With the potential to cure the “incurable”, it’s not surprising that the cell and gene therapies industry has gained popularity in recent years. Yet, launching a product in this industry is no easy task. Experts Simone Seiter and Wolfram Lux explain the steps companies need to take both to ensure a successful launch and encourage future growth.
Launching a cell or gene therapy is a complex process, yet it’s a sector that is quickly gaining ground. Receiving 70.8 billion US dollars of total funding in 2021, the cell and gene therapies (CGTs) industry is evolving at a fast and exciting pace. Yet while stakeholders rush to invest, can the industry deliver when it comes to launch?
CGT products launched today face numerous challenges. While these disease-modifying often one-shot treatments address significant unmet medical needs, uptake for many has been slow and, in some cases, far from expectations regarding their patient share. There are several factors that contribute to this.
Firstly, finding the right patients for a specific CGT product is a challenge. Since these therapies often target rare or orphan diseases, patients are naturally hard to find. This, however, is often exacerbated by the fact that many patients either don’t get diagnosed, are misdiagnosed, or are simply unaware about the treatment options available.
However, challenges arise even when the target patients are found. Some patients who have been diagnosed can become comfortable with the coping mechanisms they’ve put in place to manage their chronic illness. It takes time to educate and activate these patients to seek curative treatments and embrace medical innovations.
Lastly, even if patients or their families seek care, our recent market research revealed that industry experts report that a sizable portion of the population is still concerned about the safety of CGT and its long-term effects. As such launching CGTs is a complicated process that requires careful preparation.
In this article, we list the six crucial steps required to optimally prepare, ensuring CGT launch excellence.
Six steps to prepare for launch
1. Understand target market players and dynamics in detail
When preparing to launch a new CGT product, it’s important to consider the overall healthcare landscape of your target market. The competitive landscape, the patient journey, the healthcare environment and the policies in each target market all impact launch, so must be thoroughly understood.
For example, therapies treating orphan diseases are often the first of their kind. In that instance, it’s important to energize and activate the market. However, there are times when there are multiple competitors – though, they tend to be quite far apart in development stages, as with orphan drugs it’s often a case of first come first serve. So, if there is already a similar treatment to yours in the market, you need to decide if yours can really be better than the one that is already being developed / is available.
Consequently, understanding these nuances in your target market is crucial to successfully launch CGTs.
2. Define which patients need your treatment
As it’s difficult to find patients, it’s important to determine who should receive the treatment, what is the best treatment paradigm, and how it fits into your target market’s guidelines, if existing.
In many indications CGTs establish the first real treatment option. For this reason, the definition of the eligible patient profile is actively driven by CGT manufacturers in collaboration with several medical specialists. Often, in these situations the challenge lies in educating physicians about the diagnostic parameters of patients. This is because those readouts can vary quite significantly due to the limited number of patients. Physicians can be misled to exclude patients from therapy as they fail to recognize their eligibility.
3. Remember, every launch is different
As these therapies often tackle rare or orphan diseases, the launch of these products should be treated as unique as the diseases they are targeting. Relying on generalizations taken from past launches doesn’t pay off, as these often don’t apply to a new launch.
On the other hand, while it would be a mistake to use generalized strategies, industry experts highlight the importance of employing a team with previous launch experience. Without such practiced team members, experts say that it’s hard to facilitate a successful CGT.
4. Partner with patient advocacy groups (PAGs)
PAGs are led either by people who have worked closely with or cared for the patients suffering from the disease, or are patients who have long suffered from the disease themselves. As such, they are well-positioned to provide real-life insights, assisting both pre- and post-launch activities – e.g., shaping clinical development programs, the drug value proposition, helping with national payer engagement and reimbursement, or even influencing health policy.
It’s important to partner with PAGs from the start of the development process, so that you can be better informed about your patients’ needs, and they can be educated on how your treatment will help them. As CGTs can take up to a year to take effect, it’s crucial to continue supporting PAGs post-launch. This is so that you can better understand and manage patient expectations, support them and their families in daily routine, and provide PAGs with the necessary resources to keep helping patients in need.
5. Support and educate healthcare professionals
A healthcare professional (HCP) could spend an entire career never coming across rare or orphan diseases. This lack of exposure and awareness can lead to misdiagnosis, HCPs failing to spot the disease, or to patients getting referred from professional to professional with no results.
Even HCPs who are expert diagnosticians may not be the treatment centers of the future. As such, it’s important to partner with HCPs, informing them on optimal treatment centers for their patients.
Providing education and raising awareness of the disease among HCPs is crucial, therefore, to successfully launch a CGT product and maintain growth post-launch.
6. Build the supply chain for gene and cell therapy production
CGT manufacturing and supply chain is still in its infancy and very complex. In cell therapy, for example, many cells die during the manufacturing process, leading to rising costs and longer production times.
Meanwhile, capacity is limited for biotech manufacturing. Companies have to book a slot two to three years in advance, at a high cost, but what happens during the manufacturing process is a lot of times out of their control. There’s no guarantee that the process will succeed. So, if the process fails, companies have to get in line again.
Moreover, CGT companies must respect national and regional policies, regulations, and work with the relevant agencies to approval. For example, iIn the US, each quality step during manufacturing needs to be confirmed by the FDA.
As the process is so complex, the advice is very clear. Make manufacturing a priority from the start. Ask yourself the following questions: How much product do we need? What does the testing process look like? How will it be approved?
Also don’t forget to align with agencies early on.
What challenges need to be addressed post-launch?
While the six steps outlined above will help to better prepare you for launch, the CGT industry is still relatively new and is always changing. It will be important, therefore, to build metrics that will help you measure your impact and keep track of real-time market dynamics.
Moreover, communicating with and supporting the patient community is something that must continue post-launch. It’s an ongoing process to help patients and their families after treatment, especially considering that results aren’t always immediate. As mentioned above, continued support is vital.
Finally, be prepared to collect long-term data, to ensure that your CGT value proposition is continuously being supported, expanded, and adjusted to market needs.
Given how much there is to prepare, businesses producing CGT must begin the launch process earlier than previously thought – for instance, starting with building manufacturing sites and processes that are in alignment with regulatory authorities.
Market intelligence must be thorough and needs to happen at a granular level – national, regional, and local.
Partner with PAGs and HCPs to help you engage the patient and medical community pre- and post-launch, to provide continued support during treatment.
If you have any further questions regarding CGT launch, contact our experts Simone Seiter and Wolfram Lux.