The cost of curing blindness: Navigating challenges on the road toward curative treatment

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In a massive ballroom in Kansas City in late January, a panel of medical experts with the Midwest CEPAC, a core program of the Institute for Clinical and Economic Review (ICER), reviewed the effectiveness and value of Spark Therapeutics' new gene therapy Luxturna (voretigene neparvovec) for an ultra-rare eye disease.


The disease, called RPE65-mediated inherited retinal disease, causes severe visual impairment in children and adolescents, and nearly all become fully blind in adulthood. Picture living with lights on, then total blackness.


It's evident that even a few years of delayed vision loss can have significant advantages for individuals faced with this devastating disease. Prolonged vision buys these teenagers time to complete their education, prepare for a life of blindness, and become contributing members of society as employees, caregivers, and taxpayers.


There is no long-term overall survival data to validate Luxturna as a cure for patients, but anecdotal data suggests the treatment has the potential to delay vision loss indefinitely. That seems like an extreme value for a drug that is indeed priced at a premium. The ICER review process works to substantiate the often substantial price tags of new therapeutics.


Curative therapies will influence the way drug companies market their products. We're already seeing this in HepC with the availability of medications with a cure rate of 90% to 100%. Sitting in the ballroom during the CEPAC meeting, the challenges communicators face on the road toward curative treatment were clear.


Proving a lifelong cure

Gene therapy could have a potentially paradigm-shifting impact on healthcare. In most cases, the goal of gene therapy is to cure with a one-time and lifelong treatment. The word “cure” has been a lofty goal for researchers and patients for years. We're finally getting there with gene therapy, as well as treatments in other diseases like HepC and some forms of cancer.


This presents some unique challenges for advisory bodies such as ICER, whose cost-value framework is contingent on the amount of time the drug is effective compared with the current standard. For potentially curative therapies, however, there is limited, if any, existing data to prove true lifelong effect. This uncertainty of durability leads to challenges in establishing value. As more gene therapies promising lifelong effects with a single treatment come to market, pricing and durability disputes will continue to rage.


Basing data on novel endpoints

The lack of long-term efficacy data for potentially curative therapies ushers in the need for novel endpoints, which present their own set of challenges. Frequently, drug developers work with the FDA to create measurements that have not existed as endpoints before. This is a challenge for ICER, which argues these novel endpoints aren't validated in a real-world setting and therefore shouldn't be included in their cost effectiveness model. That skews the whole model, giving an incomplete picture of value versus cost.


As science evolves and drug developers create more first-in-class therapies with no established standard of care with which to compare, novel endpoints may become the norm.


Value is the name of the game…

Developing and manufacturing cures is a costly process, which in most cases contributes to the high price of curative treatments, but it also comes down to value. What is the value to the patient who no longer spends the rest of his or her life with a potentially deadly viral liver condition? Or without the guarantee that they one day will go completely blind? Value can be subjective, especially when it's impossible to measure the durability of a treatment, but the patient voice is powerful and helps bring into focus the everyday reality of living with debilitating conditions that drug developers seek to cure.


…But the rules of the game are changing

Communicators will quickly discover that the secret for ushering curative treatments through cost-value review bodies like ICER and regulatory agencies, such as the FDA, is to demonstrate the value to the whole person. We must consider all aspects of the patient journey, and the impact cures have on patients, caregivers, and society as a whole.


We will do that by engaging the patient community early and often in the drug development process. It is in the hands of drug developers to do their due diligence to identify and amplify the patient voice. Participate, listen, and learn.


Carolyn Sobczyk is VP at JPA Health Communications. (She works with clients in gene therapy, but Spark Therapeutics is not a client of JPA). 


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