Deeper dive: Gene therapy manufacturing
Hari Pujar, Ph.D., Chief Technology Officer, discusses the unique manufacturing and scale-up challenges involved with cell and gene therapy production.
For many of us, the term “modern medicine” is synonymous with breakthrough treatments for cancer, HIV/AIDS and other chronic diseases. We couldn’t have imagined these treatments 30 years ago and wouldn’t want to live without them today. They make it possible for people to live longer and more comfortably, with their symptoms under control, managed or diminished.
But what about the next phase of modern medicine? A world with the modern medicines we’re seeking to discover and develop at Spark Therapeutics? In this world, we aim to turn the bits and bytes of the human genome into medicine that can augment the genetic mutation that is causing disease. In the world we envision, genetic diseases have no future, and one-time gene therapies can address debilitating diseases people “just live with” or manage with chronic treatments.
It’s incredible science, but also Star Trek medicine in the age of a Flintstones reimbursement system. This next phase of modern medicine is unchartered and riddled with challenges inherent in an outdated U.S. healthcare system that is designed to support chronic treatments, with a focus on rewarding volume of care versus outcomes of care.
But certainly, where there’s a will, there’s a way.
At Spark Therapeutics, our mantra is simple: we don’t follow footsteps, we create the path. As the company that introduced the first FDA-approved gene therapy for a genetic disease, we’ve been working to create this new path brick by brick, finding solutions to old and new problems and evolving an infrastructure to embrace – and even incentivize the development of — potentially one-time, durable therapies.
To address this fundamental misalignment between what we have and what we need, urgent upgrades to the reimbursement system must be made to match the life-changing 21st century medicines we are developing, starting with a focus in two main areas:
Fully realizing the potential of one-time gene therapies starts with redefining “value” – a word that has been over used and misused by many.
For too long, we have equated better health with more healthcare. More tests, more procedures, more medicine. But this is counterintuitive to our collective goal—helping patients spend less time being tested, enduring procedures and taking medicine, and more time with the people and experiences that matter most.
Take hemophilia, for instance. It is a lifelong condition that historically requires patients to undergo weekly infusions in an attempt to prevent bleeding events. Not to mention, the hundreds of thousands of dollars spent each year, for the entirety of a patient’s life, by our healthcare system to pay for these regular infusions. If our system rewarded the quality of care versus the quantity, imagine how that could change the everyday lives for these patients?
For new, innovative therapies that may deliver long-lasting, if not lifelong, impact to succeed in the U.S. healthcare system, we must shift our focus to reward better health outcomes, not more healthcare. That suggests an ability to quickly diagnose genetic disease, using an appropriate genetic test that picks up mutations that lead to disease. That suggests treating disease at its root cause rather than treating the symptoms. That suggests a one-time intervention, rather than a life time of treatment.
Achieving better health requires a shift toward efficiency and effectiveness of medicine over quantity. We are encouraged by recent progress in this area at the state and federal levels. In fact, draft bipartisan legislation – the Patient Affordability, Value and Efficiency Act (PAVE Act) – was recently introduced in the Senate to promote the development of outcomes-based payment models for pharmaceuticals and medical services. But we need to do more and move faster.
While the recent momentum around PAVE in Washington is promising, Spark Therapeutics can’t sit back and wait for the long-standing reimbursement model to be reformed. Our goal is to ensure accessibility and accountability for our approved gene therapy by allowing payments over time and contingent on its performance.
Barriers to offering alternate payment and distribution models include the current systems’ focus on short-term value, largely because most patients switch health insurance companies on average every three years. Moreover, our government price reporting requirements are not designed to reflect one-time, up-front payments or certain outcomes-based arrangements, therefore limiting manufacturers’ ability to offer to be paid over time or significant performance-based rebates. Finally, our distribution models in the U.S. are complicated, which add costs and financial risk to parties involved in the delivery and reimbursement of specialty drugs and specialized medical care.
At Spark, we’ve had to find a way to fit our therapy’s one-time use into a single, upfront payment. To alleviate this burden and risk for insurers, we also introduced first-of-their-kind payment and distribution models for payers and patients. We’re taking both a short-term and long-term measure of our therapy’s effectiveness and will provide a partial rebate if a clinical measure isn’t met 30-90 days after treatment and again 30 to 33 months later.
Our efforts to date have helped ensure that eligible patients with commercial insurance have had out-of-pocket costs for our gene therapy and the associated administration procedure covered by our support programs. While this is a significant achievement, particularly within one year since launch, there is more to be done.
Therefore, we submitted a proposal to the Centers for Medicare & Medicaid Services (CMS), which highlighted our desire to lead the way toward a unique arrangement whereby insurers can pay for the one-time therapy in installments over time, rather than one large payment upfront, with annual payments contingent on the performance of the therapy. We think the time has come for action and to urge the government to approve this proposal, so we can begin testing payment-over-time models with a robust outcomes-based component. A program like this has the potential to enable more insurers to cover and therefore expand access to this and future gene therapies for more eligible patients.
This next phase of modern medicine is rich with promise and possibility. While the systemic changes needed in our healthcare system are significant and complex, I am confident that working together, government, policy makers, patients, insurers and industry partners can align our healthcare and reimbursement system with the 21st century medicines we are developing. If we work together, we can deliver on the true promise of modern medicine and create a world where no life is limited by genetic diseases.
Jeff Marrazzo is co-founder and CEO of Spark Therapeutics and a champion of the potential benefits of precision medicine and healthcare reform for patients. He can be found on Twitter @JeffMarrazzo.