The Be The Match BioTherapies team is back in Minnesota following a busy week at the J.P. Morgan Healthcare Conference and Biotech Showcase in San Francisco.
The highlight of the week was the Alliance for Regenerative Medicine’s Cell & Gene Therapy State of the Industry Briefing, where the organization’s CEO, Janet Lynch Lambert, kicked off the meeting with a discussion of the challenges and opportunities facing the cell and gene therapy sector. She highlighted the many achievements made by companies in the space last year, including the approvals of the first CAR-T therapies and Spark’s gene therapy Luxturna, but noted that the continued success of the industry depends on solving important challenges related to manufacturing, reimbursement and the regulatory process.
Throughout the week, conversations repeatedly returned to these topics in one-on-one meetings, lunchtime discussion panels and in chatter overheard in the crowded hotel lobbies.
Reimbursement challenges: Now that the dust has settled on the recent approvals of the first cell and gene therapies, the next challenge is bringing these drugs to patients. One key hurdle? Navigating the complex reimbursement environment. Last month, Bloomberg reported that two months after the approval of Gilead’s Yescarta, just five people had received the treatment, a holdup attributed to the complexity of developing billing codes for the treatments. As Medicare, Medicaid and large American health insurance payers work to determine a path forward, waiting lists for the treatments are swelling, with as many as 200 cancer patients hoping to receive the treatment before their cancer progresses further.
Meanwhile, Spark Therapeutics has announced pricing for Luxturna, its one-time treatment for biallelic RPE65 mutation-associated retinal dystrophies, some of which can cause permanent blindness. The $850,000 price tag on the first-ever gene therapy makes it the most expensive drug sold in the U.S., and has reignited debates regarding several core reimbursement issues – including the high costs associated with R&D at rare disease focused companies, general company profitability and the societal benefits that come from curing rare diseases. In the wake of Spark’s announcement, the non-profit Institute for Clinical Evaluation and Review (ICER), released a report arguing that Luxturna is far too expensive, while Steve Miller, the Chief Medical Officer of Express Scripts, which has historically been critical of companies that charge high prices for their drugs, called Spark’s pricing plan “reasonable” and “responsible”. We’ll be watching this story closely along with the rest of the cell and gene therapy sector, as Spark’s experience in the year to come will no doubt influence the next wave of gene therapy companies as they prepare to bring their drugs to market.
The evolving regulatory landscape: Among the challenges of bringing a brand-new class of medicines to market is navigating the regulatory landscape. Novel approaches come with a slew of new regulatory risks and uncertainties. In the case of cell therapies and other regenerative medicines, the industry lacks an established model for assessing these unique products, which are created by manipulating patients’ own cells and cannot easily be compared to existing therapies. We enjoyed Labiotech’s summary of the regulatory challenges that European and American biotech companies face in developing CAR-T and other regenerative therapies.
The globalization of the cell & gene therapy market: We were thrilled to see just how many countries were represented at J.P. Morgan. It is clear that cellular therapies are advancing across the globe, which we’ve witnessed firsthand throughout the last 30 years, as our parent company, the National Marrow Donor Program®/Be The Match®, has been delivering cell therapies to patients in need, both domestically and internationally. The cell therapy space in Europe has been developing in parallel to the U.S. for many years, but research is now accelerating in other nations as well. No country outside of the U.S. had a bigger presence at J.P. Morgan than China, which, following significant government investments in infrastructure and research has witnessed explosive growth in its biotech sector over the past several years. As The New York Times reported in a recent story on the burgeoning Chinese biotech sector, Chinese biotech and pharma companies aim to play a larger role in the global drug market, and many are setting their sights on their “ultimate goal” – U.S. regulatory approval.