While Novartis ’ gene therapy Zolgensma (AVXS-101) for spinal muscular atrophy (SMA) type 1 awaits an imminent regulatory decision from the US Food and Drug Administration (FDA), its price point will be closely watched, as it is likely to influence its availability to SMA patients.

Novartis believes that Zolgensma’s price should be in the range of $4–5 million because it is one-off gene therapy. This stands in sharp contrast to the evaluation from the Institute for Clinical and Economic Review (ICER), which proposes that the therapy is cost-effective at $900,000 on the basis of a lower quality-adjusted life year (QALY) threshold of $150,000.

Spinal muscular atrophy therapies

It is arguable that limited treatment options for a rare disease such as SMA means that approved therapies could launch at an exceptionally high price point, especially in the absence of a comparator therapy or when the natural history of the disease is used for comparisons. However, pricing based on that concept largely fails to justify the pricing framework or policy used by funding agencies in such situations, and often leads to a scenario where the therapy is not required to be reimbursed by a country’s funding agencies, which may call for more transparency regarding how the price point was initially arrived at by the parent company and the long-term effectiveness of the therapy.

That was the case with Biogen ’s Spinraza (nusinersen), which was not recommended by the National Institute for Health and Care Excellence (NICE) for use in England despite winning approval for its use in Europe in 2017. On 15 May NICE announced that Spinraza would be made available to type 1 SMA patients and that it will be funded by National Health Service (NHS) England. While the final discounted price for Spinraza in England is currently unknown, GlobalData anticipates that other high-priced therapies that are expected to launch in the future will be subjected to scrutiny by funding agencies as part of a push back against an industry that is fundamentally driven by high returns on investment.

Insights from the recently concluded 2019 annual meeting of the American Academy of Neurology (AAN) showcased the potential of evolving pipeline therapies for SMA, which are poised to compete directly with Spinraza, including Novartis’ Zolgensma and Roche ’s risdiplam, both of which are being evaluated in multiple SMA patient population types. While key opinion leaders interviewed by GlobalData are all excited by the prospect of promising new therapies for SMA and welcome such treatment options, the general consensus was that price point and patient access are expected to have an inverse relationship. For example, if a company decides to launch a promising therapy at a higher price point than expected, it could mean that the access is restricted to a specific type of patient population, as seen with type 1 SMA, the most severe form of the disease.

Alternatively, if the company aims to capitalise on more patient numbers, the therapy would have to be priced lower in order to collaborate with funding agencies and ensure wider market access. Questions remain about the long-term effectiveness of these innovative therapies, including how they function and who will pay if these therapies do not perform as expected. As such, a collective decision may be made by patients and physicians to revert back to the standard of care. Either way, it is imperative that these discussions on pricing remain ongoing both in SMA and other rare diseases, as they are expected to hugely influence wider market access.

Forthcoming report
GlobalData (2019). Spinal Muscular Atrophy: Opportunity Analysis and Forecasts to 2028, to be published