Marc M. Boutin

US House Energy and Commerce Committee chairman Fred Upton (R-MI) and Committee member Representative Diana DeGette (D-CO) announced in April the launch of 21st Century Cures, “a new initiative that aims to accelerate the pace of cures and medical breakthroughs in the United States.”

 The initiative is focusing on 3 key areas—discovery, development, and delivery&mdash;to determine what steps Congress can take to ensure that the country is taking full advantage of the advances made in science and technology to help advance the pace of medical innovation in the United States.

 The patient advocacy community applauds representatives Upton and DeGette for working in a bipartisan fashion to tackle many of the healthcare sector’s most pressing issues to improve the lives of people with chronic diseases and disabilities.

The magnitude of patient need is great. As has been pointed out in this column in the past, more than 133 million Americans—in excess of 40% of the US population&mdash;live with a chronic disease or disability.

 However, for many people there are no treatments, and existing treatments work for only 50% to 75% of the patients who currently use them.

 There are limited treatment options for too many diseases and disabilities, including mental health conditions and neurological, autoimmune, and many rare diseases; there also are limited options for prevention of various diseases and disabilities. Millions of patients struggle daily with conditions such as alpha1-antitrypsin deficiency, amyotrophic lateral sclerosis, Alzheimer’s disease, epilepsy, lupus, mesothelioma, and multiple sclerosis. Many are waiting for a single treatment, while others wait for new and better medicines.

Current policies have not kept pace with the evolution of science in the United States. As Senator Orrin Hatch explained in his recent foreword to the William Mitchell Law Review issue on the anniversary of the Hatch-Waxman Act: While “the foundation laid by the Hatch-Waxman Act 30 years ago will continue to be the mechanism by which the government incentivizes development of lifesaving drugs . . . we cannot rest on the laurels of this legislative achievement. . . . [W]e have an obligation to periodically reevaluate how the balance can be adjusted to account for the sweeping changes in the broader healthcare sector.”

WHY PROMISING MEDICINES DON’T REACH PATIENTS

Two situations currently prevent some promising medicines from being developed and making it to market and to the patients who need them: (1) a complete lack of patent protection and (2) the lack of a predictable and sufficient period of patent protection once the medicine enters the market.

First, the best new medicines do not automatically qualify for a patent, and without any patent protection, manufacturers will not continue developing the treatments, despite their potential to treat unmet medical needs and benefit patients. For any invention to secure a patent, it must be deemed as novel and nonobvious. Although these thresholds to receiving a patent are designed to encourage ingenuity, they have also created barriers to innovation in the drug development process.

As for novelty, “A drug cannot be patented if it was previously disclosed to the public; no exception is made for when the disclosed drug has not yet been tested in clinical trials and thus has not been approved by the FDA.”

 Current law allows seemingly insignificant disclosures to undermine the novelty of drugs, which makes it easy for researchers to unwittingly reveal their discoveries. “Companies [file] overly broad patent applications to establish priority over large numbers of potential new drugs. As their research advances, the companies typically disclaim most of those compounds from their applications.”

 The result is that these potentially valuable compounds, disclosed through the patent process, are in the public domain and now not eligible for patent protection.

Another criterion for receiving a patent is that the drug be nonobvious. “[O]bvious drugs are defined as ones that would have been reasonably expected to succeed at the time of their invention . . . drugs that initially look most likely to be effective are often the least likely to be patentable. . . . [T]he [nonobviousness] standard withholds patented protection from drugs based on the scientific advances that allowed researchers to identify them as ones that are likely to be effective.”

 Without congressional action, the scope of the problem caused by the nonobviousness standard is likely to expand as scientific progress increases the likelihood of success for many products, rendering more and more of them “obvious.”

Second, the unfortunate reality is that manufacturers stop developing a drug when they believe that its patent protection will not extend long enough after the drug enters the market to allow the company to recoup its investment. Because drug manufacturers must apply for patents very early in the research and development process, there can be little or no patent life left when the drug finally enters the market, even with patent term extensions granted through Hatch-Waxman. The longer the drug development process goes on, the shorter the patent term once the drug is approved.

