The critical role of regulators

The critical role of regulatorsThe importance of regulatory bodies in the health sector cannot be overstated. However these same bodies are often beleaguered by inefficiencies, corruption and a lack of resources which hampers their usefulness.


Regulatory agencies play a vital role in the research and treatment of tuberculosis (TB) and, indeed, most medical conditions. Approval by a regulatory authority is necessary to conduct a clinical trial, and later, to ensure access to treatment once research shows sufficient evidence that a new product is safe and effective. Yet most regulators worldwide are poorly equipped to rapidly and rigorously review new products to determine their safety, efficacy, and quality. And patients, doctors, programmes, and pharmaceutical entities are stuck along with them as they try to navigate the delicate balance required to be both thorough and efficient in their review of applications to study new investigational drugs, and to market medications once developed.

Regulators are often underfunded, mired in political controversies, and lack the resources and power to efficiently and appropriately regulate research and medicines. This is especially true for TB, a disease that, until 2012, went without a single approval for a new drug from a new drug class in over 50 years. This innovation gap left regulators without experience or expertise in evaluating new treatment options with potential for improving existing lengthy and often toxic cures for TB. Regulatory inefficiencies that result from gaps in resources, power, experience, and expertise can be a disservice to TB patients by causing delays in reviews and potentially even poor decisions (e.g. approving a product that has not been demonstrated to be safe or effective, or conversely, denying the approval of a product that could benefit patients).

These inefficiencies can also affect patients by discouraging pharmaceutical companies from filing for approval altogether. Companies are already reluctant to register a product in countries where they are unlikely to make any profit. A substantive amount of time and expense is required to prepare applications for research or marketing approval; especially considering that the format and language of those applications (called ‘dossiers’) are often regulator-specific. This discourages companies from widely registering their new drugs or other products. While the responsibility of registration lies firmly with the product sponsor, and activists should continue to urge companies to file for approval widely, regulators can do more to facilitate this process.

Regulatory authorities should explore regional harmonisation to facilitate broader registration without jeopardising quality. This might entail the creation of one overarching regional regulatory agency, which would require sponsors to file only one application (in one language, with one format, one list of requirements, and one set of queries to which to respond) to obtain approval in multiple countries. A regionally harmonised regulatory infrastructure would allow participating countries to pool financial and human resources and expertise, while also making the regulatory process simpler and more affordable for applicants. This would also create an opportunity to pool markets for products or demand for research across several countries, helping to promote the inclusion of countries that companies might otherwise overlook because of small or unprofitable markets, or language barriers.

In addition to functional and coordinated regulatory systems, more transparent ones – with mechanisms for the public to track and provide input into regulatory processes – are essential.


Despite facing challenges of its own, and recent legislative threats to further strip its power to regulate, the US Food and Drug Administration (FDA) sets a strong precedent for a transparent regulator. The FDA has policies and procedures in place that reduce regulatory inefficiencies and promote transparency, including clear timelines, public hearings to solicit input prior to approval, and public posting of comprehensive data supporting new drug applications prior to and post-approval. Activists should call for similar accountability and transparency from their regulators.

Another key function of regulatory authorities is to provide clear, safe, efficient, and sound mechanisms for pre-approval access when appropriate. Allowing regulated access to investigational new drugs with sufficient evidence of safety and efficacy is vitally important for patients with difficult to treat forms of drug-resistant TB, and other life-threatening diseases for whom existing approved treatment options are insufficient and who do not qualify for a clinical trial. Similarly, access to drugs that are approved elsewhere but not yet in South Africa – such as cheaper, generic forms of existing drugs – is necessary in case existing options within South Africa are inaccessible due to price or supply issues. The Medicines Control Council (MCC) laudably has allowed pre-approval access under these conditions – for example, by working with the National Department of Health (NDOH) and clinicians in South Africa to create a Clinical Access Programme to allow for controlled, pre-approval access to new TB drug bedaquiline, and allowing the employment of a Section 21 waiver to sanction the importation of a cheaper, generic form of linezolid, also important for treating some cases of drug-resistant TB. But in each of these instances, processes were slow to advance, leaving many gravely ill and infectious patients without access in the interim. In South Africa and elsewhere, clear, transparent, and efficient processes to request and grant pre-approval access – including on the basis of affordability – must exist.

Indeed, in South Africa, the regulatory system is undergoing reformation. The MCC is soon to be replaced by a new body, the South African Health Products Regulatory Association (SAHPRA).

Thus, the opportunity is ripe to ensure that the new regulatory system is functional, efficient, transparent, and designed to keep the best interests of patients in mind.

If this opportunity is not squandered, SAHPRA may indeed be able to serve as a model for other regulators, and perhaps the centre of a harmonised regulatory authority to ensure quality, efficiency, and access across the region.