Gene therapy is providing unprecedented hope for growing numbers of patients and families. This game changer in medicine restores vision in babies born with congenital blindness, reconstitutes defences against infection in inherited immunodeficiencies and offers the perspective of curing the devastating neuromuscular disease, spinal muscular atrophy.
Gene therapy is also removing the need for repeat blood transfusions in adolescents with the inherited blood disorder, beta-thalassemia. Meanwhile, in oncology, CAR-T therapies, involving genetic modifications of a patients own immune cells, are proving life-saving for children or adults with certain types of blood cancers.
All these revolutionary treatments are now approved by regulatory agencies in Europe or the US. Unfortunately, they carry astronomical price tags which prevent their effective delivery to patients. As one case in point, Bluebird Bios Zynteglo for treating beta-thalassemia, has a list price of 1.57 million.
Can high prices be justified?
Gene therapy manufacturers defend their prices by pointing to high development and manufacturing costs, small markets, and unique therapeutic effectiveness as compared to the current standard of care. However, R&D costs are kept secret, and higher numbers of patients eligible for a given therapy do not translate into lower prices.
Indeed, several arguments the manufacturers put forward are dubious or even far-fetched. As of today, claims that a single administration of a gene therapy product will ensure a lifelong cure are simply not supported by the scientific evidence.
Likewise, value-based pricing is often misconceived. As stated by the US Institute for Clinical and Economic Review in its 2017 white paper on gene therapy, the established value of a treatment reflects the maximum price society might be prepared to pay for it - but should not dictate the price that is actually paid. In an ideal world, actual prices should provide market-consistent returns for shareholders and sufficient incentive to innovate.
The EU, a pioneer in gene therapy
European scientists, institutions and charities have been central to the development of gene therapy. The world's first successful clinical trial was reported in 2000 by Alain Fischer and his team at Necker Hospital in Paris, while the first authorisation of a gene therapy product in a regulated market was granted by the European Medicines Agency in 2012.
According to the Cordis database of EU-supported research, 86 gene therapy projects for rare diseases had funding from the European Commission during the FP7 (2007-2013) and Horizon 2020 (2014-2020) research programmes. One can estimate that overall more than 1 billion has been invested in this area by the EU Commission, member states and not-for-profit organisations.
To ensure European patients benefit from these achievements and investments, it is essential to ensure reasonable pricing of gene therapies. Laudable efforts are currently being made by the World Health Organization to increase transparency, and by some member states to join forces in negotiating prices, but such initiatives are unlikely to solve the current crisis as they do not address its root, namely that the sole objective of most gene therapy companies is to maximise the return on investment and shareholder value.
A way forward: enforcing the corporate social responsibility of gene therapy manufacturers
As I recently argued with Alain Fischer and the economist Mathias Dewatripont in the journal Nature Medicine (November 25, 2019), now is the time to reflect on how to enforce the corporate social responsibility of gene therapy companies.
Among the measures we would like to see considered are the insertion of clauses into technology transfer agreements made between academic organisations receiving grants from the European Commission and for-profit companies to make reasonable pricing compulsory.
We also propose to make reimbursement of gene therapies by EU healthcare payers conditional on the companies which are commercialising these products being certified for their corporate social responsibility. This is in line with several commitments made recently by pharma companies. For example, in August 2019, the CEOs of US-based pharma companies signed the Business Roundtable Statement, affirming their commitment to generate value for all their stakeholders not just their shareholders.
Also in August, Novartis announced it had joined the Value Balancing Alliance, a body whose goal is to increase transparency around business decisions, work with external bodies to develop accounting frameworks, and shift priority from profit maximisation to optimising value creation.
Earlier this year, the pharmaceutical company Chiesi was certified as a Benefit Corporation, meaning its legally defined goals include positive social impact in addition to profit.
Of course, the effective implementation of such commitments and their translation into reasonable pricing policies will require both incentives and regulatory controls. The starting point should be a renewed multi-stakeholder conversation with industry, investors, regulators, payers and, of course, patients.
Professor Michel Goldman is Co-director of the I3h Institute at the Universit Libre de Bruxelles and former Executive Director of the EU Innovative Medicines Initiative.
