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Cell & Gene Therapy
Gene therapy

Why gene therapy lotteries are good for patients, providers, and manufacturers

Posted by on 25 February 2020
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by Jeremy Schafer, PharmD, MBA, Senior Vice President, Precision for Value

Staggered launches of a pharmaceutical across different parts of the world are a standard practice due to differences in how each nation reviews and approves drugs. For patients with a condition in need of treatment, the wait can be frustrating but as gene therapies with the potential for cure arrive, the wait can also be life threatening. Novartis, the manufacturer of Zolgensma for spinal muscular atrophy (SMA), devised a novel approach to the issue of waiting: a lottery.[1] Up to 100 doses of Zolgensma will be given away to patients in countries where Zolgensma is not yet approved.[1] Patients will be chosen at random, irrespective of disease severity or progression, in a biweekly drawing.[2] The lottery may be expanded at some point but is limited primarily by tight supply of product.[1] The program’s launch has not been without controversy as ethicists debate whether life should be contingent on the luck of the draw.[2] As other gene therapy agents near approval, manufacturers of life-changing therapies may question whether the lottery idea should remain unique to Zolgensma or be replicated. The benefits of replication can extend beyond helping more patients in need and provide additional value to the manufacturer.

Tracking outcomes

A primary cause of slow product uptake, regardless of disease state, can be a lack of experience. Gene therapies are generally directed at rare conditions, meaning the pool of real- world experience grows slowly when a product launches. A lottery, however small, can help to expand the population and diversity of treated patients. A manufacturer with a lottery program may consider a mechanism to track the outcomes of treated patients, particularly those in nations that were not part of the clinical trial program, and then highlight these outcomes in case reports and case series to build the real-world evidence case for the therapy. Real-world evidence could increase comfort with payers, regulators, and providers that the product’s value is evident outside the clinical trial setting.

Increasing advocacy

A lottery may generate advocacy. Patient and provider advocacy are an important ingredient in drug approval and uptake but can be slow to build in rare disease due to the small population and share of voice. Providers in countries without access to the gene therapy gain valuable experience using a gene therapy product if his/her patient is chosen. As providers and patients in a specific territory gain positive experience with a gene therapy, they become influential advocates. Testimonials to regulators by providers and patients with treatment experience may help to hasten approval and bring the gene therapy to more of a country’s residents sooner.

Enriching financial data

The understanding of the financial impact of a new therapeutic can be a hurdle to approval in some countries. A lottery does not solve this problem but is an opportunity to generate data on financial offsets. For conditions such as SMA, a patient may be treated with high cost, chronic therapy in addition to other health resource utilization such as clinic visits, medical equipment, etc. A patient successfully treated with gene therapy may have needs for chronic therapy dwindle or disappear entirely depending on the drug and condition treated. Changes in claims and spend for individual patients can be tracked by payers and showcased as an example of the real-world financial and health resource impact of the gene therapy. Regulators will still need to do the cost effectiveness analysis, but any modeling can be augmented with individual cases, in the country conducting the review, on the real-world savings and impact of curative therapy.

The primary benefit of a lottery program for emerging gene therapies is the opportunity to treat more patients in need. However, for prepared manufacturers, the lottery offers an opportunity to build real-world evidence, create advocates in the patient and provider communities, and generate data on financial offsets and decreases in health resource utilization among treated patients. In rare disease, the small population means every patient, and data point, counts. A lottery may increase both, for the benefit of patients, providers, the system, and manufacturers.


  1. STAT. A lottery like no other offers up a cutting-edge medicine — with lives on the line. February 7, 2020. Accessed February 13, 2020.
  2. The Guardian. Dismay at lottery for $2.1m drug to treat children with muscle-wasting disease. December 20, 2019. Accessed February 13, 2020.
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