Cell and Gene Therapies

Cell and gene therapies have the potential to cure disease in a one-time administration by targeting a patients’ cell or genetic source. Since the first FDA approved cell-based gene therapy in 2017 (a CAR T therapy), these medical technologies are transforming treatment for patients, including those with cancer and rare pediatric disorders.

Rather than using chemical compounds to treat disease, these therapies fight diseases at their source. Today there are a total of five FDA-approved CAR-T therapies (which use the body’s own immune system to fight disease) and two FDA-approved gene therapies.

Gene Therapy

A patient’s genetic material (such as DNA) is used to treat, cure or even prevent a disease.

Cell Therapy

Cells are cultivated or modified outside the body before being injected into the patient to treat a condition in which the patient’s cells are damaged or diseased.

The Complex Cell and Gene Therapy Production Process

Most conventional drugs can be produced in advance, stored in inventory and distributed when needed. With cell and some gene therapies, each treatment is unique because it starts with the individual patient’s own cells. They must be developed rapidly and administered to patients immediately. Because the production process is highly complex and individualized, quality controls are a unique consideration and require a new level of manufacturing sophistication. In most cases, the production processes for cell and gene therapies are still being optimized.

Only very specialized facilities with strong regulatory and quality controls can safely manufacture cell and gene therapies. Challenges to delivering cell and gene therapies to patients include building a specialized workforce for this highly complex manufacturing process and developing sophisticated logistics to support the time-sensitive transfer of biological materials. Investments by the biopharmaceutical industry in the coming years are likely to bring breakthroughs that will standardize manufacturing processes, simplify delivery logistics and increase competition.

The Value of Cell and Gene Therapies

Value assessments are an important tool in moving away from a traditional fee-for-service model toward better, more value-driven health care. Unfortunately, conventional value assessments, also known as health technology assessments (HTAs), typically only account for health care costs, and rely on controversial and inherently discriminatory metrics like the quality-adjusted-life-year (QALY). Furthermore, traditional, population-level methods of value assessment are particularly ill-suited for the valuation of cell and gene therapies, which are highly personalized and are anticipated to result in significant cost offsets.

Patients may experience long-lasting or even curative effects from just one administration of many cell and gene therapies, but there are unique considerations for the way cell and gene therapies are evaluated during development because of the small patient populations and rare diseases they are intended to treat. As a result, stakeholders have advocated for new ways to measure outcomes of interest most relevant to patients under treatment to better inform regulatory and payer decisions including patient-reported outcomes and real-world evidence.

Improving Patient Access and Affordability

Early estimates affirm that cell and gene therapies will remain largely affordable to much of the health care system. For instance, the U.S. market for oncology medicines is expected to be upwards of $100 billion by 2024, compared to $4 billion for cell and gene therapies for oncology. And we can make these treatments accessible and affordable by advancing innovative approaches to payment and moving toward a value-driven health care system.

To help improve access and affordability, biopharmaceutical companies are working with other health care stakeholders, including payers, to develop new ways to pay for medicines, like innovative contracts. These flexible payment arrangements include innovative contracts – also known as outcomes-based contracts or alternative financing arrangements – for biopharmaceuticals. The contracts are voluntary arrangements between manufacturers and other entities, such as health plans or risk-bearing providers, in which the price or price concession for a prescription medicine is linked to the value the medicine provides for the patients. These arrangements have the potential to lower costs and increase patient access through voluntary, market-based negotiations – as opposed to government or centralized value assessment. These contracts can function as a solution provided key barriers are addressed.

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