Leading Biotechnology Organizations Praise Effort and Express Concerns with New Follow-on Biologics Proposal
Urge Congress to Strengthen Patient Safety Measures and Incentives for Further Therapeutic Innovation
WASHINGTON, D.C. (June 27, 2007) -- The Biologics Price Competition and Innovation Act of 2007 (BPCIA), reported out today by the Senate Health, Education, Labor and Pensions (HELP) Committee by voice vote, takes an important step toward establishing a pathway for approval of follow-on biologics, ensuring access for patients to life saving biologics, and protecting continued biomedical innovation, said the Biotechnology Industry Organization (BIO), the nation’s largest biotechnology trade association, and the Massachusetts Biotechnology Council (MBC), one of the largest state biotechnology trade associations. The organizations stated, however, that the bill also includes several provisions that cause concern for biotechnology pioneers, particularly regarding insufficient protection for patient safety and the patient-doctor relationship.
The legislation, sponsored by Senators Edward Kennedy (D-MA), Michael Enzi (R-WY), Hillary Clinton (D-NY), and Orrin Hatch (R-UT), is intended to create a pathway for the approval of follow-on biologics. Biologics are used to treat some of the world’s most challenging and devastating diseases including cancer, HIV/AIDS, and Parkinson’s disease along with a host of rare diseases. Follow-on biologic products will not be copies of, but rather will be similar to, life-saving, pioneering biologics. As a result, it is critical that any pathway to approve follow-on biologics recognize and address the differences between traditional generic drugs and follow-on products.
“Senators Kennedy, Enzi, Clinton and Hatch deserve credit for their hard work in crafting this complex legislation, and for the bipartisan support they have achieved for the bill. Biotechnology innovators share the goal of ensuring all patients have access to life-enhancing and life-saving biologics. We support the development of a pathway for the approval of follow-on biologics,” said BIO President and CEO Jim Greenwood. “Toward that goal, we will continue to work with Congress to make certain the legislation is improved to ensure it supports the principles we have outlined for a pathway to follow-on products, namely providing better protections for patient safety and the patient-doctor relationship.
“In addition, the patent litigation rules included in the bill must be revised to improve protections for the intellectual property rights of innovators, ensure timely resolution of all patent disputes and maintain incentives to develop future medical breakthroughs,” Greenwood stated.
Both organizations were pleased that the bill makes a strong statement about the importance of maintaining incentives for innovation necessary if the breakthrough therapies and cures for the diseases that plague mankind are to be developed and made available to patients in the years ahead.
“We believe the bill improves in important ways upon follow-on biologics legislation introduced earlier this year by recognizing the importance of data exclusivity for innovative products,” said Mark Robinson, MBC Chief Operating Officer. “This change is a critical step forward toward helping provide incentives for the development of new cures and treatments for serious illnesses.”
“Most of our member companies are in very nascent stages of development and need financial support in the form of large amounts of public and private capital investment. Data exclusivity helps provide needed predictability for the investments required for the more than a decade typically necessary to produce a single innovative biologic treatment,” said Greenwood. “We continue to believe, however, that a strong case has been made that 14 years of data exclusivity is the needed period required to strike the right balance between providing incentives for innovation and follow-on product entry, and to put biotechnology on a par with the up to 14 years of exclusivity provided through patent term protection for small molecule pharmaceuticals under the Hatch-Waxman Act. We look forward to working with Members of Congress as the bill moves forward to address this need.”
BIO and MBC added their concern that the proposal would allow follow-on products to be substituted for the original, fully-tested innovator product without the intervention of the health care professional who prescribes the biologic product, taking medical decision-making for individual patients out of the hands of doctors. As FDA itself recently stated, “As of today, the FDA has not determined how interchangeability can be established for complex proteins.” U.S. Health and Human Services Secretary Michael Leavitt expressed his strong concern in a letter he recently sent to the Committee regarding BPCIA, noting, “In light of the current scientific limitations on the ability to make determinations of interchangeability, and because it is critical to protect patient safety, the Administration believes that patients should not be switched form the innovator biological product to a follow-on biological product (or vice versa) without the express consent and advice of the patient’s physician, and legislation should not allow for determination of interchangeability at this time.”
“Biologics are large, complex organisms derived from living cells. Even small changes can render the biologics ineffective or even potentially harmful,” noted Greenwood. “To protect patient safety, Congress should ensure that patients are not given follow-on biologics unless expressly prescribed by a physician.”
Both organizations expressed further concerns about the adequacy of the BPCIA to protect patient safety, largely because of the broad discretion provided to the FDA in making approval determinations under the bill. They expressed concern about the scope of biological products potentially subject to follow-on biologics even if the science does not exist for a particular product class to adequately protect patient safety. The bill does not require the FDA to issue guidance after receiving public input from experts and stakeholders before approving a follow-on biologic. Nor does the bill require a follow-on applicant to conduct human clinical trials to test the follow-on product in humans. The standard for finding biosimilarity is weakened significantly in the bill because the information required to be submitted by the follow-on applicant can be waived by the FDA, and it is not clear from the language of the bill that critical requirements from the application – such as animal studies, assessment of immunogenicity, pharmacokinetics or pharmacodynamics, and standards for the manufacturing facility, among others – are required to be incorporated in the approval determination.
In addition, the organizations expressed significant concerns about the patent provisions included in the bill. The legislation would restrict the ability of innovators and third parties, such as universities which often serve as incubators for life-saving biotechnology innovations, to enforce their patents against follow-on manufacturers, while enabling follow-on products to enter the market in advance of the expiration of the innovator’s patent and prior to the conclusion of patent enforcement activity. Under the proposal, follow-on applicants also could limit the number of patents resolved prior to market entry to a single patent.
"Biologics often require years of research and investments of more than a billion dollars, on average, to go from idea to product," Robinson stated. "Biotechnology researchers must have some certainty that they can protect their investment in the development of new breakthrough therapies for a sufficient period of time in order to secure necessary financial resources. If follow-ons legislation were to fail to provide adequate protections, it could jeopardize the ability of biotechnology researchers to continue to innovate.”
“We thank the Senators for their efforts to develop a bill that seeks to expand access to safe, life-saving biologics while ensuring the ability of biotechnology pioneers to develop future biomedical breakthroughs. We look forward to working with the full Senate and the committees of jurisdiction in the House to address our concerns as this and other relevant legislation moves forward,” Greenwood and Robinson concluded.
Earlier this year, BIO released a set of key principles to guide the development of a pathway for the approval of follow-on biologics. These principles can be found at: http://bio.org/healthcare/followonbkg/Principles.asp. BIO also developed a detailed rationale supporting the need for substantial data exclusivity: http://www.bio.org/healthcare/followonbkg/FOBMarketExclusivity_050307.pdf.
Forty members of the Massachusetts Biotechnology Council provided the Senate committee with a letter sharing concerns regarding proposals to establish an approval pathway for follow-on legislation. The letter can be found at: http://massbio.org/attachments/MBC_Biologics_Letter_6-18-2007.pdf.
About Massachusetts Biotechnology Council
The Massachusetts Biotechnology Council (MBC) is the nation’s oldest biotechnology trade association. Founded in 1985, the MBC is a not-for-profit organization that represents and provides services and support for the Massachusetts biotechnology industry. The MBC is committed to advancing the development of critical new science, technology and medicines that benefit people worldwide. Representing over 500 biotechnology companies, academic institutions, research hospitals, and service organizations involved in life sciences and health care, the MBC works to advance policy and promote education, while providing member programs and events, industry information, and services.