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The promise — and challenges — of gene therapy

Polly Webster, JD, MPH
Polly Webster, JD, MPH
Vice President, Association for Accessible Medicines

What good is a life-saving, ground-breaking treatment that patients and the health care system cannot access or afford?

This question was front of mind for me during last fall’s Brocher Foundation meeting about cell and gene therapies: cutting-edge therapies that aim to treat and potentially cure some of the world’s most devastating diseases. Meeting participants — including government, manufacturer, patient, payer, and academic representatives from around the world — agreed we are in an exciting era of biomedical innovation, with many promising therapies in the pipeline.

However, prices for these therapies are reaching astronomical levels, costing patients and our health system hundreds of thousands — sometimes millions — of dollars.

Gene therapies aim to fix the root cause of diseases by replacing, inactivating, or introducing a new or modified gene into the body. These therapies have yielded promising results, but they are still relatively new. Experts raised concerns that we lack the evidence needed to fully understand the benefits and durability of these therapies. Oftentimes, cell and gene therapies come to market on expedited approval pathways based on limited evidence of safety and effectiveness. These dynamics, combined with high price tags, create considerable challenges for affordable patient access, clinical care, and coverage of these therapies.

Reflecting on last month’s discussion, I’ve identified 3 important considerations we should be focusing on as more of these therapies come to market:

  • Pricing and payment: Innovation in this area is exciting and may be life-changing for many patients. But high prices present serious challenges, and health plans will need to consider innovative ways to provide sustainable access. While outcomes-based contracting and value-based arrangements may be helpful, there are many operational and legal barriers in the United States to making these arrangements meaningful and ensuring manufacturers bear an appropriate amount of risk. For example, can manufacturers and payers broker these contracts in light of barriers created by Medicaid best price rules? How will we identify appropriate metrics and clinical parameters to measure good outcomes? Can we collect this data in a nonburdensome way? And importantly, do we have enough evidence to support these high prices in the first place? This brings me to my second point.
  • Evidence development: As promising as these therapies are, they are still very new. We do not fully understand the clinical benefits and often do not truly know the durability of these treatments. The lack of evidence generated under the expedited pathways is complicating clinicians’ ability to tell their patients in good faith that these very expensive drugs are likely to work for them. Lack of evidence also further complicates formulary and benefit design decisions. These drugs are priced for their full potential clinical and social value even though we often lack the post-market data to prove this value. This enables manufacturers to shift risk onto payers and patients. Manufacturers can access reimbursement more quickly, and health plans are left to make complex formulary and clinical practice decisions without sufficient information. Manufacturers simply must be held more accountable for how their products perform. Further, the costs of administering some gene therapies may equal or exceed the cost of the drug itself. Thus, having information about long-term and post-market clinical value is critical to evaluating whether these drugs are appropriately priced.
  • Realities for patients: Ultimately, we must remember that patients who are candidates for these therapies are often in dire situations and understandably want — and deserve — the absolute best care available. This is creating high demand for these therapies, including from patients for whom there is very limited or no evidence that the therapy will work. Sustainable access to these therapies for patients who need them must continue to be a top priority. Yet we are currently in a situation where evidence and certainty of effectiveness is lacking, prices are astronomical, and demand is high. This puts clinicians and health systems in an incredibly challenging position, both in terms of conversations with patients and making difficult decisions about how to allocate resources.

These are pressing challenges that are shared around the world and not unique to the United States. I was encouraged, however, by last month’s conversation and believe we can and must make progress in these 3 areas so that patients have access to affordable, evidence-based treatments.

Download Drug Policy 101: Drug Therapies

Read more about cell and gene therapies — and how they differ from traditional small-molecule and biologic drugs.

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