Precision medicine and genome editing-based therapies offer great promise but also come at great cost: one recently developed medicine, for example, costs $1,00,000 for a single dose.   Professor Anya Prince addressed the promise of genome editing and the issue of paying for it during her Innovation, Business & Law Center faculty speaker series talk on Thursday, September 27, 2018. Professor Prince’s talk, entitled “Who Pays? Access to Healthcare and Gene Editing Technology,” explored the technology underlying gene editing, the reasons for the great expense of gene editing, how gene editing treatments might be paid for, and the challenges for the payment systems that would cover the cost of these treatments.

 

Gene editing technology

            Professor Prince explained that humans have performed forms of gene editing for thousands of years by breeding crops and animals to favor certain traits. That process takes generations to obtain the desired traits. Modern methods of gene editing, however, promise immediate genetic changes that can benefit patients. The discovery of the current generation of gene editing technology, CRISPR/Cas9, opens that promise even further. As Professor Prince stated, “[i]n recent years, the FDA has begun to approve several gene therapy treatments—an area of clinical care that we can only expect to grow with the advent of gene editing advances, like CRISPR.”

 

            CRISPR/Cas9 is a group of molecules, specifically polynucleic acids and proteins, that can home in on a desired genetic sequence, its “target,” and change it to a different sequence. The power of CRISPR/Cas9 lies in two of its features: its efficiency in making the desired change and the ease of designing CRISPR/Cas9 treatments for different targets. This allows the correction of genetic defects that cause many human diseases.  

 

“If you build it, will we pay?”

            Professor Prince posed that question and explained that numerous cost drivers cause the high price of genome editing technologies, making paying for the therapies a financial, moral, and ethical quandary.

 

            The main cost driver lies within the genetic specificity of genome editing, which allows each genome editing therapy to typically be useful in treating only a small number of patients. Many of the most common human diseases are multifactorial; that is, they are caused by a multitude of genetic and environmental factors. Examples of this include heart disease, diabetes, and most cancers. Conversely, some diseases—such as Huntington’s disease, cystic fibrosis, and hemophilia—are caused by just one genetic change. Genome editing is more suited for correcting the single genetic changes that cause the latter group of diseases. The limited number of patients increases the cost of genome editing therapy due to the increased per-treatment regulatory approval costs.

 

            Further, the curative aspect of genome editing therapies is a disincentive for insurance companies to cover such therapies. Many genome editing therapies are cures for lethal or lifelong diseases. An insurance company would favor paying for an expensive cure if it would save the company money long-term. However, patients frequently switch insurance companies, thereby disincentivizing the insurance company from paying for expensive therapies. Basically, why would one insurance company pay high upfront costs now for a preventive or curative treatment if another insurance company would be the cost beneficiary of the treatment after the patient switches insurance companies?

 

Gene editing therapy payment options

            The standard payment structure for disease treatment is for an insurance company to pay for the treatment at the time of treatment.  As this approach is problematic for the reasons just discussed, various alternatives have been proposed, at least as theories. Professor Prince described several payment options that have been proposed for expensive genome editing therapies:

 

Pay for performance

      An insurance company would only pay if the treatment is effective in the patient, or the company would pay a reduced price if the treatment is ineffective. Alternately, the insurance company could pay in installments for as long as the treatment is effective (an example would be paying for a cancer treatment as long as the patient is in remission).

 

      However, there are drawbacks to pay for performance. First, comorbidities—the presence of more than one disease in a patient—can affect the efficacy of a treatment. Second, it is not clear when payments should be made, how to measure effectiveness, or when to measure effectiveness.

 

Annuities

      An annuity, or loan-like structure, could lower the upfront costs for a patient. However, the patient still would have to pay for the treatment. Further, the patient would have to pay interest and could face default if unable to pay.

 

Reinsurance

      Reinsurance could provide insurers a back-up that would permit them to cover expensive genome editing therapies. However, Professor Prince explained that some reinsurance companies have already stated they would not cover such therapies. Reinsurance also does not put direct pressure on the pricing of genome editing therapies, as the company providing the reinsurance is not the one paying for the therapy.

 

Challenges to patients

            Patients already face challenges in paying for medical treatment, and the costs of genome editing therapies could potentially exacerbate disparities amongst patients with respect to their medical treatment options.

 

Who can pay

      Even with insurance coverage, medical treatments can be expensive. Patients usually have to pay for a deductible, coinsurance, and a copay. Even if an insurance company agreed to pay for the treatment, many patients would be unable to pay the required costs. Refusal to pay by insurance companies would cause even greater disparities between who can afford treatment and who cannot.

 

Pre-existing conditions

      Genetic predispositions to disease can be a unique type of pre-existing condition. Even before manifestation of disease symptoms, genetic testing of a patient (or even the patient’s family) can indicate that the patient will get a disease later in life. The Genetic Information Nondiscrimination Act currently prevents such use of genetic information, and the Affordable Care Act offers protections for those with pre-existing conditions. However, laws can change, and genetic predispositions to disease could someday cause further disparities in access to health insurance and care.

 

            According to Professor Prince, “[t]hese [gene editing] treatments [] come with hefty price tags that may not be covered by insurance. Without equitable access to such treatments, we may find ourselves in a society where some can afford to cure their condition, while others are ‘stuck’ with a treatable illness, further entrenching existing health disparities.”

 

Perspectives

            Genome editing technologies offer the promise to treat multitudes of diseases, along with opportunities for those interested in the business and law of healthcare. Decisions will have to be made on how to pay for the great promise of new and future technologies. Today’s students will help make those decisions and affect the future of healthcare.

 

 

            Professor Anya Prince is an Associate Professor of Law and Member of the University of Iowa Genetics Cluster. (For more information, see https://law.uiowa.edu/anya-prince.)

 

            For more information on CRISPR/Cas9, see https://www.youtube.com/watch?v=2pp17E4E-O8.