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More Information on the Drug Approval Process

Comments Concerning Disclosure of Information on Investigational Products

April 18, 2001

Public Citizen's Health Research Group's Comments On
Availability for Public Disclosure and Submission to FDA for Public Disclosure of Certain Data and Information Related to Human Gene Therapy or Xenotransplantation Proposed Rule

Docket No. 00N-0989

Public Citizen unequivocally supports the ethical principles contained in this Food and Drug Administration (FDA) proposed rule that summary safety and effectiveness data, in addition to other information, must be made publicly available during pre-market clinical testing of therapeutic interventions being developed for commercial purposes to "ensure that individual subjects understand the experimental nature of the procedures and their known and unknown risks and burdens." In addition, this proposed rule would provide a mechanism for "public education, informed discussion and participation that can form a foundation for safe and ethical research."[1] We also strongly agree with this principle of the proposed rule.

Unfortunately, the FDA has chosen to limit its proposal for disclosure to only two interventions: 1.) gene therapy; and 2.) the transplantation of non-human tissue to human beings (xenotransplantation). Regrettably, this proposed rule as it applies to gene therapy follows the practice of the National Institutes of Health's (NIH) Recombinant DNA Advisory Committee (RAC) established 20 years ago. The FDA proposed rule is a long overdue attempt to harmonize its policy with that of the NIH. Until this time, the FDA's policy has been not to disclose any information about a new therapeutic intervention, including the fact that an application made by a manufacturer for approval even exists.

The FDA now makes compelling arguments, both ethical and legal, for disclosing safety and efficacy and other information about a therapeutic intervention before it is approved for marketing. Overarching the issue has been the fact that disclosure has not impeded the commercial development of these products exploding industry's anti-science myth that successful commercial development can only take place in secrecy.

As was mentioned above, the FDA recognizes that disclosure is necessary as the foundation for safe and ethical research. Another aspect of ethical research, other than ensuring the informed consent of study participants, is to ensure that negative as well as positive results should be published or otherwise made publicly available. Provisions of the Declaration of Helsinki, the voluntary guidelines for conducting ethical research around the world, state that both the design of all studies and their results should be publicly available.[2]

The FDA's stated legal basis for issuing this proposed rule is sound, namely: "Information that is publicly disclosed by its owner cannot be confidential within the meaning of FOIA [Freedom of Information Act] and, as a result, can be made available for public disclosure by FDA." Companies that are publicly traded are compelled by law to make filings to the Securities and Exchange Commission (SEC) under the Securities Act of 1933. This law requires a company to provide a comprehensive overview of its business that includes a description of ongoing research programs including discussion of clinical study safety and efficacy results, disclosure of investigational sites and the investigators involved.

Companies using the Internet and media often provide descriptive summaries of clinical trials, press releases about positive results, recruitment opportunities for clinical trial subjects, and investment possibilities. Companies also contract with medical communication firms to manage information about new products prior to approval to create the most positive image possible in the public's eye. For example, the medical communications firm Excerpta Medica is controlled by Reed Elsevier, the publisher of a number of journals including Clinical Therapeutics, Current Therapeutics Research, and The Lancet. The Excerpta Medica web site invites potential customers to "Get a glimpse of our approach to: Pre-launch seeding; Corporate communications; Maximizing launch potential; Extending product life cycles."[3]

The FDA's longstanding failure to disclose safety and efficacy information that had already been made public by manufacturers has left patients and health professionals at the mercy of the marketing tactics of these corporations. With the right spin, a required SEC disclose becomes an advertisement with the sole purpose of creating demand for a new product even before it is approved. If government agencies have no other reason to exist it should be to ensure that the public has reliable access to scientifically accurate and objective information.

We find it extremely disturbing that because the FDA has no mechanism for reliably tracking what information has been publicly disclosed by a manufacturer about an unapproved, investigational product, the general policy has been to provide no information whatsoever to the public. An organization that is a part of the U.S. Public Health Service and one that views itself as an important consumer protection agency should err on the side of disclosing non-confidential safety and effectiveness information to the public rather than providing a curtain of secrecy that protects the interests of drug companies. The less that is known about an unapproved, investigational product the easier it is to recruit subjects into clinical trials and if the product is ultimately approved the easier it will be to sell.

The FDA should certainly be able to devise rules and regulations that will allow for the disclosure of safety and effectiveness data about unapproved, investigational products if the requestor of such information can document that the manufacturer of the unapproved, investigational product has already publicly disclosed such information.

The FDA argues that because of the potential for unique public health risks and modification of the human genome that only information about gene therapy and xenotransplantation research need be disclosed. These risks include, among others, the development of cancer and an effect on future offspring. It is neither logical nor ethical for the FDA to assume that unique risks only exist with gene therapy and xenotransplantation research and thus to limit disclosure of information to only research involving these two interventions. The small molecules that we are now most familiar with as drugs, such as cancer chemotherapeutic agents, can cause cancer and the daughters of some women who used diethylstilbestrol to prevent spontaneous abortion developed a rare type of cervical cancer. Other drugs can mutate genes that could become part of the human genome.

Public Citizen strongly supports the disclosure of safety and effectiveness information about unapproved, investigational products during their development. Disclosure of this type of information is important for proper informed consent for those patients considering participation in a clinical experiment. The public does not have reliable access to objective, scientifically accurate information about new treatments. Thus, routine disclosure of this information would also be important for the public to balance the slanted promotional information provided by manufacturers about the benefits of new products.

Unfortunately, this proposed rule is too narrow by only including gene therapy and xenotransplantation and it should have appeared 20 years sooner. Its impetus appears to be political following the tragic death of Jessie Gelsinger in a gene therapy trial and the FDA feeling the pressure to harmonize its disclosure policy with that of the NIH's Recombinant DNA Advisory Committee.

We strongly urge the FDA to extend this rule to all unapproved, investigational products.

Thank you for the opportunity to comment on this important issue concerning the public's health.

Sincerely,

Larry D. Sasich, Pharm.D., M.P.H.
Public Citizen Health Research Group

Sidney M. Wolfe, M.D.
Director,
Public Citizen Health Research Group


 

REFERENCES

[1] Department of Health and Human Services, Food and Drug Administration. Availability for Public Disclosure and Submission to FDA for Public Disclosure of Certain Data and Information Related to Human Gene Therapy or Xenotransplantation. Federal Register Vol. 66, No. 12, January 18, 2001, pages 4688 to 4706.

[2] The Declaration of Helsinki is available on the World Medical Association's, Inc. web site at http://www.wma.net/ accessed April 10, 2001.

[3] www.excerptamedica.com/hq/ accessed April 8, 2001.

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