From Patents to Patients: Rethinking Drug Patent Rights for a More Accessible Future

Cuauhtemoc Ulises Gonzalez, PhD Candidate University of Texas MD Anderson Cancer Center at the UTHealth Houston Mission

■ Reduce drug prices in the United States

■ Major Policy Recommendations to Congress

1. Remove the sole production rights obtained from a drug patent and allow generics to go through FDA approval sooner.

2. Ownership rights are maintained from a drug patent and generics must pay royalties to the drug patent owner for an extended duration longer than the current 20 years.

Introduction: Pharmaceutical patents

The Patent Act of 1952 was signed into law by President Harry S. Truman to establish the provisions and principles surrounding patent rights in the United States. Since its establishment it has been amended to adopt a 20-year patent term from the initial 17-year patent term. In addition, to address concerns regarding access to affordable drugs and to continue fostering the innovation of drugs, the Drug Price Competition and Patent Term restoration Act of 1984 was enacted, also known as the Hatch-Waxman Act. While the 20-year term derives from the Patent Act, the Hatch-Waxman Act set up a pathway for generic drugs to be developed and introduced before the expiration of patents if they can prove the patents are invalid, unenforceable, or that the generic will not infringe on the patent. Lastly, the U.S. Food and Drug administration is the regulatory agency in charge of maintaining the safety, efficacy, and quality of drugs marketed in the United States.

Current Challenges: Drug patents promote drug price hikes and obstruction of competition.

Based on 2017 statistics, the United States sits at the top in pharmaceutical expenditure with $1220 price per capita when compared with the next four countries’ price per capita of $963, $838, $823, $806 which is 21%, 31%, 32%, 34% cheaper than that of U.S., respectively. This has negatively impacted the people of United States as it limits access to healthcare for those in lower socioeconomic status. While high drug prices in the United States compared to other countries can be attributed to multiple complex factors, one factor that is rarely discussed is the large investment required to produce drugs. Pharmaceutical companies are invested in multiple drugs at a time as it is estimated that 0.1% of these drugs reach the counter, and it can take 10+ years for one drug to reach the counter due to the required FDA regulations that keep our drugs safe, effective, and high-quality. This leaves pharmaceutical companies with less than 10 years to recover their investment and profit to continue drug research and development, forcing companies to inflate their prices during their patent lifespan. This is in addition to the returns expected of investors.

Pharmaceutical companies have also found ways to circumvent the 20-year lifespan of a drug by including patents surrounding the drug formulation, delivery, and equipment. This allows them to extend their patent and prevent generics from being produced and competing. Because competition allows drugs to be cheaper to the public due to cost and demand, action must be taken to tackle the issue of pharmaceutical companies needing to recoup their investments within a 10-year timeframe.

Proposal: Win-win for the public and drug companies.

While the Hatch-Waxman Act aimed to address drug accessibility and promote the free market, it does not do this sufficiently. Therefore, I propose congress make policy changes on the patent system set for new drug development. To benefit U.S. citizens and pharmaceutical companies, the following aim must be met.

Reduce the cost of drugs while continuing to incentivize drug development.

To reduce the cost of drugs, generics must be allowed to initiate production earlier than allowed by the current patent system; therefore, the sole production rights generated by a patent must be removed, but rights to ownership should be unaffected. Drug must be allowed to be produced as a generic by the time it is FDA approved. Generics will still have to comply with current FDA approvals.

To keep pharmaceutical companies motivated to continue Research & Development programs, pharmaceutical companies will have appropriate royalties paid by generic drug producers for a longer lifespan than then currently set by patents but no longer than 50 years. This will allow pharmaceutical companies to continue to have profit and incentive for further drug development.

