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Curative Regenerative Medicines: Preparing Health Care Systems for the Coming Wave

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Curative Regenerative Medicines: Preparing Health Care Systems for the Coming Wave ❚ REGENERATIVE MEDICINE In Vivo Pharma intelligence | Curative Regenerative Medicines: Preparing Health Care Systems For The Coming Wave We may be at the dawn of a new era of curative regenerative therapies, but their inherent nature may create barriers to adoption. The Alliance for Regenerative Medicine frames the opportunities and challenges for the industry, arguing that policy makers must begin to understand 1 the ways that these therapies represent value for money. Shutterstock: Lightspring Shutterstock: BY FARAZ ALI, TED SLOCOMB mily Whitehead was diag- disease go into complete remission. AND MICHAEL WERNER nosed with an aggressive Additional companies using similar form of cancer called acute approaches for other malignancies have More than 700 companies are working lymphoblastic leukemia reported exciting early results, prompting on new gene, cell and tissue (ALL) at the tender age of many to dare speak of a “cure” for cancer. engineering therapies that have the potential for profound and durable E5 in 2010. She had relapsed twice after In fact, when US Vice President Joe Biden responses in patients with a diverse chemotherapy and was out of options called for a “moonshot” effort to “end array of serious and costly conditions, and near death when she was treated cancer as we know it,” he did so fully many of which lack current treatments. with an experimental chimeric antigen aware of the promise of such gene and receptor T cell (CAR-T) gene therapy cell therapies already under development The health care market is grappling at Children’s Hospital of Philadelphia and rapidly approaching the marketplace. with ways to articulate and assess the (CHOP) that saved her life. Emily’s story But treatments for cancer are only the value of these potential curative was published on the front page of the tip of the iceberg. treatments, some of which may be New York Times in December 2012. We are at the cusp of a global revolu- administered only once or a few times. Three years later at the American Soci- tion in medicine. Medical researchers ety of Hematology Annual Meeting, phar- and product developers are now poised There are a number of proposed maceutical giant Novartis AG revealed to bring forward new gene, cell and tis- alternative reimbursement and that Emily was not alone. More than 90% sue engineering therapies that hold out fnancing models to address the of patients with relapsed refractory ALL the promise of profound and durable potential uncertainty and economic treated with the same CAR-T therapy – responses – often with just a single treat- disincentives that may be associated being developed in a large multi-site ment – for patients with a diverse array with curative therapies. clinical study including CHOP that is of serious and costly conditions, many of now sponsored by Novartis – had their which lack current treatments. In Vivo | November 2016 invivo.pharmamedtechbi.com invivo.pharmamedtechbi.com ❚ REGENERATIVE MEDICINE The shared mission of these tech- potential of the regenerative medicine beginning to become familiar with the nologies is to establish – or restore – the industry. (Also see “Gene Therapies: technologies involved and with the op- healthy functioning of human cells Waiting To Emerge From The Bottle” – portunities and challenges facing their in patients with cellular dysfunction. Scrip, September 6, 2016.) After decades introduction to the market. Some of the new technologies also of work – and some noteworthy early represent the highest form of personal- setbacks – the fields of cell therapy, gene The Sovaldi Efect ized medicine, requiring the treatment therapy, tissue engineering and broader One curative therapy outside the regenera- to be highly tailored and specific to the regenerative medicine are progressing tive medicine area – Sovaldi (sofosbuvir) patient’s genetic background, and often through the clinic with great promise. developed by Gilead Sciences Inc. for utilizing the patient’s own cells to create As of mid-2016, we identified more than hepatitis C – has been in global headlines the necessary therapies. The term “regen- 700 companies working on regenerative in recent years. Most of the headlines have erative medicine” also includes exciting medicines, and 728 clinical trials ongo- been negative and have focused primarily developments in the use of gene editing ing for such therapies, with 66 potential on issues of price, pharmaceutical com- technologies to replace or correct genetic therapies already in active Phase III (or pany profits, health care costs and patient material with unprecedented precision. equivalent) late-stage clinical trials, access. Noticeably less media – and po- A handful of regenerative medicine almost doubling the count over the previ- litical – attention has been devoted to the products have been approved in various ous year. (See Exhibit 2.) clinical efficacy of Sovaldi or the fact that countries around the world; many other Regenerative medicines represent as a curative