Read this article:
- gene therapy | Description, Uses, Examples, & Safety ... - February 24th, 2020
- Gene therapy - Mayo Clinic - February 24th, 2020
- How does gene therapy work? - Genetics Home Reference - NIH - February 24th, 2020
- Nationwide Childrens among hospitals leading the way in gene therapy - The Columbus Dispatch - February 24th, 2020
- BioMarin gene therapy won't need an AdComm as it nabs speedy FDA review - FierceBiotech - February 24th, 2020
- Gene therapy to halt rare form of sight loss - BBC News - February 24th, 2020
- With supply tight, Novartis readies gene therapy plant for production - BioPharma Dive - February 24th, 2020
- New Gene Therapies Institute Aims to Address Regulatory Barriers - HealthITAnalytics.com - February 24th, 2020
- Bluebird's gene therapy hits another delay, this time in the US - BioPharma Dive - February 24th, 2020
- First patient undergoes Luxturna gene therapy on NHS - PharmaTimes - February 24th, 2020
- First Patients Begin Gene Therapy Treatment for Blindness through NHS - Interesting Engineering - February 24th, 2020
- Spark R&D chief High exits in wake of Roche takeover - FierceBiotech - February 24th, 2020
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- FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov - February 2nd, 2020
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- Global Gene Therapy Industry Outlook 2020-2024 - Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities - P&T Community - February 2nd, 2020
- FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com - February 2nd, 2020
- Devastation, then hope: Platteville baby first in state treated for rare condition after screening - Madison.com - February 2nd, 2020
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- Gene Therapies Make it to Clinical Trials - Discover Magazine - December 31st, 2019
- Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace - December 31st, 2019
- 2019: The year gene therapy came of age - INQUIRER.net - December 31st, 2019
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- First Alzheimer's Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients - Benzinga - December 31st, 2019
- Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18 - December 31st, 2019
- Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard - December 31st, 2019
- What were the biggest biopharma stories of 2019? - MedCity News - December 31st, 2019
- Global Gene Therapy Market 2019 Revenue, Opportunity, Forecast and Value Chain 2025 - Market Research Sheets - December 31st, 2019
- Manufacturing: the next breakthrough in gene therapy - STAT - December 23rd, 2019
- Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters - December 23rd, 2019
- Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy - December 23rd, 2019
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- Making advanced therapies takes industrializing personalization - STAT - December 23rd, 2019
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- Pharma's gene and cell therapy ambitions will kick into high gear in 2020despite some major hurdles - FiercePharma - December 23rd, 2019
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- Would you pay $1M to partake in an anti-aging gene therapy trial? - Fox Business - December 13th, 2019
- Pfizer to bring gene therapy production in-house - BioPharma-Reporter.com - December 13th, 2019
- Buyer beware of this $1 million gene therapy for aging - MIT Technology Review - December 13th, 2019
- Dr. James Wilson, a scientific pioneer, on the future of gene therapy - STAT - December 13th, 2019
- The Rise Of Patent Wars In Europe's Gene Therapy Space - Law360 - December 13th, 2019
- Pfizer cites competitiveness of inhouse gene therapy - Bioprocess Insider - BioProcess Insider - December 13th, 2019
- Investment in UK cell and gene therapy manufacturing set to continue - European Pharmaceutical Review - December 13th, 2019
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- Pfizer's 'brainstorming' payment deals as gene therapies advance, exec tells Bloomberg - FiercePharma - November 13th, 2019
- Next generation cell and gene therapies: fine tuning the promise - Business Weekly - November 13th, 2019
- Vertex invests in gene therapy manufacturing - BioPharma-Reporter.com - November 13th, 2019
- Scientists are using gene therapy to treat a heart disease in dogs. Could humans be next? - 10News - November 13th, 2019
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- Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN4001, First Multigenic Investigational Therapeutic Candidate for... - November 13th, 2019
- Five benefits of gene therapies - Echo Live - September 19th, 2019
- Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy - BioBuzz - September 19th, 2019
- FDA approves 2nd gene therapy cancer drug from Durham's Precision Bio for clinical trial - WRAL Tech Wire - September 19th, 2019
- 7-Year-Old Receives New FDA-Approved Retina Gene Therapy - University of Michigan Health System News - September 19th, 2019
- First Patient Dosed in Trial of Fabry Gene Therapy Candidate FLT190 - Fabry Disease News - September 19th, 2019
- Bluebird bio reveals further encouraging data for CALD gene therapy - PMLiVE - September 19th, 2019
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