“When it comes to patient access we believe that new, viable models to improve access can only be addressed if elevated to a higher political level within each stakeholder constituency and developed through collective conversation involving all stakeholders – which is exactly what this stakeholder meeting aimed to achieve,” says Kelly du Plessis, CEO and Founder of Rare Diseases SA.

With parties including the South African Health Products Regulatory Authority (Sahpra), the Council for Medical Schemes, IPASA as well as large medical schemes among others in attendance, dialogue focused on access not only in terms of contentious prescribed minimum benefits (PMBs) and Section 21, but also on orphan drugs and their availability and cost in South Africa.

Discriminatory

“When a rare disease is not funded purely based on the fact that the treatment is costly would it be fair to say the approach and behaviour around funding rare diseases is discriminatory?” asks Patricia Matseke, principal consultant at Elsabe Klinck Associates.

Of course, given the high complexity, limited treatments and limited number of patients, evidence-based decision for funding is difficult, only to be exasperated by a siloed and fragmented eco-system. It is for this reason, Gavin Bauer, chairman of the IPASA Access Working Group, called for a collaborative eco-system – one that views vulnerable communities such as the rare disease community in the context in which they stand, rare.
“Rare disease and access to orphan medicines require a different perspective to essential medicines and when we look at the marginal expenditure growth on these medicines and the decline in utilisation of these orphan medicines within our current medical schemes, one has to question if the medical schemes system has money to fund these orphan medicines and close the funding gap.”

He points to possible solutions including using the increasing level of scheme solvency as funding headroom for rare disease patients but what became evident through the discussion is that schemes are competing on PMBs, rather than standardising these. This needs to be overcome if we are to define PMBs for rare diseases and ensure that our healthcare spend is invested where it is needed most.

It was also collectively agreed that there needs to be new ways and agree on new solutions to ensure that:

• The prices of future orphan medicines are determined in a decisively fairer manner;
• Patient outcomes become once again the single most important factor in the decision;
• Patients are not exposed to unjustifiable access delays or restrictions; and
• Investment into our healthcare system by industry continues to be made – encouraging appropriate market access which is regulated.

No orphan drug policy

Sahpra will play a crucial role in access to orphan medicines. Dr Shyamli Munbodh, acting director clinical evaluations and trials and head of sections 21 units, Sahpra says; “Currently there is no orphan drug policy, but we are working on a broader exemption policy guideline which is expected after June this year. It is aimed at providing guidance on exemptions from the requirements for registration of a medicinal product including those that treat rare diseases.”

The Council for Medical Schemes was also optimistic about the progress being made. “The current PMB package is hospicentric and does not sufficiently include primary preventative healthcare services in support of customer care and we are working on reviewing this. It is a process and with any policy change there will need to be constant revisions and changes. What is evident is that there has to be public-private partnerships for sustainability.”

Unprecedented opportunity

Rare Diseases SA has always been committed to engaging with the Council for Medical Schemes, medical schemes themselves and the National Department of Health to find solutions to access of healthcare for the rare disease community and these stakeholder engagements are critical for advocacy and engagement.

“We have an unprecedented opportunity to address the unmet medical needs of people living with rare diseases and we need to unlock the deadlock we find ourselves in when it comes to access. Access to healthcare is a universal right – it’s not going away, and respective stakeholders need to move forward if we are to get NHI right. When we talk about universal healthcare, resources constraints alone – cannot justify inaction,” concludes Du Plessis.