107th Congress

The Human Genome Project: Future Implications for Patients -- Hearing Before the Senate Biotechnology Caucus -- March 27, 2001

Members Present

Senators Christopher Dodd (D-CT) and Tim Hutchinson (R-AR), Co-chairs, Senate Biotechnology Caucus.

Francis Collins, Director, NHGRI: Suzanne Doggett, National Board Member, Huntington's Disease Society of America; Judi Hirshfield-Bartek, Board Member, National Breast Cancer Coalition; Gualberto Ruano, Chief Executive Officer, Genaissance Pharmaceuticals.

Summary

Senator Dodd opened the forum and thanked Senator Hutchinson for establishing the bipartisan Senate Biotechnology Caucus. He stated that the public has been bombarded with information as a result of the revolution of biotechnology, including genetically-modified food and cloning. He commented that the science of genetics has outpaced the law and that people are susceptible to insurance and employment discrimination based on their genetic information. He mentioned the lawsuit against the Burlington Northern Santa Fe Railroad for requiring some of their employees to take a genetic test after filing workers' compensation claims for carpal tunnel syndrome. Senator Dodd expressed his support for S. 318, the Genetic Nondiscrimination in Health Insurance and Employment Act, sponsored by Senator Tom Daschle (D-SD). Senator Hutchinson stated that he was encouraged by the work of those like Dr. Collins and other researchers to further the understanding of genetics. He echoed Senator Dodd's concerns about the pace of the science and how legislation is needed to protect people from genetic discrimination.

Dr. Collins stated that he was pleased to have the opportunity to speak with Senators Dodd and Hutchinson about the medical promise of genetic research. He commented that all diseases have some genetic component and that all of us have "glitches" in our genetic code. Dr. Collins stated that having a defective gene, save for a few genes like the Huntington's gene, does not mean the person will definitely develop disease. The development of disease depends on the interplay between multiple genetic mutations and the environment. He mentioned the February publication in magazine of the analysis of the human genome and presented the Senators with a CD Rom of the DNA sequence and an education packet.

Ms. Doggett described how her grandfather and mother died of Huntington's disease and explained why fewer than 5 percent of people at risk do not want to be tested. She said that people should not have to fear genetic discrimination and that no one should be forced to take a genetic test. She also commented that information produced in clinical trials should be kept in the highest confidence.

Ms. Hirshfield-Bartek made four points to Senators Dodd and Hutchinson:

Senator Hutchinson asked Dr. Ruano if any additional legislation would be beneficial. Dr. Ruano encouraged Congress to look at patent issues. He stated that the Commerce and Trade Office is faced with new claims. He urged the Senators to remember that the future of commerce depends on intellectual property. Dr. Collins added that he agreed intellectual property rights are crucial. However, he explained that in order to receive a patent for a gene, the applicant must know its uses and be able to describe what the gene does in detail. He mentioned that one problem with issuing so many patents on genes is that scientists studying a multitude of genes might face a complicated myriad of licensing agreements. He mentioned that the U.S. Patent Office issued guidelines which specified that in order for a gene to be patentable, the applicant has to demonstrate the utility of the gene. He stressed that not only are patent issues important, but also the licensing agreements. He also noted that if only one laboratory is doing the testing for a genetic disorder, there is no incentive to drive the cost down.

Senator Hutchinson asked about the length of time it takes from the time a gene is discovered to the development of a drug to treat a genetic disorder. Dr. Collins explained that with the database complete a gene can be easily identified. However, it takes time to translate having the gene sequenced into developing a drug that will interfere with the process of the defective gene. He then briefly outlined the FDA approval process, including the four phases of clinical trials that must be completed to test the safety and efficacy of the drug. Dr. Collins explained how all drug companies now have a genomics division. He commented that the biotechnology industry has "grown up" and is fueled by genomics. He also stressed the importance of private/public partnerships.

Senator Hutchinson closed the hearing by asking Ms. Doggett for any advice for the caucus. She lightheartedly responded that the funding of NIH should be tripled. She added that people need to be confident that their genetic information will be kept in confidence and not used against them.

Hirshfield-Bartek made four points to Senators Dodd and Hutchinson:

While the current genetic code may offer women information about their life-time risk of developing breast cancer, it cannot prevent or cure the disease. Taking advantage of this technology may mean that an individual becomes vulnerable to discrimination based on her genetic information. The fear of the potential discrimination often interferes with the delivery of medical care and hampers advancement in medical research. Federal protections against genetic discrimination are absolutely necessary to ensure future benefits of genetic science.

Dr. Ruano described his company's study of pharmacogenetics and their work to develop drugs that can be tailored based on an individual's genetic make-up.

Senator Hutchinson asked Ms. Hirshfield-Bartek if legislation were passed to prevent genetic discrimination, would her concerns be addressed. She answered in the affirmative and added that the fear of discrimination is so overwhelming and legislation would help with the recruitment of patients for clinical trials. Senator Hutchinson asked Dr. Ruano if any additional legislation would be beneficial. Dr. Ruano encouraged Congress to look at patent issues. He stated that the Commerce and Trade Office is faced with new claims. He urged the Senators to remember that the future of commerce depends on intellectual property. Dr. Collins added that he agreed intellectual property rights are crucial. However, he explained that in order to receive a patent for a gene, the applicant must know its uses and be able to describe what the gene does in detail. He mentioned that one problem with issuing so many patents on genes is that scientists studying a multitude of genes might face a complicated myriad of licensing agreements. He mentioned that the U.S. Patent Office issued guidelines which specified that in order for a gene to be patentable, the applicant has to demonstrate the utility of the gene. He stressed that not only are patent issues important, but also the licensing agreements. He also noted that if only one laboratory is doing the testing for a genetic disorder, there is no incentive to drive the cost down. Senator Hutchinson asked about the length of time it takes from the time a gene is discovered to the development of a drug to treat a genetic disorder. Dr. Collins explained that with the database complete a gene can be easily identified. However, it takes time to translate having the gene sequenced into developing a drug that will interfere with the process of the defective gene. He then briefly outlined the FDA approval process, including the four phases of clinical trials that must be completed to test the safety and efficacy of the drug. Dr. Collins explained how all drug companies now have a genomics division. He commented that the biotechnology industry has "grown up" and is fueled by genomics. He also stressed the importance of private/public partnerships.

Senator Hutchinson closed the hearing by asking Ms. Doggett for any advice for the caucus. She lightheartedly responded that the funding of NIH should be tripled. She added that people need to be confident that their genetic information will be kept in confidence and not used against them.

Prepared by OLPA