In response to the myths about access to investigational drugs perpetrated by the creators of the Right to Try movement and the patient-hostile and possibly unconstitutional provisions of the laws and bills they have spawned, the NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access released positive, practical, and patient-friendly policy proposals for improving access to experimental drugs, based on three years of work with all stakeholders: patients and patient advocates, industry, healthcare providers, healthcare institutions, ethicists, legislators, and venture capital. The following is from a report recently issued by the group.
“Right to Try” legislation is based on two myths:
Myth #2: Right to Try legislation would let patients get experimental drugs faster by cutting out FDA. Reality: Only drug companies decide whether to provide their drugs to patients, and companies have many reasons to say no. No patients have gotten drugs via Right to Try that they did not have access to via FDA’s expanded access program. FDA involvement helps patients: Its objectivity and knowledge of all the various drugs in development can help patients determine their best options, and its review and its ability to require changes to treatment plans help physicians design plans most likely to help, and not hurt, patients. Right to Try could place drugs for which there is very little safety or efficacy data in the hands of doctors who are unfamiliar with the drugs; thus, despite their best intentions, doctors may have insufficient know-how to help—and not hurt—their patients. FDA’s requirement that any severe or unexpected outcomes be reported is designed to protect future patients.
More reality: “ASCO is concerned that existing and proposed RTT laws do not adequately protect patients, do little to facilitate patient access to such therapies, and potentially interfere with recent reforms that are already streamlining patients’ access to investigational agents.” —American Society of Clinical Oncology position paper, April 2017
“Any legislation should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients.” —Pharmaceutical Research and Manufacturers of America (PhRMA) spokesperson (RAPS.org, 2/17/17)
Real, workable, proposals to help patients get access to unapproved treatments:
1. To help patients get access to experimental drugs, they, and their doctors, must understand how to request access. Then, such access must be made appealing to and practical for drug companies.
2. Strengthen FDA’s expanded access program. It works, but it can be improved.
3. Preserve FDA’s role in reviewing expanded access proposals. This protects patients from charlatans and improves the proposals by adding FDA’s expert input.
4. FDA’s Office of Health and Constituent Affairs and the Division of Drug Information help physicians and patients with expanded access. They need more resources, autonomy, and/or personnel to function optimally.
5. Require FDA to expand its current Guidance Document on Expanded Access to include clear, specific information about what, if any, the consequences would be of an unexpected or severe adverse event occurring in the context of expanded access.
6. Strengthen FDA’s ability to require physicians to report serious and unexpected adverse events.
7. FDA must prioritize the Reagan-Udall Foundation’s in-development expanded access navigator, which will help physicians and patients who seek access to experimental drugs. It must be easy-to-use, up-to-date, accurate, appropriately designed and built from the outset, and properly maintained if it is to help patients.
8. Use PDUFA to require FDA to develop an outreach plan to address fears and misperceptions about expanded access for industry, advocacy organizations, patients and the public.
9. Use PDUFA to require FDA to better educate healthcare professionals and researchers on the expanded access process and reporting requirements.
10. Encourage FDA to work with industry to investigate how data from expanded access can serve to advance drug development and supplement clinical trial data.
11. The 34 state Right to Try laws have created confusion for healthcare professionals and organizations about whether to follow state or federal rules governing access to experimental drugs outside of clinical trials. Congress must clarify that, according to precedent, FDA has authority over non-approved drugs.
12. Explore, with the pharmaceutical and biotech industries, ways to make expanded access a more appealing prospect to companies large and small.
13. Companies are concerned that expanded access may threaten their drug development timeline due to FDA or Wall Street concerns. Legislators and industry must partner to mitigate risks to drug development and companies.
14. Some companies, particularly small ones, may be unable to provide experimental drugs to patients even if they wish to. They may lack the human and financial resources to create a sufficient supply of drug and/or handle the requirements of an expanded access program, such as monitoring the proper use of the drugs and overseeing the required reporting. Legislators and industry must work together to make these activities financially feasible for companies.
15. Companies are concerned about possible liability for adverse outcomes resulting from an experimental drug. Legislators and industry must create solutions for this concern. We suggest considering the possibility of immunity only when three conditions are met: the FDA expanded access pathway is used; sellers and prescribers act with a reasonable belief that the treatment is in the patient’s best interests; the patient or his legal representative provides informed consent.
16. Commission an expert group to explore whether institutional review board (IRB) oversight is needed for single-patient expanded access.
17. Utilize legislative and other means to promote awareness of, access to, and equity in clinical trials — the safest, most valuable, and most common way for patients to get access to an experimental drug.
Lisa Kearns, MS, is a senior research associate at the NYU School of Medicine’s Division of Medical Ethics and a member of its Working Group on Compassionate Use and Pre-Approval Access.
The views expressed by this author are their own and are not the views of The Hill.