Skip to main content

Advertisement

SpringerLink
Log in

Regulatory Concerns with Gene Therapy and Recombinant Therapeutic Proteins

Implications for Drug Development

  • Review Article
  • Disease Treatment Review
  • Published:
Clinical Immunotherapeutics Aims and scope Submit manuscript

Summary

Knowledge gained from genetic research and the human genome project has created opportunity to understand human disease and to discover new modes of therapy. Gene therapy offers hope for the treatment of conditions such as cancer and AIDS as well as for genetic diseases and defects. In spite of the benefits of gene therapy, concerns exist about the long term implications of treatments that involve the manipulation of the human genome. Ethical questions have been raised about the rights of future generations as well as about the protection of individual rights if genetic information becomes available to health insurers and employers.

The US Food and Drug Administration has recently proposed specific guidelines for gene therapy. The European Community has centralised regulatory process for biotechnology products. The three major pharmaceutical markets (the European Community, the US and Japan) are working to standardise pharmaceutical regulatory requirements on a global scale. It is hoped that such cooperation will reduce the barriers to entry into the global market for innovative products such as gene therapy.

Regulations affecting the healthcare market are likely to affect research into gene therapy. Escalating healthcare expenditure is a global issue that has resulted in governmental control of pharmaceutical prices. The likely effect of such regulations is to delay innovation. Therefore, pharmaceutical scientists need to include healthcare policy makers in their audience.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Similar content being viewed by others

References

  1. Letsch SW. National health care spending in 1991. Health Aff 1993; 12(2): 94–110

    Article  CAS  Google Scholar 

  2. Cost effectiveness research is underfunded in health system, CEA chair tells senate labor; increases should be built into higher biomedical research funding. FDC Rep 1993 Oct 25; 55(43): 6

    Google Scholar 

  3. Moore SD. UK demands 2.5% reduction in drug prices. Wall Street Journal 1993 Aug 11; B6

    Google Scholar 

  4. European pharma sales decline. Scrip 1993 Jul 6; 1835: 8

    Google Scholar 

  5. Burrill S, Lee K. Biotech’ 93: accelerating commercialization [report]. Ernst & Young, 1992, San Francisco

    Google Scholar 

  6. 143 Biotech drugs in US development. Scrip 1993 Oct 19; 1865: 23

  7. Human Genome Sciences Inc. has discovered 14,000 novel genes. FDC Rep 1993 Oct 25; 55(43): T&G 8–9

    Google Scholar 

  8. Bienz-Tadmor B. Biopharmaceuticals go to market: patterns of worldwide development. Biotechnology 1993; 11: 168–72

    Article  PubMed  CAS  Google Scholar 

  9. The Orphan Drug Act. Public Law 1983; 97–414

  10. The Drug Export Amendment Act. Public Law 1986; 99–660

  11. Watson JD. The Human Genome Project: past, present, and future. Science 1990; 248: 44–8

    Article  PubMed  CAS  Google Scholar 

  12. Watson JD, Cook-Deegan RM. Origins of the Human Genome Project. FASEB J 1991; 5: 8–10

    PubMed  CAS  Google Scholar 

  13. Dickman S, Alhous P. Helping Europe compete in human genome research. Nature 1991; 350: 261

    Article  PubMed  CAS  Google Scholar 

  14. Ikawa Y. Human genome efforts in Japan. FASEB J 1991; 5: 66–9

    PubMed  CAS  Google Scholar 

  15. Caskey CT. Molecular medicine: a spin-off from the helix. JAMA 1993; 269: 1986–92

    Article  PubMed  CAS  Google Scholar 

  16. Chase, M. New test tells whom a crippling disease will hit — and when. Wall Street Journal 1993 Dec 8; Al, A6

    Google Scholar 

  17. Macilwain C. Genome project ‘to be done by 1994’. Nature 1993; 362: 488

    PubMed  CAS  Google Scholar 

  18. Goldspiel BR, Green L, Calis KA. Human gene therapy. Clin Pharm 1993; 12: 488–505

    PubMed  CAS  Google Scholar 

  19. Human Genetics Committee. Position paper on human germ line manipulation presented by Council for Responsible Genetics; Fall 1992. Hum Gene Ther 1993; 4: 35–7

    Article  Google Scholar 

  20. Wivel NA, Walter L. Germ-line gene modification and disease prevention: some medical and ethical perspectives. Science 1993; 262: 533–538

