PhRMA’s Anne McDonald Pritchett PhD highlights the remarkable development of cell and gene therapies in recent years and the three ways in which the US healthcare system can better accommodate them: creating manufacturing systems that take treatment timelines into account, a health system that values treatments based on patient outcomes, and new approaches to financing.


These new therapies have the potential to cure previously incurable diseases and to fundamentally alter the trajectory of many other life-threatening conditions, including many rare diseases

It was only in the 1980s that scientists first began researching the potential of gene therapy to cure genetic disorders. Today, scientists and researchers at America’s biopharmaceutical companies are developing nearly 300 cell and gene therapies to treat cancers, neurological diseases and more.


Gene and cell therapies use genes and cells to treat disease. This sounds simple, but the reality could not be more complex from a scientific and clinical standpoint. With gene therapy, the treatment involves making an addition to a gene or altering a gene. With cell therapy, cells are taken either from the patient themselves or a donor and may be genetically altered to treat disease. These treatments are often referred to together because the treatment may involve removing cells from the body, treating them with gene therapy, and then putting them back into the patient.


These new therapies have the potential to cure previously incurable diseases and to fundamentally alter the trajectory of many other life-threatening conditions, including many rare diseases. In fact, more than 70 percent of the gene therapies in development are for rare diseases.


Take sickle-cell disease as just one example which impacts approximately 100,000 Americans. The disease is life-threatening, due to potential complications from blocked blood vessels, which can include stroke, difficulty breathing, pulmonary hypertension and other organ damage. Today, scientists are exploring new ways to use established medicines and cutting-edge technologies such as RNA interference, gene-edited stem cell therapy and gene therapy. One therapy in the development could potentially be a one-time treatment for sickle cell disease – and this is just one of the nearly 20 sickle-cell disease therapies in development or in US Food and Drug Administration (FDA) review.


These therapies are the result of decades of research and are incredibly challenging to research, develop, manufacture and deliver to patients.


While these therapies reflect harnessing of the very latest scientific advances and many are potentially one-time curative treatments, our current healthcare system is not structured to handle potential one-time therapies. Additional challenges for the current healthcare system are that many of these therapies need to be delivered as early as possible to patients to provide maximum clinical benefit, which can be complicated by prior authorization policies for these therapies, and the challenges for insurance plans in estimating how many patients may need these therapies in a given year.


PhRMA and our member companies are focused on identifying ways to further evolve the US healthcare system to ensure continued headroom for these innovative therapies.


First, we need manufacturing systems that take treatment timelines into account. Biopharmaceutical companies are continuing to explore existing and new technologies to more efficiently and cost-effectively develop and manufacture cell and gene therapies, including exploring the feasibility of manufacturing therapies at the point of care.


Second, we need a health system that values treatments based on patient outcomes. Biopharmaceutical companies are increasingly exploring innovative-contracting arrangements in which payment reimbursement for new treatments is tied more closely to patient outcomes—research is showing that these types of contracts can reduce health system and out-of-pocket costs and improve patient access and health outcomes.


Third, we need to continue to explore new approaches to financing these medicines. Just as there won’t be one size fits all gene and cell therapies, there is not a one-size-fits-all approach to payment. Biopharmaceutical companies and payers are exploring a range of creative approaches from innovative contracting arrangements that can include value-based arrangements, extended payment plans, and annual subscription plans.


As stated by the FDA, some of these therapies “are almost certainly going to change the contours of medical practice, and the destiny of patients with some debilitating diseases”—it is critical that we work together to develop a coverage and payment system that can ensure timely patient access, manage short-term affordability challenges, and continues to foster the development of these new treatments.