Limitations: Economic analysis

Not enough studies have measured the lifespan use of an FDA approved drug before it is replaced by a newer drug or better treatment, therefore, it may be difficult to quantify the appropriate royalties. However, royalties should be calculated so pharmaceuticals can recover costs in 40 years after FDA approval and recalculated as more generics enter the market. This allows pharmaceuticals to spread the same profits which would be generated in 10 years to 40 years but reduce the cost to the public. However, the specific details of these proposals would require careful consideration and economic analysis by government officials.

Implementation: Reward successful drugs

To initiate the new method and motivate pharmaceutical companies to transition, pharmaceutical companies would be allowed to apply for small NIH funded grants for newly developed FDA approved drugs that aim to reimburse drug developers a small fraction of the cost and aid new research and development. In a sense, reward drug developers who have produced a novel FDA approved drug and have allowed generics to be produced immediately after FDA approval.

Conclusion

In conclusion, the current pharmaceutical patent system, along with the Hatch-Waxman Act, have tried to balance drug accessibility and innovation. However, challenges remain in ensuring affordable drug prices and promoting market competition. I propose congress to make policy changes that address these challenges by allowing earlier production of generic drugs while still incentivizing drug development through longer royalty payments. These policy amendments, along with additional policy changes, have the potential to benefit both the public and pharmaceutical companies. Allowing pharmaceutical companies to be apply for small reimbursements by the NIH can further incentivize pharmaceutical companies to transition towards more affordable and accessible drugs. By addressing these issues, along with other issues in healthcare, we can foster a healthcare system that ensures access to affordable medications while encouraging innovation for the benefit of patients and society.

From Cells to Humans: Are We Too Eager to Eliminate Animals in Research?

Donnell White, III, PhD Louisiana State University Health Sciences Centers New Orleans

Executive Summary

On December 29, 2022, President Joe Biden signed the FDA Modernization Act 2.0 (S.5002) into law, removing the requirement that animal studies must be done to obtain a license for biological products, such as vaccines or pharmaceutical drugs. This poses an enormous risk to human health, as there is no standardization process for these alternative models, and they have yet to meet the needs that animals provide for screening of adverse events. The overarching goal is to ensure these products are safe for human clinical trials while decreasing animal research use. I propose increasing funding to make more effective, innovative, and efficient alternative models, and raise awareness efforts to educate the general public and policymakers about the critical role that animals have and continue to play in saving millions of human lives.

Current Landscape of Pharmaceutical Testing and Public Perception of Animal Use in Science

Animals have been used in biomedical science for hundreds of years. Specifically, mice and rats are the most utilized subjects for studies investigating modernized vaccines and potential pharmaceutical candidates. Animals have been involved in scientific breakthroughs such as vaccine development, and the discovery of insulin, penicillin, cochlear implants, and inhalers. Even with the groundbreaking impact animal testing has had on human lives, animal research is a controversial and emotional topic for many. Recent statistics show that about half of the Western population favors animal testing, while the other half opposes it. Animal rights groups, such as People for the Ethical Treatment of Animals (PETA) and the Humane Society of the United States (HSUS), are dedicated to advocating for the complete opposition of animal research or demanding significant limitations when using animal models.

The FDA Modernization 2.0 Act allows alternative models to be sufficient before testing in humans, bypassing animal experiments altogether. These alternatives, called New Approach Methodologies (NAMs), aim to limit animal use by utilizing cell-based models and artificial intelligence that mimic how an organ system might respond to a bioactive compound. Data collected from NAMs are combined to help understand possible interactions between different organ types. Although this may seem a great solution to a controversial issue, significant concerns remain:

■ Obtaining adequate data to derive a conclusion from a disjointed physiological system is challenging and, in many instances, impossible.

■ Comprehensive systemic and organismic-level biology cannot be studied using current alternative models.

■ Public misinformation surrounding animal research remains prevalent, leading to social and policy barriers that harm human health. Bypassing animal testing before human trials will cause detrimental harm to human health, as alternative models are not adequate substitutions at this time.

Approach 1: Increase the quality and standardization of alternative models.

