Cell and Gene Therapy Holds Great Promise, but Not Without Hurdles

Editor’s note: Dan Schroen, head of Photo 51, a consultancy focused solely on advanced therapeutics such as cell and gene therapy, virtually attended the Reuters Events: Cell & Gene Therapy USA 2020 conference. Below is a conference recap, including the current state of the industry and the challenges being faced.

In a space that was unthinkable just years ago, the advent of cell and gene therapy (C&GT for our purposes) represents one of the most exciting, remarkable, revolutionary points of time in healthcare, characterized by energy, paradigm-shifts, and large investment. Sometimes called “medicine 2.0 driven by omics,” C&GT is forging new space as the next wave of medicine, the new promise. Early research and progress in C&GT is now coming to fruition, with learnings from monoclonal antibodies (mAbs) and recombinant protein showing the ability to address many technical, operational, cost, and performance considerations.

C&GT can be indicated for patients of rare diseases who are refractory to current treatments, have relapsed, or have few to no other treatment options. These advanced therapies have been able to demonstrate remarkable benefits to patients, particularly in terms of clinical efficacy, overall response rate, and durability. These are life-changing—potentially lifesaving—therapies with the possibility to cure disease, not just treat it. For instance, with its ability to replace or repair DNA, gene therapy can offer long-term and potentially lifelong effects from a onetime treatment. In this sense, gene therapy differs markedly from traditional small molecules, which typically act as inhibitory agents. Conversely, most genetic diseases involve loss of function, so gene therapy can add function back.

C&GT treatments are increasingly going to market, and the number of patients served is growing dramatically. By the numbers, 17 products have been FDA-approved since 2019, with 39 potential new therapies by 2022, and more than 300 therapies in development for chronic and rare diseases. Affecting 350 million people worldwide, there are 7000 known rare diseases, and 80% of them are genetic. More than 100 rare diseases are being explored, with 900 investigational new drugs (INDs) filed, 362 in development, 132 for rare disease, and 6 approved so far. About 200 INDs are expected annually in the coming years, with an estimated 50 to 75 therapies approved for the US market by 2030.

Challenges, hurdles to overcome

With this new promise comes disruption and unique issues and challenges to overcome, thus requiring an agile, innovative mind-set. For instance, the diseases treated by C&GT often involve long treatment odysseys, where thought leaders may be the specialist caregivers delivering the targeted therapies to patients. C&GT also differs from traditional drugs in terms of supply chains and manufacturing, and they are often at odds with traditional healthcare delivery, pharmaceutical business models, and revenue streams. There exists a need to balance the capacities of both manufacturing facilities and treatment centers so there is neither undersupply nor oversupply. Questions are often asked of how the industry can supply more value, streamline development, and launch and get more patients treated with C&GT. The adage holds true that “new treatments can’t squeeze into old systems.”

Apply automation, digitalization, electronics, data-based decision-making

With C&GT, there’s an increased need and urgency across the entire value chain to digitalize experiences, use data and analytics, and provide the tools to increase availability of treatments. There are a lot of different moving parts to an organization, with many factors in the value chain that rely on one another and the need to ensure each function has the right information. Manual approaches take too much time and are prone to human error, so digitalization can create seamless, end-to-end experiences. 

Cooperate, collaborate, communicate, integrate, partner

Complex diseases and therapies require frequent communication, data sharing, cooperation, and trust between all key stakeholders. Working together is more important than ever, especially considering the uniqueness of C&GT. This comprehensive, highly integrated, cross-functional collaboration is critical among all groups and functions, including technical, medical affairs, patient advocacy, policy, program management, admins, nursing, financial, physicians, technologists, pharmacists, billing and coding, and data management.

Internal partnerships are important, as companies need to galvanize organization internally to get it right—sooner, better, and by using the forces of agility and innovation. 

Strategic partnerships with different companies and entities, large and small, can be key to the success of C&GT, and they are anticipated to increase. Such external collaboration and inter-company alignment are still relatively rare, so it would be great to see more unification and better communication in order to break down silos. As one example, collaboration with treatment centers can help develop infrastructure through rapid certification and process design. And teams shouldn’t forget to forge collaborations with national and state governments.

Be patient-centered 

In this era, patients and their needs are paramount, and this is especially true with C&GT. The goal here is to deliver the right treatment to the right patient, at the right place and time, within guidelines, using trained experts, with reimbursement worked out, to deliver successful clinical outcomes. C&GT demands a wholistic patient experience, focusing on what and who they experience during treatment, including healthcare practitioners (HCPs), nurses, staff, points of care, attention to small details, and quality of life during treatment. Continue to include advocacy organizations to answer patient questions, as they are a conduit to the patient. Peer-to-peer patient communities are changing how patients interact, exchange information, and reach others.

Technology advances, transformation

Innovations and new technologies in the C&GT space are providing opportunities for process improvement, time savings, cost reduction, and for new novel science and treatments to move ahead. Consistent delivery of C&GT at treatment centers is improving the outpatient experience and value, while reducing cost of care, services, and follow-up activities. Anything that streamlines and benefits healthcare is good, in terms of streamlined supply chain, quick treatment, affordability, safety, freed-up outpatient beds, and fewer visits. Evolution of platforms will differ depending on the therapy, but regardless, more mass-customization is needed as opposed to specialized, handcrafted approaches. Technology and equipment innovations spur progress in the scale-up of C&GT and help reduce their cost of goods. C&GT will see jumps in technology from one stage to another, and not just incremental, stepwise volume increases as we’ve seen with mAbs.