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Are Right-To-Try Laws A Last Hope For Dying Patients — Or A False Hope?

(Washington Post) Mar 26, 2017 - The battle over access to unapproved treatments moves from the states to Congress.

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William McGivney, PhD (Posted: March 30, 2017)

quotesThe issue of offering access to “investigational” treatments for serious and life-threatening illnesses is a very difficult issue from a clinical perspective, especially with respect to the provision of a treatment with an ill-defined adverse event profile. However we must remind ourselves that we are talking about people with serious and life-threatening illnesses such as ALS, lung cancer etc. There are ways to and examples of how the issue has been addressed. Clearly, the FDA has compassionate use guidelines. Further and even better, during the rise of the AIDS epidemic in the 1980s, the FDA produced and finalized the Treatment-IND regulation that made drugs that had shown some early efficacy and had been granted the Treatment-IND designation available to patients with AIDS before FDA approval. The road to finalization of the Treatment-IND was a controversial and a rocky path; but it happened. In my view the Treatment-IND, pushed by then Commissioner Frank Young, MD, was successful in terms of offering hope to patients, establishing a bridge to possible better drugs, and spurring on innovative research, especially the involvement of smaller biotech companies. The support of the American Medical Association was key and I was fortunate to be the FDA liaison for the AMA at the time and work with Stuart Nightingale, MD Associate Commissioner for Health Affairs at the FDA. I took the lessons from that experience with me to Aetna in 1991. Six days after joining Aetna as VP Clinical and Coverage Policy, Aetna was on “60 Minutes” for the second time for having denied coverage for high dose chemotherapy/bone marrow transplant. I was charged with addressing the issue of the use of investigational treatments in patients with less than a year to live. The bottom line was the establishment in 1991 of the Aetna “Terminal Illness Policy” whereby investigational treatments, especially HDCT/BMT, were evaluated on an individual patient/beneficiary basis for patients with limited life expectancies. The limit of life expectancy was treated with great flexibility. This program was highly successful and well-respected by patient groups, providers, other payers, employer customers, state legislators, etc. A modified version of this policy still exists at Aetna. Through it all, I learned one of the commandments for my decision-making in setting coverage policy and making individual patient medical necessity determinations at Aetna: “The more serious and life-threatening the illness, the lesser degree of certitude about efficacy and the greater risk of harm about treatments that patient, provider and payer should accept.” This real-world commandment came into decision-making play every day and still does. quotes

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