BIO President Addresses FDA on PDUFA

Washington, D.C. (September 18, 2000) Carl B. Feldbaum, president of the Biotechnology Industry Organization (BIO), issued the following statement on the reauthorization of the Prescription Drug User Fee Act (PDUFA) at an FDA public meeting September 15, 2000:

"I feel like I’m here wearing two hats today. I’m president of the Biotechnology Industry Organization, but I am also a cancer survivor. If it were not for a diagnostic test, developed by a biotech company and approved by the FDA, my cancer might have gone undetected for years. Instead it was caught early, and now I am just fine.

"Getting more and more effective diagnostics and drugs to patients is what the biotech industry is all about. To accomplish that, our industry works in a rather rare, professional relationship with the FDA.

"Let me take a shot at better defining that relationship. First, let me say what it is not. It is not, as some have called it, a partnership nor is it necessarily congenial or even neighborly. But it is also not antagonistic, spiteful or hierarchical. What it is, is a relationship essentially between peers, strictly professional, arms-length, civil but also attentive, approachable, responsive. Cordial, but hardly companionable.

"BIO and the FDA have worked together to produce the world’s safest and most effective new medicines for patients. During the 1997 negotiations over the FDA Modernization Act, BIO worked with Congress and the FDA to implement meaningful reforms that, to the amazement of many, passed the Congress in unanimous votes. BIO was then an advocate on behalf of the FDA, and we defended the agency’s critical role in the drug development process from some real radicals who said, We don’t need an FDA; let the markets decide what works. What rubbish.

"No other nation has an agency that compares to the FDA, and no other consumers in the world have as much confidence in the safety and efficacy of its food and drugs as citizens of the United States. Right now the furor in the United Kingdom and continental Europe over biotech foods, or as they call them GMO foods is, in some part, attributable to the fact that while in the United States, citizens are skeptical about the government in general, we do trust the FDA to protect the public health. That is a long and hard-earned trust.

"Most European citizens, however government-centric or even socialist they may be, do not share such trust in any government agency devoted to protecting their public health, and they have been unnerved by their experience with tainted blood supplies and widely perceived government bungling over mad cow disease.

"Our rather strict FDA-biotech relationship has resulted in development of nearly 100 biotech drugs and vaccines over the last 25 years. These medicines have now helped more than 250 million people worldwide. In the next couple years, working together and apart, we can do even better.

"Because of flat federal appropriations over the past several years, the true operating budget of the FDA has shrunk dramatically just as biotech companies are coming online with a tsunami of new treatments for formerly intractable diseases, many suffered by the Medicare population. The agency actually has less money now than it had in 1993 for activities other than those funded by user fees or covered by tobacco and food safety programs.

"Of necessity, BIO’s first order of business this year was to lead the charge for greater FDA appropriations. We organized a coalition of groups representing many of the FDA-regulated industries. The effort was successful with both the House and Senate appropriating much-needed increases in funding.

"The second way we walk the walk: BIO consistently defends the FDA’s role as the primary regulator in all drug development areas, including gene therapy and stem cell research.

"Third, our staunch support for PDUFA is perhaps the best evidence of our commitment to a strong, independent science-competent FDA.

"Regarding PDUFA, which by the way is the worst Washington acronym (without the corruption) since CREEP (President Nixon’s Committee to Re-Elect the President). But regarding PDUFA, BIO hopes to use the next reauthorization cycle of negotiations as a springboard to explain to Congress why the FDA needs more appropriated resources.

"Biotechnology is a very highly regulated industry, and we tell Congress, that a highly competent, science-proficient FDA is essential to the development of the U.S. biotech industry. An industry that is by far the global leader, not only in innovation, but also in new jobs in congressional districts in all 50 states. We want a rigorous review process that is science-based and is more predictable. Predictability is particularly important for many of our small companies, whose value and ability to continue to fund their research, since they often have no revenues, certainly no profits often hangs on every nuance, every syllable the FDA utters publicly.

"We at BIO are currently developing our recommendations for the potential reauthorization of the user fee act.Based on initial feedback from our mostly small biotech companies, I can tell you that we have a few main concerns:

1. Our companies need a more predictable review process that includes better communications and more timely interaction with FDA reviewers.

2. Our members report that FDA is unwilling to agree to protocol specifications in writing. We believe this is contrary to the spirit of FDAMA, which requires collaboration in protocol development between our companies and the agency.

3. Action must be taken on widely perceived slow, indeed glacial, implementation by FDA of FDAMA provisions designed to guide our companies as they disseminate health-care economic information and certain scientific information.

"We at BIO look forward to working with you in our usual plain-spoken way, always thoroughly, carefully dealing with our shared responsibilities. First, to ensure the safety and efficacy of our products; and second, to borrow an simple expression from the Supreme Court of the United States in the landmark case, Brown v. Board of Education, to make sure we work together with all deliberate speed to get our new medicines to the patients who need them.Thank you."

BIO represents more than 900 companies, academic institutions and biotech centers in all 50 states and 26 other nations. BIO members are involved in the research and development of health-care, agricultural, industrial and environmental biotechnology products.