The Medicine of the Future is Here – And It’s Alive!


Disruptive therapies that can remedy once-incurable disorders are coming to market very soon. Big pharma will need to manage pricing resistance early on if they want to build success.

Severe Combined Immune Deficiency, colloquially referred to as SCID, is a genetic disorder in which children are born without an effective immune system. Deprived of effective treatment, most infants with the disorder do not survive past the age of two.

Hemophilia, a genetic disorder that inhibits blood clotting, is a potentially life-threatening condition with death rates nearly six times that of a healthy individual.

For years, these genetic disorders and others have plagued society, reducing the quality of life for millions and offering little hope of a long-term solution. But after more than 30 years in development, cell and gene-based therapies that can remedy disorders once thought incurable are set to enter mainstream healthcare markets in the U.S. and Europe.

The potential is massive. Johnson and Johnson, the largest pharma company in the world, recently announced its collaboration with the University of Pennsylvania’s Gene Therapy Program to combat Alzheimer’s disease. Pfizer has partnered with Sangamo Therapeutics, Inc. to develop a gene therapy to treat motor neurone disease, or ALS. Swiss pharma giants Novartis and Roche have backed gene therapy start-ups such as Vivet Therapeutics since early last year.

In principle, millions of patients stand to benefit from these types of therapy. Yet at the same time, the unfamiliarity surrounding them and the relative infancy of the clinical experience will raise questions that the healthcare sector and big pharma will need to address to overcome skepticism and, crucially, the price tag.

What Are Living Medicines?

Cell and gene-based therapies comprise a unique branch of biomedical science that seeks to target disease at a cellular or DNA level. They modify cell or gene expression with a positive therapeutic result, creating what are, for all intents and purposes, “living” medicines.

Following market approval of the first batch of cell and gene therapies (not only designated for genetic disorders, but acquired diseases as well), the healthcare sector is set to embark on an unprecedented journey into personalized medicine.

The healthcare sector is set to embark on an unprecedented journey into personalized medicine.

Consider cancer in general, and specifically, Acute Lymphocytic Leukemia (ALL). The risk for developing ALL is highest in children under the age of five, according to the American Cancer Society, who estimates that there will be 5,960 new cases in the U.S. among adults and children by the end of 2018. As cell and gene-based therapies make their way to the pharmaceutical main stage, that number could drastically decrease. Kymriah, a T-cell immunotherapy designed to treat ALL, is the first gene therapy to receive FDA approval for use in pediatric and young adult patients. That decision comes after Phase III studies showed a remission rate in relapsed leukemia patients of 83 percent — an astonishing result in a hard-to-treat blood cancer.

Communicating the Cost

Though promising, challenges abound for marketing this product and similar therapies. Communicators must do more than simply explain to the public how these new medicines work; they must also be prepared to address potential pushback on cost.

U.S. biomedical company Spark Therapeutics got this right with Luxturna, a gene therapy for a rare retinal disorder. Recognizing that the $850,000 per treatment price tag might be a fixation point for journalists, Spark built its launch story around a broad package of pricing and reimbursement options, including deferred payment plans. Additionally, Spark pointed out that these options were developed in collaboration with insurers and pharmacy benefit managers. The company also published key opinion leaders in ophthalmology to contextualise the point that a curative therapy for blindness could not be measured purely in financial terms. As a result, the media coverage was grounded and balanced.

Spark demonstrates how communicators tasked with telling the story of a cell or gene-based therapy will need to be proactive with messaging when taking their solutions to market.

Here are three best practices to follow when constructing a communications strategy for these new life-saving therapies.

  1. Speak the Language

  2. Health Economics and Outcome Research (HEOR) studies and reports detailing the positive fiscal outcomes of a medicine’s introduction are often overlooked as communication assets. At trial stage, these reports offer significant value in developing an outcomes argument. Communicators can make a case for keeping patients outside health systems by illustrating how short-term treatment from these specific therapies can lead to long-term health benefits. Therefore, a good communications strategy starts by gaining access to HEOR reports at an early stage and using them to frame the narrative.

  3. Define the R&D Spend

  4. A perennial problem for pharma has been how to justify pricing policy based on an R&D process lasting several years. Indeed, the industry has regularly faced accusations of “profit before patient.” Following the extensive early-stage price of a therapy’s initial discovery, the costs of scaling up a cell or gene therapy and generating associated viral vectors — all of which is necessary for executing a meaningful trial — are arduous. Communicators would be well advised to get ahead of these issues by working closely with market access and R&D personnel to craft a narrative that pre-empts media challenges on price.

  5. Celebrate the Science

  6. The in vivo results of gene therapy studies have been, in many cases, astounding. For example, Harvard Medical School restored full auditory function in genetically-deaf mice by activation of the TMC-1 gene. Given that it may take another decade to bring such a therapy to children, Harvard wisely communicated this breakthrough study as a celebration of its potential, while being careful not to raise false hope of early access by explaining the complex transition from animal to human studies. It still received wide coverage in popular science and health media outlets worldwide, but with an appropriate level of expectation. Harvard’s approach is a reminder that communications should begin at early stage trials — not at filing.

This could be the year that next-generation living medicines, once considered fringe, start to become part of the fabric of healthcare service delivery. Now is the time to ramp up communications strategy to be ready for it.

© Copyright 2018. The views expressed herein are those of the author(s) and not necessarily the views of FTI Consulting, Inc., its management, its subsidiaries, its affiliates, or its other professionals.
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