Animal testing limits unnecessary adverse events that may occur in human trials. Alternative models do not currently have the capabilities to understand how a pharmaceutical candidate might interact between organ systems in the human body. Thus, significant progress must be made to create high-quality, standardized models that can provide adequate human-like physiological data in response to biological products.

Standardized NAMs will decrease animal use in research as they can more efficiently streamline projects, resulting in only the most promising pharmaceutical candidates advancing to animal experiments.

Approach 2: Increase public awareness of animal treatment and care in research.

Support from the general public is essential, as policymakers want to ensure they receive support from their constituents regarding the policies they vote for. With the current pharmaceutical landscape, it is vital that both the general public and government officials be better informed of the contributions animals make to scientific discovery and innovation to keep animal testing legal and required for biological products.

Increasing awareness can help the general public understand the intense review processes before animal experiments to ensure they are cared for in the best way possible.

Recommendations for Policy Change

Recommendations for Approach 1: Policymakers should devote a subset of NIH-funded grants to alternative model standardization and utility.

■ Every year, millions of dollars are spent on animal care and housing at academic institutions across the United States. Animal use in screening products can be minimized by aliquoting a subset of animal care grant funding to create better physiologically replicative NAMs. This action would ensure that only the most promising developments are used for animal experiments.

■ Educating investigators via NIH-funded workshops on NAMs’ usability and protocols will increase use and awareness among investigators, ensuring that valuable funding is not spent on unnecessary animal work when NAMs would meet similar screening needs.

Recommendations for Approach 2: Public and policymakers’ perceptions of animal testing must become more positive to maintain adequate support and funding for animal research.

■ Implement an NIH-supported “Animal Research Awareness Day” for the public and policymakers, providing tours of animal research facilities and informational sessions about established organizations like the Association for Assessment and Accreditation of Laboratory Animal Care (AAALAC) and Institutional Animal Care and Use Committees (IACUC).

■ Provide and advertise government funding for organizations that find animals long-term housing after experiments are complete, ensuring the continued care of these animals after the lab. This can be accomplished through partnerships with non-profit organizations that focus on animal care post-experiments. The initial connections and partnerships required for this initiative can be created through the annual Animal Research Awareness events.

■ A subset of the current animal care funding allotment should provide the resources and training for this approach. The organization and execution of Animal Research Awareness Day should be tasked to the NIH’s Office of Laboratory Animal Welfare.

Conclusion

The safety of future patients and clinical trial subjects should be the primary concern and goal of all researchers, scientists, and advocates. Ensuring the safety of both animals and humans can only be determined with comprehensive animal studies. Although both animal advocates and scientists aim to eliminate the need for animal testing eventually, it is doubtful and unrealistic that animal models will become irrelevant for many years. However, thanks to innovative technologies and forward-thinking scientists, alternative models can provide more data than previously, allowing for advanced screening before entering animal models, reducing costs and resources, and decreasing the use of animals when not necessary.

Maternal Vaping: A Safer Alternative or a Potential Threat to Next Generation

Sabrina Rahman Archie Texas Tech University Health Sciences Center

The widespread use of electronic cigarettes or vaping among young populations including pregnant women presents a serious public health concern. This policy brief covers the popularity of maternal vaping, explains the potential health consequences of maternal vaping on pediatric health, summarizes the current policy on e-cigarette use regulation and recommends the necessary steps required for policy development to protect the next generation.

What is Vaping?

Vaping is the use of a battery-powered device, known as electronic cigarette or e-cigarette (e-cig), to inhale an aerosol derived from a heated liquid compound. It was introduced in the U.S. market in 2007 as a safer alternative to conventional tobacco smoke (TS) and has become extremely popular among all age groups and sexes. Apart from recreational purposes, it is being used as a smoking cessation and tobacco replacement therapy.

Prevalence of Maternal Vaping

E-cig use is increasingly prevalent among pregnant women, ranging between 5% and 15% of the surveyed pregnant population within the last five years. Several factors are responsible behind e-cig popularity among pregnant women, including usage out of curiosity or as a TS replacement therapy. In fact, sometimes pregnant women perceive e-cig as a safer alternative of TS and switch from tobacco smoke to e-cig during pregnancy.