This uncertainty discourages companies from pursuing medicines with long development timelines in favor of those with shorter development timelines. In cancer, for example, this leads to more research on and development of drugs intended to treat later-stage cancers, which often have shorter development timelines.

 Conversely, development of promising drugs that are intended to prevent cancer or treat early-stage diseases but that have longer development timelines is discouraged because of shorter periods of patent protection once the drugs are approved. This leads to increased research and development in the later cancer stages at the expense of the enormous public health benefit of studying drugs to treat early-stage cancer or to prevent cancer. Longer development times are also likely for innovative drugs that could treat a disease that has never had any treatments, for a drug with a new mechanism of action, or for a drug to prevent, cure, or slow the progression of a disease or disability.

House Energy and Commerce Committee Ranking Member Henry Waxman (D-CA) took bold steps to overhaul the pharmaceutical industry with both the Hatch-Waxman Act and the Orphan Drug Act. These laws have made a huge difference in the lives of patients. However, it is once again time for courageous action. We need to realign our economic incentives and policies to encourage the development of treatments for people with unmet medical needs.

Patient advocacy organizations have already begun to address the patent protection barriers to developing new treatments by crafting a bill titled H.R. 3116, the Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network Cures Act of 2013, or the MODDERN Cures Act.

The bill was introduced in the House in September 2013 by Representative Leonard Lance (R-NJ) and is currently cosponsored by approximately 80 Democratic and Republican members of Congress. The MODDERN Cures Act aligns incentives with the needs of patients by setting a term of regulatory exclusivity for medicines intended to treat unmet medical needs. In addition, the legislation encourages the development of innovative diagnostics that help identify which patients will benefit from a specific therapy. The MODDERN Cures Act aims to ensure that the most promising therapies for unmet medical needs are not shelved because of uncertain patent protection.

Specifically, the MODDERN Cures Act provides for a drug or biologic to be designated as a “dormant therapy” if it is a new medicine being studied to treat an unmet medical need. A designated dormant therapy can receive regulatory exclusivity, which protects the drug from generic competition for a certain amount of time after FDA approval. This frees up manufacturers to decide a medicine’s fate not by whether enough patent protection may exist at an unknown date in the future, but by the drug’s potential to benefit patients and enhance the public’s health. This will result in increased research and development into medicines with the potential to prevent disease or disability or treat early-stage conditions.

Finally, the MODDERN Cures Act contains the additional benefit of consistency with the increasing demand that clinical study data be made public (ie, a policy of enhanced data transparency). Many anticipate that increased data transparency will benefit patients by helping to eliminate unnecessary and costly duplication of clinical studies, allowing others to confirm or challenge study conclusions, facilitate learning about existing medicines, and help to inform patient decisions on treatment and physician prescribing—all accelerating research and enhancing patient outcomes. Under the MODDERN Cures Act, dormant therapies receive a set term of regulatory exclusivity, which decreases industry reliance on the use of trade secrets to protect their products. Additionally, the MODDERN Cures Act requires that manufacturers waive patent enforcement beyond the period of regulatory exclusivity, thereby creating a predictable timeline for generic manufacturers to bring their products to market. This bill contains the strongest “antievergreening” protections ever included in legislation, preventing an innovator company from filing new patents for the existing drug to extend its exclusivity rights.

Congress has recently demonstrated its willingness to legislate needed fixes by enacting certain provisions from the MODDERN Cures Act incentivizing innovative diagnostics. These provisions were enacted on April 1, 2014, as part of the Protecting Access to Medicare Act of 2014. The new law establishes a value-based payment system for diagnostic tests and a process for assignment of a temporary reimbursement code to a new test.

All patients who continue to wait for new treatments for their unmet medical needs deserve a modernized regulatory system that incentivizes innovation and helps to bring life-saving therapies to the people who need them. Passing the MODDERN Cures Act of 2013 is a much-needed step to attain the goals of the 21st Century Cures Initiative.

Articles

Congress needs to take the necessary courageous steps to remove barriers to the development of promising treatments that will meet patientsâ€™ medical needs.