    Article  PubMed  CAS  Google Scholar 

  21. Murray TH. Ethical issues in human genome research. FASEB J 1991; 5: 55–61

    PubMed  CAS  Google Scholar 

  22. Aldhous P. Who needs a genome ethics treat? Nature 1991; 351: 507

    PubMed  CAS  Google Scholar 

  23. Swinbanks D. Japan reviews first guidelines for gene therapy trials. Nature 1993; 362: 684

    Google Scholar 

  24. Gene therapy patient registry for long-term side effects proposed by FDA. FDC Rep 1993 Nov 1; 55(44): 19–20

    Google Scholar 

  25. Hakim A, Kucukarslan S. Regulation of biotechnology in the global pharmaceutical market: the case of the European community and the United States. Clin Ther 1993; 15: 442–58

    PubMed  CAS  Google Scholar 

  26. Hile JP, Keech BH, Gund CK, et al. A compilation of government regulation of biotechnology. In: Chiu YH, Gueriguian JL, editors. Drug biotechnology regulation: scientific basis and practices. New York: Marcel Dekker, 1991: 525–44

    Google Scholar 

  27. Food and Drug Administration. Points to consider in human somatic cell therapy and gene therapy. Rockville MD: Center for Biologics Evaluation and Research, 1991

    Google Scholar 

  28. Kessler DA, Siegel JP, Noguchi PD, et al. Regulation of somatic-cell therapy and gene therapy by the Food and Drug Administration. N Engl J Med 1993; 329: 1169–73

    Article  PubMed  CAS  Google Scholar 

  29. Application of current statutory authorities to human somatic cell therapy products and gene therapy products. Federal Register 1993; 58(197): 53248–51

    Google Scholar 

  30. New drug, antibiotic and biological drug product regulations: accelerated approval. Federal Register 1992; 57(239): 58942–60

    Google Scholar 

  31. Pulmozyme development may be prototype for other biotech products. FDC Rep 1994 Jan 3; 56(1): 8

    Google Scholar 

  32. Guidance on alternatives to lot release for licensed biological products. Federal Register 1993; 58(137): 38771–3

    Google Scholar 

  33. EC concertation progress reviewed. Scrip 1993 Aug; 1850: 7

    Google Scholar 

  34. Common registration package is ‘realistic possibility’ of harmonization, FDA’s Kessler tells ICH 2. FDC Rep 1993 Nov 1; 55(44): 12

    Google Scholar 

  35. Drug applications can be filed based on six-month safety data from 300–600 patients — ICH 2. FDC Rep 1993 Nov 1; 55(44): 13

    Google Scholar 

  36. Myers MJ. Possible impact of the new Medicaid Rebate Law. American Druggist 1991 Feb: 69, 73, 76

    Google Scholar 

  37. Breakthrough drug review provisions of modified Clinton health reform plan would create council under HHS instead of subgroup of National Health Board. FDC Rep 1993 Nov 1; 55(44): 4–5

    Google Scholar 

  38. Biotech product separation from drug price controls. FDC Rep 1993 Jul 12; 55(28): T&G 8–9

    Google Scholar 

  39. Dinner A, Fose JF. Industry’s view on government regulatory activities. In: Chiu YH, Gueriguian JL, editors. Drug biotechnology regulation: scientific basis and practices. New York: Marcel Dekker, 1991: 371–88

    Google Scholar 

  40. Faulds D, Benfield R. Interferon beta-1b in multiple sclerosis: an initial review of its rationale for use and therapeutic potential. Clin Immunother 1994; 1: 79–87

    Article  Google Scholar 

  41. Chiron/Berlex Betaseron gains first biotech accelerated approval. FDC Rep 1993 Jul 26; 55(30): T&G 1

    Google Scholar 

  42. Schering-Chiron drug for MS to cost patients up to $10,000 yearly. Wall Street Journal 1993 Aug 3; B8

Download references

Author information

Authors and Affiliations

  1. College of Pharmacy, The Ohio State University, 500 W. 12th Avenue, Columbus, Ohio, 43210-2499, USA

    Suzan Kucukarslan

Authors
  1. Suzan Kucukarslan
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Kucukarslan, S. Regulatory Concerns with Gene Therapy and Recombinant Therapeutic Proteins. Clin. Immunother. 1, 358–368 (1994). https://doi.org/10.1007/BF03258513

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/BF03258513

Keywords

Search

Navigation