Over the past decade there has been a groundswell of interest in personalized medicine, where the selection of a particular therapeutic, or its dosage, is individually tailored for specific patient populations based on shared genetic characteristics. Beyond providing patients with more effective care, personalized medicine offers to reduce healthcare costs by eliminating the time and expense associated with ineffective, or even harmful, treatments. Personalized medicine may also allow pharmaceutical companies to recoup resources by identifying subsets of patients that respond to a medicine that failed to prove effective for the patient population as a whole.
These benefits notwithstanding, however, there are a number of issues that may hinder the widespread implementation of personalized medicine. One important impediment is the current legal battle over the ability to obtain patents on isolated nucleic acids, so-called “gene patents,” which generally protect DNA sequences that can predict whether a patient with the particular genetic characteristic is likely to develop a disease of be responsive to a particular therapy.
We currently await the decision of the Court of Appeals for the Federal Circuit in the Myriad Genetics case, in which the American Civil Liberties Union (ACLU) is challenging the patent eligibility of isolated genes that are predictive of breast cancer risk. Opponents of gene patents, like the ACLU, worry that such patents may allow companies to overcharge for diagnostic procedures by prohibiting other companies from offering competing tests, and thus limit patient access to potentially life-saving diagnostics. They argue that what is being patented is not an invention at all, but merely a product of nature. However, a ban of such patents would threaten the monetary incentives that are needed for the development of such tests in the first place.
In addition to the genes themselves, the diagnostic methods based on such biomarkers have also been under attack as being ineligible for patenting. Up to now, the courts have endorsed the patenting of methods that involve either the use of a "specialized machine" or employ a step that includes a "physical transformation," though the ambiguity of these terms has formed the basis of the current legal disputes. The courts have recently clarified that many of the conventional steps used in diagnostics, such as mixing a blood sample with a detection agent, satisfy the transformation requirement, and therefore, methods based on such steps are patentable.
By one estimate, over 4,000 gene-related patents have been granted, including patents on almost 20 percent of the human genome. The existence of so many patents creates what is commonly referred to as a “patent thicket,” posing challenges to developers of genetic diagnostics in obtaining their own patents, as well as avoiding, or licensing, the patents of others. Such patent thickets have made it difficult for researchers to obtain all the patent rights necessary to develop the genetic diagnostics involved in personalized medicine, especially those involving multiple biomarkers.
Significant time and money must be invested to identify all the relevant patents, determine whether licenses are available, and individually negotiate these licenses, which is not always possible. Currently an owner of a minor but necessary patent can leverage it in order to extract higher fees than the patent would normally be worth, resulting in the "holdout" problem. In addition, some patent owners may simply be unwilling to grant any licenses and require that they carry out all testing covered by their patents.
One promising solution to patent thickets is the use of patent pools. A patent pool is an agreement by multiple patent owners to license their patents as a package to anyone willing to pay royalties, which are distributed to the patent owners. In this way, pool members and third parties may license all the patents that they need in one package and avoid taking the time to research the relevant patents and separately negotiate all licenses. Although several biotechnology patent pools exist, such as those for Golden Rice™ and green fluorescent protein, the use of pools in the context of personalized medicine is still in its infancy. However, one company has already begun the process of developing a patent pool for diagnostic genetics testing, and it is likely that similar pools will be developed as this technology continues to mature.
Given the significant benefits of personalized medicine, all stakeholders should consider how best to overcome the current challenges facing its widespread implementation. Whether it is participation in patent pools or otherwise facilitating a change in how the patents are issued and licensed, patients, doctors, and pharmaceutical companies all have significant roles to play in promoting the promise of personalized medicine.
Jennifer Gordon, Steven Lendaris, and Anna Volftsun are lawyers who specialize in life sciences-related patent law. Jennifer Gordon is moderating a panel discussion to review the issues presented above at the BIO conference later this month.