Uncovering behind-the-scenes challenges in bringing gene therapies to market

More than 900 gene therapies are in clinical development. While each of these specialty medications has the potential to change lives, they typically come at a high financial cost to patients, employers and health plans.

The U.S. Food and Drug Administration (FDA) expects a steady increase in gene therapies coming to the market – but the complexity around research and development and other aspects of the gene therapy pipeline have caused timelines and predictions to shift. Here are some reasons the gene therapy development pipeline is ever-changing and complex.

Research and development of gene therapies

Any new pharmaceutical requires a rigorous research and development (R&D) process. The steps from initial discovery to clinical trials and then FDA approval can take years to complete, with clinical trials alone taking an average of six to seven years. This brings the average cost to research and develop a successful drug to $2.6 billion.

The average cost soars to $5 billion to research and develop a gene therapy, which is a highly targeted medication that introduces genetic material into a person’s DNA to “edit” and replace faulty or missing genetic material that leads to disease. Gene therapies also take longer to bring to market than other drugs, with the latest estimates pointing to a 15-year minimum for R&D. Gene therapies that have a rare or orphan status – meaning they are used to treat extremely rare diseases – have an even longer timeline.

The higher cost for researching and developing gene therapies may also be passed on to patients and their insurance providers. One gene therapy treatment can carry a six- to seven-figure price tag.

Developments in gene therapies represent medical innovations that advance the health care model. While not all patients will be appropriate candidates for these therapies, they are potentially curative, preventing the need for long-term adherence to complex maintenance medications and improving patients’ quality of life. For example, there have been a multitude of success stories around gene therapies to treat spinal muscular atrophy (SMA), a rare condition that damages nerve cells in the brain and spinal cord, affecting a child’s ability to crawl, walk, sit and control head movements. Gene therapies for SMA have proven to help children stand and walkwithin developmentally normal age ranges, which significantly impacts their overall quality of life.

Smaller patient populations for gene therapies

Finding patients to enroll in clinical trials for gene therapy can be challenging, particularly for rare diseases that may have been previously untreatable. For example, there is a product in the gene therapy pipeline for AADC deficiency, an ultra-rare genetic disorder. It affects how cells send signals to each other. People born with this condition don’t make enough aromatic l-amino acid decarboxylase (AADC). That’s an enzyme that helps the brain and nervous system work. The incidence is 1 in 11 million. Another example is X-linked adrenoleukodystrophy, a genetic disease that affects the nervous system and the adrenal glands (small glands located on top of each kidney). People with this disease often have progressive loss of the fatty covering (myelin) that surrounds the nerves in the brain and spinal cord. The incidence is 1 in 318,000 male births.

According to McKinsey, rarities like these cause “intense competition for a limited patient pool to treat.” That competition impacts R&D timelines – particularly at the clinical trial stage – and overall costs.

Gene therapy delivery

Supply chain demands also pose challenges for gene therapies. PWC explains it well: “Gene therapy companies often must rely on a robust supply chain with advanced capabilities.”

One key capability is referred to as a “cold chain,” which ensures that products get temperature monitoring along the journey and are stored and shipped properly, from when they leave the manufacturer to their arrival at a specialty pharmacy or distribution center, all the way until they are administered to the patient. “A single temperature failure in the supply chain could render the product useless, even dangerous,” PWC writes. These products may need storage to the extreme – in one case, the product must be stored at less than negative 65 degrees Celsius. This requires unique storage and shipping equipment and monitoring.

Other examples of advanced capabilities may include logistical safeguards to ensure that the product reaches the patient’s site of care at the right time, compliance with federal supply chain regulations, and the ability to provide practitioners and patients with timely information and updates.

Specialty pharmacies like Accredo are well-positioned to handle, store, and dispense therapies for people living with rare or complex health conditions. Beyond their supply chain capabilities, specialty pharmacies support very complex and diverse patient needs across a spectrum of rare disorders. They provide high-touch patient care and are adept at navigating unique drug requirements for very sensitive and expensive therapies.  

Plan sponsors and gene therapies

Understanding more about the challenges faced in bringing rare, innovative therapies to market can help plan sponsors better understand the dynamics of the pipeline and therapy availability for patients. An awareness of gene therapy complexities also helps to better understand the many critical steps that need to be carefully coordinated in order to deliver treatment to a patient.

Plan sponsors should be planning accordingly for how their organizations will approach gene therapies as they become more prevalent and available. Part of doing that is ensuring you’re working with a partner who can model impact to your plan in a sophisticated way and who can help you manage your costs more predictably in the future.