The SA needs a rare disease policy framework more than legal action

The SA needs a rare disease policy framework more than legal action

Historically, healthcare has been divided into two pillars, emergency care and chronic care. But now genetic medicine has arrived, with new treatments for rare, previously untreatable diseases. As exciting as these treatments are for patients, their cost is an issue.

To meet the cost challenge, South Africa urgently needs to develop a rare disease policy framework that defines what a rare disease is, what rare disease medical care requires and how it should be paid for (both at the level of private and public health care).

Until then, patients with rare diseases will be forced to struggle to access treatment on a case-by-case basis. This generally starts with protracted medically assisted fights and ends up easily in court. Public health system patients, it should come as no surprise, currently have no viable course of treatment at all.

Our media provides glimpses into this process through occasional coverage of various court cases. But while every victory in South Africa’s battle for access to gene therapies matters — not just for litigants but for everyone living in similar circumstances — legal action is unlikely to change the national situation.

thorny paradox

One recent example is The lawsuit was initiated by Cheri Nel and Section 27 The price challenge of a new cystic fibrosis drug, recently covered by Daily Maverick.

Their action was against Vertex Pharmaceuticals Inc, a US pharmaceutical company that had patented Tricafta, a cystic fibrosis treatment priced at about R5,722,400 per patient per year. If the application is successful, the courts will grant a compulsory license, and the generic manufacturer will allow Trifakta to enter the South African market, hopefully at a more realistic price for the local economy.

While Cheri Nel’s case may be a legal precedent, the problem with precedent is that it must be enforced. This is very difficult with rare diseases, because there are so many different conditions, and each only affects a small segment of the population.

It’s a thorny irony because rare diseases aren’t actually rare — as a group. One in 15 South Africans will be affected by a rare disease in their lifetime. The idea that our country would need to go through 7,000 different legal processes to apply the precedents that can be set by cases like Sherri Neal’s is preposterous. But this is our current reality.

Our challenge is systemic

The historical lack of clinical options for rare diseases meant that most of them were ignored when the prescribed minimum benefits (PMB) legislation was put in place in the country. This means that at present, even members of the Medicaid Scheme with rare conditions listed by the PMB are often denied even the most basic levels of care. And in the public health system, many rare diseases are not diagnosed, let alone treated.

Take muscular dystrophy, which is an umbrella term that describes a group of related muscle wasting conditions. Muscular dystrophy is listed by PMB, code 513A. According to South African law, both public and private health funders must cover the cost of treating muscular dystrophy conditions.

The problem comes with the next column in the infamous Appendix A, which describes the required treatment. For Code 513A it says: ‘Preliminary Diagnosis; initiate medical administration; Treatment of acute complications and exacerbations. This is an explanatory formulation, written for a different, hopelessly different clinical context. Many of our medical aides choose to ignore the spirit of the PMB legislation and focus on the vague wording of denial of claims. In the Muscular Dystrophy Foundation of South Africa (MDFSA) We regularly meet with patients whose claims for Medicaid for essential care (such as continuing physical therapy, or mobility aids) have been denied.

Thus, despite the clarity of our Constitution, South Africans who suffer from certain types of disease, such as cancer, receive care and treatment via public and private healthcare systems. But those with other types of disease, such as muscular dystrophy, are often eliminated.

The Department of Health is supposed to review the provisions of the PMB, in accordance with the Explanatory Note to Appendix A, but failed to do so. No one has much idea when the next review will take place. When that happens, an organization like MDFSA will need to make a compelling clinical case for the care needed with best practices, from physiotherapy to generic medicines. If the MDFSA cannot push its foot through the bureaucratic door at this time, it is questionable whether the PMB codes for muscular dystrophy will ever be successfully rewritten.

Now imagine the scenario of conditions not listed in the PMB. And remember, there are over 7,000 of them.

The danger is also systemic

The high prices that drug companies charge for new-generation gene therapies depend not only on the research and development required to develop the treatment, but also on the current cost of care for funders. In Western countries with strong budgets and functional health systems, a person with a muscle wasting condition receives comprehensive care for symptoms, and so there is financial rationale for paying for expensive treatments with the potential to halt progression of the condition and reduce the cost of care.

But in South Africa, the cost to funders of these patients is often minimal. It’s easy to see why local Medicaid is frustrated at having to spend millions a year treating patients who currently cost them so little.

If the status quo continues, South Africa could easily be caught between its constitutional principles and hard reality, as it happens in other countries.

in 2022 The New York Times I reported on the situation in Brazil, in which a parent about to have a child with the same condition I have — spinal muscular atrophy (SMA) — won a lawsuit to force the National Health System to pay for Zolgensma, which was offered by drug giant Novartis at a list price of $2.1 million for a single injection. After about 100 similarly successful lawsuits, the Brazilian government announced that it would pay for the drug for infants with severe forms of SMA, at a reduced cost of $1 million per dose. But even this reduced rate is now putting unbearable pressure on an already fragile health system.

Good and bad news

The good news is that a body called the Rare Disease Access Initiative (RDAI) has been around for several years. led by the NGO, Rare diseases SAIt includes drug companies, medical assistance programs, the National Health Administration and other players. The Rare Disease Corporation has drafted an outline of the required policy framework, which is currently being deliberated on by this body.

However, the bad news is exactly the same. As good as a job has been done, the RDAI has no legal force. It has no deadlines. It does not have a website. Few people seem to have any sense of when it may or may not reach the point of getting the required legislation, let alone a Parliament. Rare Disease SA cracks the whip as best it can, but it’s a small NGO, with a small whip.

Around the world, rare disease advocacy groups—people so desperate for a cure—have forced funders, drug companies, and governments into agreements. But in South Africa, our poor diagnostic ability means that the cohorts of patients with rare diseases are very small. The few NGOs that do exist face an ongoing crisis of securing just enough funding to provide physical care and support to their members.

Participation in legislative advocacy and development is a skill-intensive process that requires specific expertise, and therefore budget. However, our NGOs receive almost no public funding from players in the medical industry to facilitate their supposedly critical advocacy role.

At best this is ill-conceived. It’s cynical at worst. Novartis reported profits of $36 billion in fiscal year 2022. Discovery Health made a profit of R5.5 billion in the same period. If patient advocacy groups are so important to their thriving business and the communities they say they serve, these companies should provide at least some public funding to these groups to allow them to play their role effectively. But they don’t.

South Africa urgently requires improved levels of accountability. Vague assurances that everyone is doing their best, through RDAI, are undermined by the fact that South Africa is making no clear progress in finalizing the required policy framework. Public pressure does not apply, in part because so few people are aware of the problem (until they or a loved one becomes ill with a rare disease), but also because if you never say when you will complete a project or what you think its results will be, it is unlikely That you face any meaningful pressure to achieve your goals.

Only when key RDAI role players announce their intentions, desired outcomes and intended timeframes does that precious nugget we call “political will” approach reality.

Until then we will continue in the same condition. Those who are able will go to court to file a case for treatment. Our NGOs will fight the best fight possible. and the remainder would be left to nurse themselves in their coffins, at great personal expense. DM

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