Post-Trial Access to Communities: The Case of Haematological Malignancies

The issue of post-trial equitable access to essential medicines for treating non-communicable diseases (NCDs) in low and middle-income countries is raising increasing concerns. This article suggests some short-term measures to fill in the relevant gaps

 Post-Trial Access to Communities : The Case of Haematological Malignancies

 by Raffaella Ravinetto*

Head of Clinical Trials Unit, Antwerp Institute of Tropical Medicine

 

The issue of access to medicines in Middle and Low Income Countries (LMICs) has often been investigated from the perspective of transmissible diseases, such as HIV/AIDS, malaria, tuberculosis, and of transmissible tropical diseases. However, non-communicable diseases (NCDs) are more and more prevalent in LMICs: according to the WHO, 80% of deaths from NCDs now occur in LMICs. Inevitably, the issue of access to essential medicines for treating them is raising increasing concerns. A few countries like India and Indonesia have issued compulsory licenses to improve access to some life-saving cancer drugs. According to Bollyky (2013) the controversy over patented medicines for NCDs and their affordability in developing countries is comparable to the one prompted, more than one decade ago, by antiretroviral drugs, and addressing this new access-crisis will require a transformation in global health (1).

As a first step of a broader investigation, and as an attempt to contribute to the ongoing debate, we explored the model of hematological malignancies, and we illustrated our findings in a Comment which has been recently published in the Lancet Hematology (2). We chose this therapeutic field for different reasons. First, new and innovative medicines today allow to cure hematological malignancies in an important group of patients. Second, the high price of these medicines makes them hardly accessible in LMICs, including Middle Income Countries (MICs) like South Africa (3). Third, clinical trials for marketing authorization purposes are often carried out in MICs: our preliminary analysis concerning twelve haematological drugs recommended by international guidelines and/or under development shows that thirty % of phase 3, interventional, commercial trials registered in ClinicalTrials.gov, involved sites in MICs, where the  likelihood that the medicine will be accessible to all is poor, due to pricing issues.

This raises, in addition to the general access issues, the specific issue of post-trial access to the general population in the countries where the trials took place. According to the main international ethical guidelines (4-6), the obtainement of scientific results in a vulnerable group should be linked to health benefits for that population, so that conducting a clinical research in a MIC where all or a part of the population cannot benefit from the study finding is unethical, and even exploitative.

In practice, looking at the clinical development plan adds a layer of complexity -and of ethical challenges- to the problem of access to medicines. In our paper, we suggest some short-term measures for achieving an  equitable distribution of the burdens and benefits of clinical research. First, regulatory agencies should require an “€œethical clause”€ binding the marketing authorization holders to offer innovative, patented drugs at tiered prices in all MICs where trials are conducted. Second, physicians and patients in MICs, hopefully joined in this effort by peers in high income countries, should lobby for a “juxtum pretium” for patented, life-saving drugs tested in trials in their countries.

The mobilization of patients, physicians and civil society has played a major role in the effort to fill the gaps in access to life-saving therapies for HIV/AIDS, and this lesson should now be retaken to correct the unequal distribution of  burdens and benefits in globalized  clinical research, and more generally to fill in the unacceptable gap in access to life-saving therapies for non-communicable diseases.

 

REFERENCES

1) Bollyky TJ. Access to Drugs for Treatment of Non-Communicable Diseases. PloS Med 10 (7): e1001485. doi: 10.1371/journal.pmed.1001485.

2) Ravinetto R, Guenzi PD, Massat P, Gaidano G. Globalisation of clinical trials and ethics of benefit sharing. Lancet Haematology (2014); 1: e54-e56. Accessible upon registration at http://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(14)00004-0/fulltext?version=printerFriendly

3) Experts in Chronic Myeloid Leukemia. The price of drugs for chronic myeloid leukemia (CML) is a reflection of the unsustainable prices of cancer drugs: from the perspective of a large group of CML experts. Blood 2013; 121:4439-42

4) World Medical Association. Declaration of Helsinki: Ethical Principles for Medical Research Involving Human Subjects. JAMA 2013;310: 2191–4

5) The Belmont Report. Ethical Principles and Guidelines for the Protection of Human Subjects of Research. The National Commission for the Protection of Human Subjects of Biomedical and Behavioural Research, 1979.

6) International Ethical Guidelines for Biomedical Research Involving Human Subjects, Council for International Organizations of Medical Sciences (CIOMS) in collaboration with the WHO. CIOMS 2002, Geneva.

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*Raffaella Ravinetto holds a Pharmacy Degree from the University of Torino and a Postgraduate Diploma in Tropical Medicine from the Antwerp Institute of Tropical Medicine.   

After a seven-year experience as a Clinical Research Scientist in the private pharmaceutical sector, she worked in emergency and development programs in the Balkans and in Africa. In 2002, she joined Médecins Sans Frontières (MSF), where she followed various dossiers on access to essential medicines and quality of medicines, while performing regular field assessments. She currently works at the Antwerp Institute of Tropical Medicine, as head of the Clinical Trials Unit, coordinator of the Switching the Poles Clinical Research Network and promoter of Quamed (a Network promoting evidence-based strategies for universal access to quality medicines). She was president of the Italian branch of MSF (2007-2011).   

Her main areas of interest include North-South collaborative clinical research, research ethics (particularly in relation to resource-constrained settings) and access to health.