Gene and Cellular Therapy Update

Precision medicine is growing at a rapid rate producing novel gene and cellular therapies, which are targeted to be curative treatments for genetic, oncologic and rare diseases. Within the next five years, the landscape of gene and cellular therapy is expected to change dramatically; bringing more treatment options to patients. The FDA anticipates there will be many more cell and gene therapies as more than 900 investigational new drug (IND) applications will be submitted for ongoing clinical studies in this area¹. These therapies are expensive to develop and manufacture; with a recent therapy costing $2.1m for a one-time dose². Industry leaders have partnered to collaborate regarding strategies to address access, scaling manufacturing and overcoming financial hurdles associated with bringing these costly therapies to market.

According to the Alliance for Regenerative Medicine (ARM) 2019 annual report³, it is estimated that 4,500 – 5,000 people have been treated with FDA and EMA approved gene therapies and gene-modified cell therapies. Gene and cellular therapy clinical trials continue to grow at an exponential rate with over 1,000 active trials in 2019 alone⁴. The number of patients targeted for enrollment in regenerative medicine clinical trials has climbed to 60,000+ globally. In an effort to meet the rising demand of FDA approvals for oncology drugs, gene and cellular therapies, the FDA has created an Oncology Center of Excellence, aimed at improving efficiency of review and transition from translational science to market. Based on the success of this program, efforts are underway to create a new Center of Excellence for Rare Diseases⁵.

Costs for gene and cell therapies become even more substantial when there is a large patient population. Using sickle cell disease as an example, with an estimated 100,000 sickle cell disease patients nationwide⁶, a gene therapy priced at $1 million per person, would cost $100 billion. Major insurers are developing new strategies to help mitigate the cost of gene therapies including installment payment plans, per-member-per-month service fees to access gene therapy networks, gene therapy coverage stop-loss programs, and performance-based rebate programs. Just one individual receiving a $1 million therapy is too much for many small employer self-funding plans to absorb.

Some health economists believe that there will be a mixed approach to funding high cost therapies, depending upon the nature of the disease state and what can be expected of the clinical benefit. If gene therapy completely eliminates the need for high cost chronic therapy in diseases like spinal muscular atrophy or hemophilia, milestone payments may work best. Whereas something like Luxturna® (voretigene neparvovec), for which there are no treatment alternatives, but has a very small market, might work best with a performance-based rebate.

The cost for research and development, gene and cellular therapies pose unique challenges for manufacture scaling, including shorter shelf lives, temperature sensitivities, complexity and cost related to purity and identity testing, synchronized collection, modification, and delivery of biologic material. ARM has collaborated with industry leaders to create manufacturing best practice guides, which aim at addressing these challenges and remove barriers to scaling up production. The over-arching goal is also to drive down manufacturing costs to make these therapies more affordable and improve access.

In order to meet the expected growth and demand for gene and cellular therapies, efforts are underway to remove and mitigate barriers to state adoption of payment-over-time and outcome-based agreements for advanced therapy medicinal products. Value-based payment models have been developed to propel regulation and legislation to improve access to Medicare and Medicaid recipients. ARM worked with the FDA to successfully drive the refinement of CMS guidance for regenerative medicines and to develop indication-specific guidelines for gene therapy development. Conversely, reinsurers are also analyzing pricing models and strategies to address the expected frequency of these costly therapies. While there is no doubt that we will continue to see more of these therapies come to the market, finding creative and collaborative solutions to address the many challenges these costly treatments pose on the health care delivery system will require leadership amongst pharmaceutical companies, insurers, reinsurers, legislators and health care providers.

Contact your PULSE + Plus® Clinical Consultant for more information on gene and cell therapy approvals as well as potential savings opportunities.