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Market access challenges in a world of rare diseases

On their own, these diseases are rare, but together they comprise a considerable health challenge to be addressed. There are thousands of rare diseases which require innovative approaches to treatment, allowing for care to be provided for those patients most at need.

In terms of rare diseases, time is everything — especially degenerative diseases where deterioration can be irreversible.

Access for everyone

Revolutionary progress has been made in recent years regarding our understanding of rare disease treatments, including CAR-T therapy, protein replacement therapies, oligonucleotides and gene/cell therapies.

Most of these treatments are transformative, often addressing the root cause of disease. Therefore traditional payment and reimbursement methodologies, designed for chronic therapies, may need redefining.

For example, across Europe, access to treatments is heterogeneous, and with one-time gene therapies disrupting conventional reimbursement processes, it is imperative early access solutions are tailored by the market to attain timely convergence with regulatory approval. Emerging signs show healthcare systems are beginning to translate the science and adapt to transformative access models.

A closer look at the spinal muscular atrophy (SMA) space, a rare, degenerative neuromuscular disease, demonstrates the challenges for access to innovative therapies and how we can overcome them.

Five years ago, it was still a dream that patients with SMA could be treated with a one-time gene therapy and that it would be available through a nation’s health service. Since the approval of the first gene therapy for SMA, however, 32 markets globally have established access pathways for the medicine.

Unlocking excellence

The key to success lies in early engagement to co-create solutions which address unique market-specific dynamics – requiring stakeholder cooperation across the full healthcare ecosystem. One important factor to any access strategy’s success is early collaboration and education of stakeholders.

It’s the nature of innovation that established methodologies may have to be reset and it is important to highlight necessary challenges at an early stage.
In today’s individual-centric world, a ‘one size fits all’ approach is outdated. Instead, we must deliver bespoke agreements tailored to every healthcare system’s particular needs.

The 32 established access pathways for our gene therapy – including those in European countries – come in many forms depending on payer preference. To meet these needs, Novartis Gene Therapies offers flexible payment models, including retroactive rebates, deferred payments and installment options – offering the flexibility and partnership required.

Just do it

We have had a tremendous opportunity to be a catalyst incubating a new playbook for 21st century medicines in healthcare systems that were designed from chronic therapies. As more breakthrough cell and gene therapies are discovered and continue to come to market, more customised commercial pathways will need to be established.

These innovative pathways aren’t solely beneficial to patients, but have a knock-on effect on healthcare systems, paving the way for future transformative treatments to reach patients as fast as possible.

Tay Salimullah is Global Head of Value & Access at Novartis.
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