Treatments for rare diseases have received a boost from advances in cell and gene therapy development. While exciting, the dilemma is how to pay for them, especially when they tend to cost more and treat a smaller pool of patients.

Industry is working with governments and payors to educate them about the value of the therapy, and how it can be cost-effective even with a multimillion-dollar price tag: A one-time, potentially curative treatment vs. lifelong care that not only burdens hospital systems but also patients, caregivers and the economy.

That’s why communicating the value of your drug, especially those for rare diseases, is ever more important.

“The concept of value doesn’t necessarily differ for rare disease versus more common disease. However, it can be more challenging to provide evidence of that value when patient numbers are so small,” Amanda Cole, PhD, Associate Director at the Office of Health Economics, said in an email interview. “Many believe we should be prepared to pay more for drugs that treat rare disease, to address current inequalities in access to diagnosis, treatment and care for rare disease patients.”

Communicating the value of a drug and justifying the price has always been difficult for drug companies, which are facing increased scrutiny over pricing, particularly in the US with the Inflation Reduction Act and amid an election year.

“Cell and gene therapies hold the promise of huge long-term benefits but very high upfront costs,” Cole said. “Even when a therapy is acknowledged to be cost-effective, it may still be unaffordable in the short-term. Coupled with high uncertainty over long-term benefits, there is a need for innovative payment models that could spread payment over time, and also help address decision-uncertainty by offering a mechanism to tie payment to patient outcomes.”

These innovative payment models are gaining traction, particularly for rare diseases in Europe. And with recent approval in the US of the first two gene therapies for sickle cell disease, which address a wider population and can cure a chronic painful disease with a single round of treatment, industry and payors are ever more motivated in figuring out a way to pay for it.

Testimonies from patients who say they have been effectively cured of a lifelong disease that has kept them from going to school or work and has been a daily burden on their caregivers and families speak volumes when it comes to defending the cost of the therapy.

“Patients should be at the heart of any value proposition,” Cole said. “Any concept of value needs to capture the benefits to patients and value to the health care system. Some people think it should also capture value to society as a whole.”

If you would like to hear more about how we can help you craft a communications strategy, please get in touch: healthcare@cardewgroup.com