Will Right-to-Try Laws Enhance the Importance of Specialty Pharmacy Counseling?

Pharmacists need to be aware of the medication and policy trends governing the use of early-stage drugs that have not been approved by the FDA.

Today’s patient is becoming acutely aware of emerging products that may cure a serious or life-threatening disease. Recently, we have seen the development of a new movement called the “right to try” in which patients who are running out of time and options are fighting to gain access to experimental medications. Needless to say, there is the potential for serious risks to patients who want to use medications that have not been approved by the FDA. On the other hand, patients may view potential risks as worth it compared with their alternatives. The important questions surrounding right-to-try laws are whether it is appropriate to allow patients access to early-stage medications, and if so, who should receive them?

Since some specialty pharmacies already interact with these patients, they may be wondering whether these laws will provide any benefits. We’ll examine whether the FDA has any policies addressing this issue and whether the right-to-try laws provide patients with an easier way to access these medications. Knowing both the medication and policy trends governing this issue can help specialty pharmacies counsel patients on making fully informed decisions going forward.

Before we analyze this issue, it may be helpful to understand how a new drug becomes approved for commercial use. According to the FDA, the process is lengthy¹:

• The first step for a company seeking approval is to perform laboratory tests to learn how the drug works and if it will be safe enough to be tested on humans. The company then submits an Investigational New Drug Application for FDA review.
• Next is a series of clinical trials in humans in 3 phases, which is monitored by the FDA.
• The compiled data then go to the FDA’s Center for Drug Evaluation and Research (CDER) where a team of CDER physicians, statisticians, toxicologists, pharmacologists, chemists, and other scientists review the data and proposed labeling.
• If this review establishes that a drug’s benefits outweigh its known risks for its proposed use, then the drug is approved for sale.

This brings us to the question of whether there are any existing FDA policies that allow patients to gain access to medications before the completion of a New Drug Application (NDA). The FDA has an expanded access or “compassionate use” policy, which is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition. In addition to meeting these qualifications, the drug manufacturer and the patient’s doctor must make special arrangements, authorized by the FDA, to obtain the drug.

While we can understand the patient’s motivation for wanting to acquire these medications, there are other competing interests. Manufacturers may be reluctant to use their product outside of clinical trials for a few reasons. Allowing a patient early access to a product could result in unknown adverse events that would need to be disclosed. Another question manufacturers face is how to determine which patient would get access to the product, in order to avoid the appearance of arbitrary distribution. Manufacturers may have only a finite supply of product available for clinical trials, so producing more could be costly and time consuming, especially when the return on investment remains unclear at that point in production. Additionally, insurance companies may not provide coverage for experimental medication, which creates uncertainty for how the manufacturer will distribute the product to an approved patient. Lastly, the physician would have to both evaluate the risks and closely manage the patient.²

There are other FDA protocols related to this discussion. The FDA has both a breakthrough therapy designation and a fast-track designation that are designed to expedite the development and review of drugs for patients with serious or life-threatening conditions. According to the FDA, the difference between the 2 programs is that the breakthrough therapy designation is for a drug that treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint over available therapies. On the other hand, fast-track designation is for a drug that treats a serious or life-threatening condition and for which nonclinical or clinical data demonstrate the potential to address unmet medical needs for the serious condition.³

The first right-to-try law (HB 14-1281) was signed this past May by Colorado governor John Hickenlooper. The law allows, but does not require, eligible patients to participate in clinical trials and use investigational drugs, biological products, and devices. The bill defines an eligible patient; clarifies that a health insurer is not required to pay for the investigational drug, biological product, or device; and prohibits any action against a physician’s license for his or her recommendations. Louisiana and Missouri also have enacted laws that follow the one in Colorado, and Arizona is expected to consider the issue later this year. While the law’s intent can be seen as getting patients access to medications and providing protections to physicians who make recommendations on these experimental products, it can be argued that these laws aren’t that much different than what the FDA’s policies already offer.

As these laws start to spread nationwide, Congress has begun investigating how to modernize our health care system to get life-saving products into the market faster. This spring, House energy and commerce committee chairman Fred Upton announced the launch of “21st Century Cures,” a new initiative that aims to accelerate the pace of cures and medical breakthroughs in the United States. For the next several months, the committee will examine this process from discovery to development to delivery to understand how Congress can ensure that the United States maintains its innovative edge.

So where does this leave specialty pharmacies? With specialty drug spending expected to dramatically accelerate over the next few years, it is no secret that specialty will play a key role in patient care. In addition to coordinating patient clinical care, you may be asked for your guidance on whether there are options for patients to access experimental medications. Currently, there is a hole in the system: patients don’t understand why they can’t try experimental medications that may save their lives, while the manufacturers and the FDA don’t have the data they need in order to predict how these experimental medications interact with other medications these patients may be taking. Since specialty pharmacies see the entire picture, the opportunity may exist for specialty pharmacies to provide valuable information that could both help quicken the drug’s evolution and enhance current patient care. SPT

References

• How FDA evaluates regulated products: drugs. FDA website. www.fda.gov/AboutFDA/Transparency/Basics/ucm269834.htm.
• Access to investigational drugs or biologics outside of a clinical trial (expanded access). FDA website. www.fda.gov/forpatients/other/expandedaccess/default.htm.
• Frequently asked questions: breakthrough therapies. FDA website. www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/fdasia/ucm341027.htm.

About the Author

Ron Lanton, Esq, is the owner of True North Political Solutions LLC, a health care government affairs firm based in New Hampshire. Ron has worked in government affairs including various activities on the municipal, state, and federal levels. Most recently, he worked for H. D. Smith, where he created and oversaw the wholesaler’s government affairs department, served as its exclusive lobbyist, and also advocated for the company’s health care customers. Previously, Ron worked at a government affairs consulting firm in Arlington, Virginia, where he worked on health care, energy, commerce, and transportation issues. He also clerked for a federal magistrate, was appointed as a municipal commissioner on environmental issues, and has consulted Wall Street firms on financial issues. He has been a featured industry speaker on issues such as pharmaceutical safety and health care cost containment. Ron holds a Juris Doctor from The Ohio State University Moritz College of Law and a Bachelor of Arts degree from Miami University of Ohio. He is also a “40 Under 40” award recipient.