America’s population and health expenditures continue to steadily grow, and as the use of biologic drugs becomes more commonplace, the cost-savings and availability of drugs created through the development of a biosimilar biologic market will be both substantial and necessary. Grabowski discussed the potential cost-savings for the developers of biosimilars, how data exclusivity complements incentives for patents, and breakeven times for new pharmaceuticals in money invested in research and development.

Brodeur explained the necessity to balance safety, efficacy, and cost in developing a biosimilar biologics market. He discussed the need for competition within drug classes to provide greater choice and cost-savings to patients, especially since several diseases treated by biologics target minority and elderly populations who may also be lower-income populations.

Respondents:

Shawn O’Neail, National Multiple Sclerosis Society (NMSS), discussed the need for biosimilar biologics to enter the market to provide more cost-effective alternatives since most of the drugs used to treat multiple sclerosis are currently biologics and are very expensive. “No matter how good the drug is supposed to be, if the patients cannot afford it, the drug is no good to them.” High drug prices often force patients to either make sacrifices in order to pay for their medications or to sacrifice their drug treatments entirely.

Ruth Hoffman, Candlelighters Childhood Cancer Foundation, discussed the need for innovation in the field of biologics. The Candlelighters Childhood Cancer Foundation advocates for the development of new biologic drugs to be used in the treatment of pediatric cancer; they do not want any newly proposed bills to hinder innovation and deprive the industry of incentives. While patients would not have to pay so much for biosimilar biologics, new and better biologic drugs for pediatric cancer need to be developed before generic versions of those drugs can be created.

Discussion:

Discussion following the second panel focused on the potential cost-savings of a biosimilar biologic market. Several participants noted that the introduction of biosimilar biologics to the marketplace may spur moderate savings at the beginning, but those moderate savings will evolve into much greater savings over time. Miller noted that the annual savings for traditional pharmaceutical generics have grown from a trivial amount in the initial decade to billions of dollars in the long-run, and the same will probably be true for biosimilar biologics. The market needs a safe pathway in place, and then the progression and savings from biosimilar biologics will evolve accordingly. Some pointed out that the savings will free up money for greater investment in R&D. Some argued that it is imperative to create and foster competition, instead of price controls, in order to make drug prices more affordable for the public. Others noted that drugs should be able to extend their exclusivity based on their levels of benefit and effectiveness (not just cost-savings) among the patient population.

 

Lunch Speaker – Susan Dentzer

Susan Dentzer, health correspondent for The NewsHour with Jim Lehrer, described her experiences as a health journalist, including the challenges and complexities surrounding biosimilar biologics that keep stakeholders and the American public from reaching a consensus on the issue. She emphasized the need to work together collectively to inform the public, to explain the pertinent issues, and to help them understand that the debate on biosimilar biologics is one where there are no clear rights or wrongs.