Some Recommended Papers on Healthcare and Gene Therapy

Gene Therapy

How will gene therapy survive its success? (Kaemmerer W)

Bioengineering & Translation Med 2018

Interesting discussion of the author's hypothesis of the "hype cycle" surrounding gene therapy, and gives an overview of the major past and current hurdles facing gene therapy commercialization.

Gene therapy clinical trials worldwide to 2017: An update​ (Ginn S et al)

Journal of Gene Medicine 2018

Some numbers on gene therapy clinical development.

Advancing Gene Therapies And Curative Health Care Through Value-Based Payment Reform (Daniel G, Leschly N, Marrazzo J & McClellan M)

Health Affairs Blog 2017

Authored by blubird bio and Spark Therapeutics' CEO, this blog discusses various value-based reimbursement strategies and their potential use for gene therapies.

Gene therapy: evidence, value and affordability in the US health care system (Hampson G et al)

J. Comparative Effectiveness Research 2017

Meeting report, discusses three broad challenges facing gene therapies (evidence of efficacy, assessing value, and affordability) and suggests potential solutions as informed by KOL discussions.

GENE THERAPY: Understanding the Science, Assessing the Evidence, and Paying for Value (ICER)

ICER Report 2017

Gene therapy’s out-of-body experience (Scott C & DeFrancesco L)

Nature Biotechnology 2016

The special case of gene therapy pricing (Brennan, T. & Wilson, J.)

Nature Biotech 2014

The payers' perspective on gene therapies (Touchot, N. & Flume, M.)

Nature Biotech 2015

Gene Therapy: Twenty-First Century Medicine (Verma I & Weitzman M)

Ann. Rev. Biochemistry 2005

Financing Healthcare / Financial Engineering

Value-Based Pricing and Reimbursement in Personalised Healthcare: Introduction to the Basic Health Economics (Garrison L & Towse A)

Journal of Personalized Medicine 2017

Buying cures versus renting health: Financing health care with consumer loans (Montazerhodjat, V., Weinstock, D., and Lo, A.)

Science Translation Medicine 2016

Financing drug discovery via dynamic leverage (Montazerhodjat V, Frishkopf J, & Lo A)

Drug Discovery 2016

Financing cures in the United States (Basu A)

Exp. Rev. Pharmacoeconomics & Outcomes Research 2015

How do we value a cure? (Husereau D)

Exp. Rev. Pharmacoeconomics & Outcomes Research 2015

Specialty Medications: Traditional And Novel Tools Can Address Rising Spending On These Costly Drugs (Lotvin A, Shrank W, Singh S, Falit B, & Brennan T)

Health Affairs 2014

Despite High Costs, Specialty Drugs May Offer Value For Money Comparable To That Of Traditional Drugs (Chambers J, Thorat T, Pyo J, Chenoweth M, & Neumann P)

Health Affairs 2014

Five Features Of Value-Based Insurance Design Plans Were Associated With Higher Rates Of Medication Adherence (Choudhry N et al)

Health Affairs 2014

Establishing new payment provisions for the high cost of curing disease (Gottlieb S & Carino T)

American Enterprise Institute Blog 2014

Commercializing biomedical research through securitization techniques (Fernandez JM., Stein R. & Lo, A.)

Nature Biotechnology 2012

Leveraged Lease Financing of Capital Equipment (Stiles, N & Walker M.)

The Business Lawyer 1972

The Drug Lifecycle

Breakthrough Cancer Treatments Raise Difficult Questions (Maschke K, Gusmano M, & Solomon M)

Health Affairs 2017

The Challenge of Paying for Cost-Effective Cures (Zettler P and Brown E)

Amer. J. Managed Care 2017

Financing and Distribution of Pharmaceuticals in the United States (Dabora M, Turaga N, Schulman, K)

JAMA 2017

The Questionable Economic Case for Value-Based Drug Pricing in Market Health Systems (Pauly M)

Value in Health 2017

Financing a Cure for Diabetes in a Multipayer Environment (Basu A, Subedi P, & Kamal-Bahl S)

Value in Health 2016

Preventives Versus Treatments (Kremer M & Snyder C)

National Bureau of Economic Research 2015

Decline In Economic Returns From New Drugs Raises Questions About Sustaining Innovations (Berndt E, Nass D, Aitken M)

Health Affairs 2015

Biomedical Innovation In The Era Of Health Care Spending Constraints (Robinson J)

Health Affairs 2015

Breaking the Bank: Three Financing Models for Addressing the Drug Innovation Cost Crisis (Kleinke J & McGee N)

American Health and Drug Benefits 2015

Unintended Consequences of Expensive Cancer Therapeutics—The Pursuit of Marginal Indications and a Me-Too Mentality That Stifles Innovation and Creativity: The John Conley Lecture (Fojo T, Mailankody S, Lo A)

JAMA Otolaryngology 2014

Removing Costs From The Health Care Supply Chain: Lessons From Mass Retail (Agwunobi J & London P)

Health Affairs 2009

Economic Evaluation of a Bayesian Model to Predict Late-Phase Success of New Chemical Entities (Schachter A, Ramoni M, Baio G, Roberts T, & Finkelstein S)

Value in Health 2007

Factors affecting U.S. manufacturers' decisions to produce vaccines (Coleman M, Sangrujee N, Zhou F, and Chu S)

Health Affairs 2005

Perspectives: The Impact Of New Technology On Health Costs (Goldsmith J)

Health Affairs 1994