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Experimental Drugs: an Overview Vijay Mahant* Mahant J Transl Med (2020) 18:253 https://doi.org/10.1186/s12967-020-02427-4 Journal of Translational Medicine REVIEW Open Access “Right-to-Try” experimental drugs: an overview Vijay Mahant* Abstract The “Right-to-Try” experimental drugs act passed by Donald Trump in 2018 provides an opportunity of early access to experimental drugs for the treatment of life-threatening diseases and a potential boon to many young and under-capitalized biotechnology or pharmaceutical companies. The pros and cons of experimental drugs, including a number of “cutting edge” scientifc, clinical, and a number of synergistic approaches such as artifcial intelligence, machine learning, big data, data refneries, electronic health records, data driven clinical decisions and risk mitigation are reviewed. Keywords: “Right-to-Try” experimental drugs, Precision medicine, EHR, Artifcial intelligence, Machine learning, Panomics, RWD, RWE, caTrip and caBIG Background (Erbitux), which was then undergoing clinical trial for the Over a century ago, most medications were the “wild treatment of colorectal cancer. Due to her ineligibility to west” with alleged therapeutic benefts. Most such medi- participate in the clinical trials and denial by the FDA, cations sold to the public ofered negligible or no evi- Abigail’s father, Frank Burroughs sued the FDA in 2003 dence-based therapeutic efcacy and safety to the patient for access to the experimental drug, Erbitux, on the pre- at large, except the “placebo beneft”—and hope—at the text that an investigational drug by terminally ill patients best. Te drugs were unregulated and sold to the public after phase I approval was a constitutional right. Abigail’s in the USA and many parts of the world. To protect the tragic story was one of the primary precursors and a “cat- public against the sale of misbranded, mislabeled and/ alyst” that inspired patients and non-patients, including or adulterated foods and drugs, the US in 1906 enacted advocacy groups for access to unapproved therapies by [1] the “Pure Food and Drug Act” and this resulted in the the FDA. establishment of the federal agency, the Food and Drug In May 2018, President Donald Trump signed the Administration (FDA). “Right-to-Try” Act [5]. Te legislation overcame many of the regulatory barriers, limited the risks to the spon- Historical overview sor while implementation of the act inherently burdened Te “Right-to-Try” experimental drugs, however, origi- the sponsor. Te “Right-to-Try” legislation is in essence nated [2–4] from the Abigail Alliance for Better Access a derivative of the Expanded Access Programs (EAPs). to Developmental Drugs versus von Eschenbach. Due to Advocates such as patients, families, friends and advo- Abigail’s high expression of EGFR, her oncologist rec- cacy groups of the “Right-to-Try” legislation argue that ommended Abigail for her terminal head and neck can- the legislation is in line within the pre-existing frame- cer to try an investigational EGFR-targeted drug, C222 work of EAPs and that the legislation: (i) provides a “streamlined” avenue for making eligible drugs available to eligible patients with no other options; (ii) it increases *Correspondence: [email protected] MediLite Diagnostika, Inc, 12364 Carmel Country Rd, San Diego, CA patient’s engagement; (iii) it is a patient’s journey of self- 92130, USA actualization; (iv) it empowers the patient about his or © The Author(s) 2020. This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creat iveco mmons .org/licen ses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creat iveco mmons .org/publi cdoma in/ zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. Mahant J Transl Med (2020) 18:253 Page 2 of 6 her own health, well-being and quality of life; (v) it pro- with an FDA-approved drug and appropriate tools are vides optimism and access to novel interventions with employed for monitoring efcacy and safety of the ther- potential therapeutic benefts that may prolong life and apy. Treatment of metastatic cancer patients with comor- improve quality of life; and (vi) the patient can be treated bidity using experimental drugs would be even more in the USA with valuable family time, more comfort and challenging and riskier with many implications that may fewer risks than being treated overseas. Te critics, on warrant further considerations that are in the best inter- the other hand, argue: (i) there is an inherent safety risk est of the patient’s health, well-being and life, including that may potentially cause more harm to the patient or additional fnancial burden. even death than the beneft because the experimental drug did not undergo rigorous testing; (ii) there is a lack The “landscape, the ecosystem and the dynamics” of oversight by the FDA, except posting of the consoli- Te implications of ethics, law, regulations, government dated annual summary report; (iii) the patient in most policies, constitutional rights by terminal ill patients, cases has limited understanding of the informed consent patient advocacy groups, including stakeholders about due to complexity and confusion of the medical termi- the pros and cons echoed through the “ecosystem” of nology used in the consent form; (iv) there are therapeu- early access to investigational drugs. Under the “Right- tic misconceptions combined with high expectations and to-Try” legislation, the eligibility to participate include: optimism by the patient; (v) there is potentially a con- (i) the patient must have been diagnosed with a debilitat- siderable fnancial burden by the patient or the patient’s ing or life-threatening disease; (ii) the patient must have family because payors currently do not provide cover- failed all standard of care treatments; (iii) the experi- age and deny hospice care; (vi) there is a potential loss of mental or the investigational drug must have completed trust in the regulatory agency, the sponsor and the health at least phase 1 trial; (iv) the patient must have signed care provider; and (vii) there is a liability “immunity” for an informed consent [6]; and (vi) the pharmaceuti- the health-care provider, including the drug sponsor for cal company must be able to provide the experimental potential negative outcomes of the treatment unless the drug to the patient. Due to the potentially negative out- medical provider and the sponsor were engaged in “gross come about the therapeutic efcacy combined with the negligence, reckless or “wilful misconduct.” safety issues, most sponsors developing medications for Some of the major inherent limitations and often over- life-threatening diseases have had reservations about looked about the “Right-to-Try” experimental drugs are: participating in expanded access or the “Right-to-Try” (i) patient’s vulnerability due to lack of oversight by the programs. To de-risk the potential negative outcomes FDA; (ii) there is a lack of clinical study protocol, includ- and the implications combined with an opportunity to ing the lack of sufcient statistical power to detect the target a much larger number of patients than to a few eli- intended efect(s); (iii) the information is collected in gible patients under the early access programs, the spon- a “piece-meal”; and (iv) a lack of systematic reporting sors’ primary goal has been to have full FDA approval of about efcacy and the safety of the experimental drug the drug. Te drug approval process, of course, is lengthy that may potentially result in limited information for the and highly risky due to potential clinical trial failures health and safety of the public. Some of these issues may along each step of the approval process. It is an expensive be addressed and resolved by utilizing EHR systems. EHR process—currently, estimated to be between US$ 1.9–2.5 systems are maintained by health care providers and billion [7, 8]. health care organizations for delivering patient care. EHR On the other hand, China for almost two decades systems can thus easily lend themselves for integrating approved a number of experimental drugs. From 2003 to real-time electronic health care information about the 2005, the SFDA approved H101, experimental oncolytic patient across multiple health care providers. viral therapy for head and neck cancer, an angiogenic One of the most important considerations and occa- Endostar inhibitor for treating non-small cell lung cancer sionally overlooked is comorbidity; it is quite common and Gendicine for treating head and neck cancer [9–11]. among cancer patients and it can potentially afect the However, in December, 2019, the Drug Administration treatment outcome, worsen the adverse efects due to Law (DAL) by the SFDA came into efect [12]. Te DAL polypharmacy and may even shorten the life. Te
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