therapy it may also be cost- new therapies are nearing the market, a wave of innovation coming to the effective because of the prevention of the currently in mid- to late-stage clinical shores of our health care system that costly long-term consequences associated trials. We expect that several will enter is approaching much faster than most with hepatitis C. the market within the next few years. realize. With the assistance of groups like Without debating the merits of the (See Exhibit 1.) the Alliance for Regenerative Medicine case here, the Sovaldi experience has un- These diverse examples are just a (ARM, www.alliancerm.org), policy mak- doubtedly helped pour fuel on a broader glimpse of the depth, breadth and ers, payers, physicians and patients are fire raging against biopharmaceutical Exhibit 1 2 On- Or Near-Market Regenerative Medicines COMPANY/TREATMENT INDICATION STATUS Lipoprotein lipase defciency Approved by the EMA in 2012. First approval of a gene therapy uniQure/Glybera (LPLD), a rare and ofen fatal fat using adeno-associated viruses (AAVs) metabolism disorder Approved by the FDA in 2015. First approval of a gene therapy Amgen/Imlygic Melanoma based on oncolytic virus technology Repair corneas and restore sight in Approved by the EMA in 2015. First approval of a tissue Chiesi/Holoclar patients with burns to the eyes engineered medicinal product derived from stem cells GlaxoSmithKline/ Specifc form of severe combined Approved by the EMA in 2016. First approval of a gene therapy Strimvelis immunodefciency (ADA-SCID) based on ex vivo autologous retrovirus technology Mesoblast & JCR Acute graf-versus-host disease in Pharmaceuticals Co. Ltd./ Launched in Japan Feb. 2016 children and adults TEMCELL Gene therapy using AAV for a form Spark Therapeutics/ of inherited blindness called Leber’s FDA submittal 2016; potential approval 2017 SPK-RPE65 congenital amaurosis (LCA) CAR-T cell therapy for advanced Kite Pharma/KTE-C19 BLA fling early 2017; commercial launch anticipated 2017 non-Hodgkin lymphoma CAR-T technology for relapsed/ Novartis/CTL019 FDA submittal 2016; potential approval 2017 refractory pediatric ALL SOURCE: Alliance for Regenerative Medicine In Vivo | November 2016 invivo.pharmamedtechbi.com invivo.pharmamedtechbi.com ❚ REGENERATIVE MEDICINE Exhibit 2 There have been commendable recent Gene And Cell Therapy Clinical Trials efforts by various groups to attempt to develop new frameworks to evaluate the value of biopharmaceutical products. Gene And Cellular Therapies And Other Such efforts include the American So- Regenerative Medicine Products: ciety of Clinical Oncology (ASCO) Value Framework, the Institute for Clinical CLINICAL TRIALS and Economic Review (ICER) Value Ph. I: 223 +40% Assessment Framework and the Memo- rial Sloan Kettering Cancer Center’s Ph.II: 439 of current clinical 728 DrugAbacus project, as well as numerous { Ph.III:66 trials are in oncology tools developed by for-profit firms. (Also see “Drug Pricing: With “Value” Debate In Full Swing, ICER’s Influence Grows” – In 12% Vivo, November 2, 2016.) However, even of current clinical trials these attempts have been criticized and are in cardiovascular are at odds with each other, and none of them formally address unique attributes SOURCE: Alliance for Regenerative Medicine of potentially curative therapies that should contribute to an appropriate as- sessment of their value. drug pricing and the growing costs of é How can innovators in the biopharma- We therefore lack a common vocabu- health care. In doing so, it may have also ceutical industry be better partners to lary even to begin a conversation about unintentionally tainted the optimism and the stakeholders involved? the value of curative therapies. And yet, excitement that should be attached to the it is critically important for policy makers development of curative therapies, and This article – as well as planned subse- to begin to understand all the ways in caused policy makers to fear that the new quent papers – is an early effort of ARM which potentially curative regenerative 3 era of regenerative medicine represents to help frame the opportunities and the medicine therapies may represent value an era of Sovaldi-sized problems. challenges of potentially curative gene for money. We introduce here a simple We are concerned that political rhetoric and cell therapies and to spark a con- framework with four distinct categories may turn into broad policy and legislation versation about specific policy proposals to describe the potential value of regen- that may fail to recognize the uniquely high that will benefit patients, the health care erative medicines. (See Exhibit 3.) value of curative regenerative medicines system and ultimately, society. and that may instead inadvertently create 1. Clinical Impact: In part because of the significant impediments to their funding, What Is a Pound of Cure Worth? nature of the technologies involved, development and adoption. Many stakeholders will intuitively ap- such therapies may have a transforma- Against this backdrop, it is therefore preciate the uniquely high value of tive impact on the underlying diseases critical for major stakeholders in the emer- potentially curative therapies.
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