Biomedical innovation is a major societal priority, and public and private funders invest heavily in the development of new health technologies such as medicines and medical devices. However, the innovation pathway is complex, involving multiple processes that include pre-clinical and clinical testing, product registration by the Therapeutic Goods Administration (TGA), subsidisation on the Pharmaceutical Benefits Scheme (PBS) or Medicare Benefits Schedule (MBS), and integration into clinical practice. All of these processes need to be successfully navigated before patients are able to access new health technologies.
In recent years, consumers, clinicians and the biopharmaceutical industry have begun to question the length of time it takes to register new health technologies and secure their subsidisation in Australia, as well as the standards of evidence that regulators and funders require for their assessments. The concern is that these evaluation processes are excessively slow and conservative, and that Australian patients are “missing out” compared to patients in other countries. These concerns have prompted calls for faster access to more health technologies and have resulted in a series of inquiries and reviews, including a Senate Inquiry into the Availability of New, Innovative and Specialist Cancer Drugs in Australia,1 and an Expert Review of Medicines and Medical Devices Regulation.2
Consumers have played an important role in these processes, advocating for more rapid and less stringent health technology assessment processes on the basis that:
- there are patients in desperate need of treatment who don’t have time on their side; and
- certain groups of patients (e.g. those with rare diseases) are missing out on access to new treatment options because it is difficult to demonstrate that the products they require are safe and effective.3
While these arguments are compelling and underscore the importance of timely access to the products of biomedical innovation, it is important to keep in sight the harms that can occur when new health technologies are offered to patients without sufficient regulatory oversight.
Tens of thousands of patients have since undergone surgical revision, while many more suffer adverse side effects and require ongoing monitoring.
Recognition of the need for stringent regulation of health technologies reaches back to the thalidomide disaster in the mid 20th Century, when market forces drove the wide distribution of an inadequately tested drug that affected tens of thousands of children.4 A more recent example that illustrates the ongoing need for rigorous regulation was the premature approval of new metal-on-metal hip replacement technology in Australia and the European Union during the 1990s. This product, which was marketed by DePuy Orthopedics (a subsidiary of Johnson & Johnson) was soon found to cause serious side-effects as a result of metal leaking into surrounding tissue. Although there was ultimately a worldwide recall of the product, a confluence of financial and professional conflicts of interests encouraged the early adoption of the product into clinical practice, even as evidence of side effects was mounting.5 Tens of thousands of patients have since undergone surgical revision, while many more suffer adverse side effects and require ongoing monitoring.6
The current class action lawsuits against the manufacturers of surgical mesh in Australia, the United Kingdom and the United States offer another illustration of the dangers of inadequate pre-market assessment of health technologies. Originally approved for use in hernia repair, manufactures of some types of surgical mesh secured regulatory authorization to market it for the treatment of pelvic organ prolapse under ‘fast-track’ approval mechanisms.7 These authorizations enabled rapid and widespread distribution of a health technology that has subsequently caused serious complications in recipients, including severe abdominal pain, organ perforation and sexual dysfunction.8 Plaintiffs in the various class-action lawsuits are arguing, among other things, that the devices were approved (and remain on the market) despite being defectively designed and misleadingly promoted.9
Further evidence of the need for rigorous regulation is demonstrated in cases in which health technologies that are on the market have subsequently proved to be harmful but have not been withdrawn in a timely manner.10 Perhaps the best-known example of this is the anti-inflammatory drug rofecoxib (Vioxx), which caused heart attacks in thousands of patients (including at least 300 in Australia) before it was withdrawn from the market.11
In this context, it is difficult to sustain the argument medicine should be viewed as a marketplace shaped by the choices made by fully-informed consumers.
In arguing for more rapid access to medicines, consumers frequently point out that, while cases such as those described above are tragic, it is they—not researchers, doctors or the health system—that are taking the risk, and that they, as informed consumers, have the “right” assume this risk if they so choose.12 While it is certainly true that consumers should be allowed to choose how much risk to take on, it is also important to bear in mind that consumers—and, indeed, their doctors—are not always well informed about the risks associated with new health technologies. This is not only because of the uncertainties inherent in evidence-based medicine, but also because messages about safety and efficacy may be actively manipulated by those with vested interests. In this regard, it is noteworthy that in both the Vioxx11 and vaginal mesh cases,9 there is evidence that doctors and consumers were actively misled by those manufacturing and/or selling these products. In this context, it is difficult to sustain the argument medicine should be viewed as a marketplace shaped by the choices made by fully-informed consumers.
None of this is to argue against consumer advocacy for faster access to health technologies. It does, however, raise two fundamental questions for consumer advocates: first, what do consumers really know and want when it comes to access to health technologies? And what responsibilities do consumer advocates have to balance (valid) calls for access to health technologies against (equally valid) demands for thorough regulatory oversight?
Photo by Alvin Engler on Unsplash
- Parliament of Australia. Availability of new, innovative and specialist cancer drugs in Australia http://www.aph.gov.au/Parliamentary_Business/Committees/Senate/Community_Affairs/Cancer_Drugs/Report Accessed November 8 2015
- Department of Health. Expert Review of Medicines and Medical Devices Regulation http://www.health.gov.au/internet/main/publishing.nsf/Content/Expert-Review-of-Medicines-and-Medical-Devices-Regulation Accessed
- Ghinea N, Little M, Lipworth W. Access to high cost cancer medicines through the lens of an Australian Senate Inquiry – defining the’goods’ at stake. Journal of Bioethical Inquiry. 2017;Published online 18/7/2017.
- Ridings J. The Thalidomide Disaster, Lessons from the Past. In: Barrow P, editor. Teratogenicity Testing. Methods in Molecular Biology (Methods and Protocols), vol 947. Totowa, NJ: Humana Press, 2013.
- Johnson J, Rogers W. Joint issues–conflicts of interest, the ASR hip and suggestions for managing surgical conflicts of interest. BMC medical ethics 2014;15:63.
- Carr A. Follow-up of patients with metal-on-metal hip replacements: British Medical Journal Publishing Group, 2017.
- Hutchison K, Rogers W. Hips, Knees, and Hernia Mesh: When Does Gender Matter in Surgery? IJFAB: International Journal of Feminist Approaches to Bioethics 2017;10:148-74.
- Maher CM, Feiner B, Baessler K, Glazener CM. Surgical management of pelvic organ prolapse in women: the updated summary version Cochrane review. International urogynecology journal 2011;22:1445.
- Drugwatch. Transvaginal Mesh Verdicts and Settlements https://www.drugwatch.com/transvaginal-mesh/verdict-settlement/ Accessed September 27 2017
- Onakpoya IJ, Heneghan CJ, Aronson JK. Delays in the post-marketing withdrawal of drugs to which deaths have been attributed: a systematic investigation and analysis. BMC Med 2015;13:26.
- Krumholz HM, Ross JS, Presler AH, Egilman DS. What have we learnt from Vioxx? BMJ 2007;334:120.
- Right to Try. FAQs http://righttotry.org/faq/ Accessed September 7 2017