For years, Sara worked in a hospital, making care possible for others. Now, after being diagnosed with breast cancer, she was the one in need of care.
By Ned Wydysh, Ph.D., Vice President, Health Advances
When developing cell and gene therapies (CGTs), drug companies face two significant hurdles. The first is gaining market approval from the FDA. The second is getting insurance companies to pay for these treatments that can potentially cure patients of debilitating diseases such as spinal muscular atrophy and retinal dystrophy but at a very high cost.
For example, the EMA approved a gene therapy to correct lipoprotein lipase deficiency, a rare inherited metabolic disorder that causes pancreatitis. This was the first gene therapy ever approved in Europe. It was also the most expensive drug in the world at $1 million per dose. By 2017, the drug manufacturer announced that it would not seek to renew its marketing authorization, remarking that “usage has been extremely limited.”
This illustrates the need for developers to start thinking about their commercial strategy much earlier in the product development process. The following three steps can help improve the chances of getting a new cell or gene therapy reimbursed by payers and pave the way for commercial success.
1. Start earlier than you think you should. In the early stages of product development, it isn’t easy to estimate a product candidate’s price and therapeutic value. The magnitude of the treatment effect, side effects, and the exact competitive landscape are often unknown. Nonetheless, it is important to address this early. From the beginning, good developers conduct extensive clinical research to determine how effective a new treatment is likely to be. Even better developers also conduct high-level forecasting to understand the range of commercial outcomes, such as what indications the therapy is likely to get approval for and how much revenue it might generate. Armed with this knowledge, developers can shift direction as needed. For example, we recently helped a small gene-editing company with limited funding rethink its portfolio strategy and prioritize product candidates that could generate value faster. In this case, they started planning—and pruning—product candidates early in preclinical development.
2. Identify the right outcomes. One of the biggest challenges of getting payer reimbursement for a cell or gene therapy is the steep price. To justify the cost, developers need to figure out the types of endpoints and outcomes that matter most to payers. This is more challenging than it sounds because regulators, patients, and payers don’t always agree on what constitutes a transformative or curative treatment. For example, if the therapy eliminates cancer cells in the blood or the bone marrow of a blood cancer patient, can that be considered a cure? Or, if the treatment improves vision in patients with conditions that cause blindness, does that qualify as a proxy for a long-term cure?
Regulators routinely approve cancer drugs based on the endpoints of overall response rate, such as tumor shrinkage or destruction and progression-free survival—how long a person lives without the illness getting worse. Most patients care about how long the treatment can prolong their lives and improve their quality of life. Payers tend to focus on a product’s overall cost-benefit ratio and its impact on the total cost of care.
Drug developers are used to focusing on the outcomes and endpoints that matter to regulators in clinical trials because product licensure is the holy grail. But few consider the kind of data they need to convince payers of the treatment’s value for money. Small and emerging companies, in particular, think about this too late. By getting regulators, payers, and patients to align on relevant outcomes and endpoints, developers can incorporate those measures into clinical development from the beginning.
3. Take the time to develop a robust plan. Developing a cell or gene therapy can be a fast process because they treat conditions in much smaller, more tightly defined patient populations than other drugs. And many CGTs qualify for expedited regulatory pathways that also cut the time to market. But just because companies can move quickly doesn’t mean they always should. It’s better not to barrel down the clinical development path to proof of concept without a strategy to prove a therapy may qualify as curative once it reaches the market. Cell or gene therapy trials require carefully nurtured relationships with experienced Clinical Research Organizations. Developing a detailed clinical development plan that produces the most robust possible data package to demonstrate a treatment’s full value to payers is just as important as winning regulatory approval. It’s worth investing more time and resources on the front end to avoid repeating studies or adding studies later that would ultimately be significantly more costly.
As the CGT landscape grows more competitive, developers need to take steps to ensure the products’ full value is realized and reflected in reimbursement decisions. Only then can these treatments become viable options for patients – the people who matter most.
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