WFH campaign pays off as the World Health Organization (WHO) retains coagulation factor concentrates on its recommended list of “essential medicines.”
The WHO Expert Committee on the Selection and Use of Essential Medicines has decided to retain plasma-derived factor VIII and factor IX concentrates on its Model List of Essential Medicines. The move followed a 15-month WFH campaign involving doctors, medical organizations, patient associations, industry representatives, and other WHO departments.
“The WHO Expert Committee decision will save lives by continuing to make safe and effective treatment available,” says WFH President Mark Skinner.
“The Essential Medicines list is important, because governments refer to WHO recommendations when making decisions on health spending.”
WHO’s list is a guideline for governments that want to ensure they have an inventory of medicines needed by every health system. The list had long included hemophilia treatment products, but in 2003, members of the Essential Medicines committee decided to review the public health relevance of factor concentrates.
The WFH and its supporters pressed strongly for retention of hemophilia treatment products on the list and sent a total of 19 submissions to the WHO committee.
Skinner and WFH executive member, Professor Mammen Chandy were invited to Geneva to present the case for retaining the treatment products.
The WFH submission argued that removing the products from the list would violate WHO’s founding principle that access to life-saving medicines is a fundamental human right. “The retention of factor concentrates will support the efforts of the WFH and hemophilia organizations to lobby health departments for adequate treatment, ” says Skinner.
“The concentrates have enjoyed an excellent safety record for over 12 years. They also meet the list’s other requirements, particularly in the area of public health relevance. One in 10,000 people is born with hemophilia and the number of people who need factor concentrates increases greatly with the addition of those with von Willebrand disease and other bleeding disorders.”
The WFH submission was prepared by Chandy, a leading Indian hematologist, who hopes health authorities see the WHO decision as endorsement of the “essential” nature of clotting factor concentrates for the care of people with bleeding disorders.
“Unavailability of safe concentrates has resulted in bleeding, HIV/AIDS, and hepatitis C as chief causes of death for many people with hemophilia,” he says.
“Death and disability rates in developing countries will increase if governments do not make more safe products available or if viral-inactivated concentrates are replaced, for economic reasons, by products that have potential for viral infection.
“Assessments of the cost of factor concentrates also need to consider the alternative “cost” of early death, life-long disability, and possible viral infection, as well as the burden on families and society by people who do not receive adequate care. The WFH sees provision of on-demand therapy as a more humane and economic proposition.”
The WFH wishes to thank all the individuals and organizations that provided submissions and support for this campaign. To view the 14th Model List of Essential Medicines go to:
Specialists at the University of Essen’s Nephrology Department in Germany have described the first liver transplant in an HCV/HIV-coinfected hemophilic B patient.
The patient was a 44-year-old man who tested positive for HIV and HCV in 1986 and later developed terminal cirrhosis.
In July of 1998, he underwent the liver transplant, after which the new organ immediately started synthesizing normal FIX levels. Subsequent tests showed that he continued to be HCV positive. Furthermore, treatment with AZT, 3TC and saquinavir had improved his HIV-related symptoms, as evidenced by stable CD4 levels and undetectable viral load (fewer than 50 copies).
More importantly, however, is that twelve months after the transplant there was no acute rejection of the new organ and the patient was phenotypically cured of hemophilia B.
The authors of this study use the results to affirm that a liver transplant is an option worth considering in cases of HCV/HIV-coinfected individuals, or at least in those of HCV/HIV-coinfected hemophilic patients. We believe this case-study offers great support for those in favor of organ transplants, specifically liver transplants, for seropositive HIV patients that may need one. There are fewer and fewer reasons for why an organ transplant should be denied, unless these involve extratherapeutic (e.g. political) factors.
Zimmermann, U., and colleagues, Abstract 1056, «VII Conferencia ECCAT, Lisboa, 23-27 de octubre de 1999».
The Basque Country has not changed its position regarding the prevention of hemophilia. The Basque Health Advisor, Gabriel Inclán, presented the conclusions to a report on new orthogenic techniques in preventing hemophilia and other sex-linked disorders.
The report recommends not adding new techniques to the ones already applied, given the fact that these new methods are not yet apt for incorporation into normal clinical practices.
Inclán also guaranteed the security of information regarding centralized medical records. Inclán presented before the Autonomous Parliament the conclusions to a study carried out by the Basque Health Service, Osakidetza, on the prevention of sex-linked disorders (especially hemophilia) via orthogenic techniques.
The following is the Basque Country’s analysis of prevention techniques for sex-linked disorders:
Technique and clinical practice:
- Artificial insemination using gametes from a healthy male and oocyte donations from healthy females. Used despite donor shortage.
- Preconception diagnosis: sperm washing and polar body biopsy. Not clinically applicable.
- Preimplantation diagnosis: early embryo biopsy and blastocyst biopsy. Not clinically applicable due to ineffectiveness and legal limitations.
- Prenatal diagnosis: amniocentesis, chorionic biopsy and cordocentesis. Practiced clinically and considered to be the most effective.
Osakidetza-Basque Health Organization According to the health adviser, “of the four existing techniques (see table), Osakidetza only allows the last one to be performed, as it has proven to be the most successful, practical, effective and reliable. It is also by far the one with the best cost-benefit relationship”.
The Basque Health Organization also permits the implantation of fertilized ovules from a healthy donor, “even though is a limited alternative because of egg donor shortage”.
With respect to the other techniques considered by Osakidetza, the report concluded the following: preconception diagnosis is not developed enough to be clinically applicable; some preimplantation diagnostic techniques cannot be used on hemophilic patients, while others only allow for sex selection that is neither completely safe nor entirely effective.
There is doubt as to whether these two techniques are legally viable under current national law, considering the current problems associated with their practice.
Confidentiality The health adviser also responded before the Health Commission of the Autonomous Parliament to the opposition’s criticism regarding the centralization of medical records.
Inclán guaranteed that maximum information security and confidentiality was maintained throughout the entire process and affirmed that, contrary to some politicians manifested, user authorization was not necessary.
According to Inclán, the Osabide system is secure because a person’s medical records can only be consulted for medical reasons by authorized users. He also insisted that “centralization is nothing new in Osakidetza’s primary medical care.
A person’s medical records are now available on an Autonomous Community level, rather than in just one health center”.
The health adviser indicated that Osabide has undergone several outside security measures, all of which yielded positive results.