Exciting Novel Therapies but With Questionable Accessibility and Affordability!
Risks and Solutions for High-cost Therapies
In the last few years, we have seen novel therapies for some complex, rare, and even previously untreatable diseases being approved – Brineura for a rare disorder of the brain, Luxturna for a specific kind of retinal dystrophy, Zolgensma for spinal muscular atrophy, etc. These drugs offer a huge potential to treat and potentially even cure diseases that previously were very difficult to crack. While such novel therapies hold a lot of promise to provide very high value to patients with complex/rare diseases, they come at an ultra-high cost.
Examples of some of the most expensive drugs:1,2
| Drug Brand Name | Cost per patient (USD) |
| Zolgensma | $2.12 million (single dose gene therapy) |
| Danyelza | Up to $2 million per year |
| Zokinvy | Up to $1.08 million per year |
| Folotyn | Avg $900,000 per year |
| Luxturna | $850,000 (1-time treatment) |
| Soliris | Up to $700,000 per year |
| Brineura | Up to $700,000 per year |
This brings us to the much-discussed question of accessibility and affordability. How many patients can really afford such ultra-expensive therapies and how many payers are willing and capable to cover such therapies?
Most insurers and employers today are not set up to take such big financial blows to their plans, especially when the therapy involves a hefty upfront payment like in case of gene and cell therapy. There are different risks and challenges associated with such high-cost therapies3, not just to payers but also patients and manufacturers:
Even for larger payers who are able to afford the high upfront payment, there are other downstream challenges like long-term patient tracking and monitoring, data collection, data analytics, etc. which could add to ancillary costs to an already expensive treatment.
To tackle the various challenges and risks of novel therapies we will need equally novel solutions in terms of financial models and programs that can help the entire healthcare system handle such extremely beneficial but high-cost therapies.
Some potential solutions that are being recommended (some are already available) in the marketplace are:
Ironing out and implementing these novel solutions would come with its own set of challenges, and would require a change in mindset for everyone involved, by brainstorming together and agreeing to contribute towards this cause. For example, it won’t be easy to come up with measurable and practical outcome measures for each applicable therapeutic area, one that all stakeholders agree with. Additionally, how would the installments be split based on milestones and who decides that.
To get a head start for finding a practical solution to such high-cost therapies is for all stakeholders (payers, manufacturers, providers, governments, patient advocacy groups/patients, etc.) to collaborate right from the development stages of the drug – perhaps manufacturers can take the lead on this. This would help achieve a few things – define outcomes that all agree would be important for the drug to meet, and look to collect them right from the clinical trial stage, ensuring there would be at least some relevant initial clinical data available to get started with.4 This would also help develop contracts and agreements better and quicker. Manufacturers could also look to experiment with new business models from their side – like mix of for-profit and non-profit models – to ensure those who are in need but cannot afford the therapy can also benefit.5
With the advent of a healthcare revolution that is finding more patient-centric, curative therapies, it is going to need dramatic changes to legacy payment models to avoid current structures enduring severe strain due to high costs. Unless all stakeholders get together and agree to work on and accept new agreements to share risks for such high-cost but potentially life-saving and life-changing therapies, it may be difficult to make them accessible and affordable to those who need them. It is time to start looking for those novel solutions that would bring in equity for such therapies.
References
- 10 of the Most Expensive Drugs in the US. https://www.drugs.com/slideshow/top-10-most-expensive-drugs-1274. Accessed in Sep 2022
- 10 most expensive drugs in the world. https://pharmaoffer.com/blog/10-most-expensive-drugs-in-the-world/. Accessed in Sep 2022
- Payer Perspectives on Reimbursement of One-Time High-Cost Durable Therapies. https://www.pharmexec.com/view/payer-perspectives-reimbursement-one-time-high-cost-durable-therapies. Accessed in Sep 2022
- Biologics and the Principles of Health Insurance: What is the purpose of health insurance? This review of basic insurance concepts provides a context for discussions about coverage of biologics. Biotechnol Healthc. 2012;9(2):14-18.
- Making Medicines Affordable. https://nap.nationalacademies.org/download/24946# Accessed in Aug 2022
15 years’ clinical research experience, including 13 years in medical writing; led multiple global medical writing projects; Authoring experience spans across regulatory writing, safety writing, and medical communication; experience of setting up robust medical writing processes.