H.R. 1548 better than alternatives on new drug class

July 8, 2009
Op-Ed

The Hill

H.R. 1548 better than alternatives on new drug class

Posted: 07/08/09 12:06 PM [ET]

Thefield of biotechnology is the future of medicine. Today we're justscratching the surface of the potential for biology-drivenbreakthroughs that hold the promise of treating cancer, diabetes,arthritis, Alzheimer's and other deadly and debilitating diseases. Theinnovation and discovery in this vital field will transform medicine inthe coming decades, but these biologic treatments are expensive. Tomeet our national priority of improving health while reducing costs, wemust look for ways to deliver safe and effective, lower-pricealternatives.

Comprehensive healthcare reform offers a timely and appropriate vehicle in which to accomplish this.

One successful model for reform was the 1984 Hatch-Waxman Act.Introducing small-molecule generic drugs ushered in a new era ofcompetition and helped consumers afford innovative pharmaceuticals. Thesame concept inspires us to create a marketplace for follow-on versionsof biotechnology products. Our challenge is to replicate the success ofthe generic drugs marketplace, while developing a legal and scientificframework that addresses the complex research, efficacy and patientsafety variables that make biologics fundamentally different.

Manyof us take a prescription or over-the-counter drug frequently. Eachtime we reach for a pill, we expect the same safety and effectiveness,whether using a brand-name or generic drug. The small-molecule chemicalcompounds of traditional drugs are ideal for replication as generics.These products have well-defined structures that can be thoroughlycharacterized and copied, and generic drugs are chemically identical tothe reference products they copy. Doctors and patients can expect thatgenerics will have the same properties, the same efficacy, and the samesafety characteristics as the innovative product they copy.

Biologicalproducts are fundamentally different. A biologic is a large, complexmolecule, which is "grown" in living systems such as a microorganism, aplant or animal cell. The resulting protein is unique to the cell linesand the specific process used to produce it, and even slightdifferences in the manufacturing of a biologic can alter its nature. Asa result, biologics are difficult, sometimes impossible tocharacterize, and laboratory analysis of the finished product isinsufficient to ensure its safety and efficacy.

Even if abiosimilar is proven to be safe and effective, it will likely stillhave different properties than the original innovative product. Theremay be differences in dosing, different side effects or safetyprofiles, and differences in effectiveness for certain diseases orpatient groups.

The fundamental uniqueness and complexity ofbiologics demands more rigorous regulation than what exists today forgeneric small molecule drugs, and new legal considerations.

Ihave introduced legislation, H.R. 1548, the Pathway for BiosimilarsAct, which is based on sound science and does more than any otherproposal to protect patient safety. Under my bill the FDA retainsultimate authority to make critical determinations related to testingand interchangeability, but the agency must solicit views andinformation from experts and stakeholders before waiving key patientsafeguards. My legislation ensures that physicians make criticaldecisions about appropriate treatments - not insurance companies,pharmacy benefit managers, or pharmacists.  These standards areconsistent with recommendations made by the FDA's Chief Scientist.

Biologicsare expensive and risky to develop. A soon-to-be-released studysponsored by the National Venture Capital Association analyzed therelative costs for investors in biotechnology and found that the "costof capital" for start-up biotech companies is more than double thecosts that other companies must pay. These costs stem from longdevelopmental timelines of typically 10 years or more, extraordinarylevels of risk (fewer than 1 percent of biologics make it to market),and the large amounts of capital required to support development.

Topreserve existing incentives for investment and innovation the Pathwayfor Biosimilars Act provides a data exclusivity period equivalent topatent protections for small molecules. The Congressional Budget Officehas determined that 11.5 years is the average length of time that drugsare marketed under patent. In other words, innovative drugs andbiologics typically stay on the market for about 12 years before facingcompetition. My legislation maintains this level of protection forbiologics.

Small-molecule drug manufacturers rely on patent protection torecoup the hundreds of millions of dollars it takes to bring a drug tomarket. Valid patents are very effective in preventing generic patentinfringement because generic drugs are required by law to be chemicallyidentical to innovative products. However, biosimilars will obviouslybe only "similar" to the innovative products they compete with, givingbiotechnology companies a greater challenge to prove patentinfringement and less legal protection for massive investments.

Today,the ability to work around patents has limited appeal to a prospectivebiosimilar producer. Innovative biotechnology companies are assuredthat the costly clinical trial results and data that they developduring their approval process cannot be used by competitors to secureapproval and enter the market, even if their patents do not prevententry. In effect innovators now have "infinite" data protection, whichallows for competition but does not permit "free riding" on their data.

Mylegislation proposes allowing competitors access to this data and ashortcut into the market, but also preserves the existing incentivesfor innovators by maintaining a 12-year period of concurrent dataprotection as a "backstop" to existing patent protections. The billalso sets forth a process to resolve all patent litigation prior tolaunch of biosimilars, providing the opportunity to resolve importantintellectual property issues and certainty to all parties withoutmarket disruptions. Unlike any other proposal, my bill also provides anopportunity for third parties (universities, medical centers) toprotect their rights.

I've been joined by a diverse group ofmore than 100 bipartisan cosponsors in the House, including leadcosponsors Jay Inslee (D-Wash.) and Joe Barton (R-Texas). Our bill isthe only legislation endorsed by the Association of AmericanUniversities, the National Venture Capital Association, theBiotechnology Industry Organization, the governors of four states, anda wide array of patient and industry groups, including the AIDSInstitute and the ALS Association.

The time has come toprovide a regulatory pathway for safe - and cheaper - versions of thebiologics that save thousands of lives and improve the quality of lifefor countless more.


Eshoo is a member of the House Energy and Commerce Committee's health subcommittee.