Potential Health Consequences of Maternal Vaping

The main ingredient in most of the e-cig products available in the U.S. market is nicotine at various concentrations. However, a vast array of other toxic chemicals including any number of approximately 7,000 flavoring agents, humectants, contaminants, (e.g. metals, formaldehyde, acrolein, and tobacco-specific nitrosamines) as well as thousands of unknown chemicals are present in e-cig liquid. When e-cig was introduced in the U.S. market 15 years ago, there were limited preclinical and clinical studies to ensure the safety of e-cig in maternal and pediatric health. Since then, however, several studies have reported that maternal vaping may increase the risks of fetal health outcomes including but not limited to pre-term birth, reduced birth weight, cerebrovascular, respiratory, and cognitive dysfunction. These preclinical findings should raise concern among those who believe that the use of e-cig is less harmful than traditional tobacco cigarettes for pregnant smokers.

Current Policy on e-cig Use Regulation

■ Federal Regulation: The U.S. Food and Drug Administration (FDA) passed a rule in 2016 (effective from August 08, 2016) deeming that all tobacco products be brought under the purview of Federal Food, Drug, and Cosmetic Act; thus, authorized the FDA to regulate all tobacco products including e-cig to prevent a younger generation from becoming addicted to nicotine through e-cig products.

■ State Regulation: As of December 31, 2022, all 50 states have passed legislation prohibiting the sale of e-cig to underage persons, 33 states have passed legislation that requires a retail license to sell e-cig over the counter, 17 states have passed comprehensive smoke-free indoor air laws that includes e-cig and 30 states have passed legislation imposing a tax on e-cig.

Policy Recommendation on Maternal Vaping to Protect the Next Generation

As of today, there is no legislative, regulatory or policy guidance for e-cig use during pregnancy. The following recommendations for e-cig use during pregnancy can be implemented based on maternal smoking regulations and current trend of maternal vaping.

1. Public Awareness: Develop comprehensive public awareness campaigns targeting both pregnant women and healthcare professionals to build awareness about the risks of maternal vaping and promote tobacco cessation resources.

2. Regulation on Labeling: Reinforce regulatory frameworks to warrant appropriate labeling for e-cig products, including clear warnings about the potential health risks of maternal vaping on the product labeling.

3. Education and Training: Educate and train the health care professionals on the risks of maternal vaping so that they can provide accurate information, counseling, and cessation support to pregnant women.

4. Research and Surveillance: Invest in further preclinical and clinical research to elucidate the long-term health effects of maternal vaping on postnatal health and establish comprehensive surveillance systems to monitor trends and inform evidence-based interventions.

5. Affordability: Reduce the affordability of disposable e-cig products by implementing taxation to disincentivize purchasing.

6. Regulation on e-cig Advertisement: Implement stricter regulation on the advertisement and promotion of e-cig products on social media. Currently, the Advertising Standards Authority and Committee on Advertising Practice does not have rules for advertisement and promotion at point of sale, and this loophole needs to be addressed.

7. Multi-Sectoral Collaboration: Collaborate among healthcare providers, public health agencies, researchers, policymakers, and advocacy groups to develop policy and implement comprehensive strategies to control maternal vaping.

8. Industry Responsibility: Encourage e-cig manufacturers to prioritize public health by transparently disclosing ingredients, reducing nicotine content, and supporting evidence-based research on the safety of their products during pregnancy.

Conclusion

Maternal vaping may pose potential risks for maternal and fetal/postnatal health. Hence, policy development for the regulation of maternal vaping is required to address this emerging public health concern. By implementing the recommended policy guidance, policymakers can raise awareness, promote research, provide support for pregnant women; thus, can help to reduce the prevalence of maternal vaping and protect the health of neonates as well as mothers.