Paradigm Biopharmaceuticals Ltd (ASX:PAR) has conducted global market research to better understand the willingness of patients to pay for and doctors to prescribe Zilosul® for osteoarthritis (OA) of the knee (kOA).
Through this research, Paradigm has determined Zilosul® has potential to become a "blockbuster therapy", dependent on variables such as patient uptake, regulatory extensions and its adoption as an early intervention therapy.
Paradigm recently received US FDA clearance to begin Phase 3 clinical trial for its proprietary drug Zilosul® – formulated from Pentosan Polysulfate Sodium (PPS) – for the treatment of pain associated with knee osteoarthritis, adding to Australian and EU approvals, and moving the company one step closer to commercialisation.
Global market research
Paradigm conducted research in four primary markets, the United States, Germany, France and the United Kingdom, to better understand willingness-to-pay and willingness-to-prescribe Zilosul® for kOA.
As is customary with market research, neither the product name nor the company involved were identified when the product characteristics of Zilosul® were presented. The research findings for the US market are presented here.
Three key pricing and uptake questions were explored by global market intelligence and research organisation Decision Resources Group (now Clarivate) via in-depth interviews with specialist physicians and public payers:
- How is the proposed product profile of Zilosul® perceived by key physicians and public payers?
- How will Zilosul® fit in the treatment algorithm and how will physicians use it?
- How much will public payers pay for Zilosul®?
Responders were asked to consider the above questions for two potential approved indication scenarios for Zilosul®:
- Reduce pain and improve function in kOA; and
- Reduce pain, improve function and prevent progression of disease in kOA.
Overall, the proposed product profile for Zilosul® was regarded positively by physicians and payers. Assuming the treatment retains its efficacy and continues to produce robust safety data, physicians and payers consider Zilosul® to provide high potential value in the treatment of kOA by covering some important unmet needs.
Specifically, Zilosul® has the potential to provide an alternative treatment to reduce pain and improve function in kOA patients, whilst preventing common side effects of currently available therapies, and potentially, preventing disease progression (subject to collection of supporting data).
Positioning of Zilosul® in the OA treatment algorithm would depend on its approved indication and funding in each market.
Broad market potential
With approval for reduction of pain and improvement in function, physicians would likely use Zilosul® as a second line therapy, that is, after nonsteroidal anti-inflammatory drugs (NSAIDS) and in-line with intra-articular therapies.
Specialist physicians estimate that up to 50% of patients would be offered treatment with Zilosul® in this scenario.
However, with a disease modifying indication, physicians would consider Zilosul® much earlier in the therapeutic algorithm. In this scenario, Zilosul® would be used in line with, or if label and funding restrictions permit, prior to NSAIDS.
Payers in the US suggested funding for Zilosul® as a therapy to reduce pain and improve function in kOA would likely be acceptable at a price of US$2,000 to US$3,000 per year of therapy.
If approved by the FDA with a disease modifying label, then price per year of therapy in the US could increase to US$6,000 and potentially higher.
Paradigm says that the results from this market research confirm the company’s earlier estimates of US$2,500 per year for Zilosul® are achievable and indicate the potential for a higher price for disease modification.
With up to 50% of patients likely to be offered Zilosul® as a therapy to manage the pain and functional impairment of kOA by specialist physicians, and even higher uptake if used as an earlier intervention for disease modification, Paradigm believes this research further confirms the blockbuster potential for Zilosul®.
Osteoarthritis as a market
OA is the most prevalent form of joint disease, affecting up to 16% of the population in the developed world, with more than 72 million people in the US, EU5 (France, Germany, Italy, Spain and the United Kingdom), Canada and Australia suffering from OA.
It has a significant impact on day-to-day functioning and, although the levels of pain and disability may fluctuate, OA has no known cure or spontaneous remission and is associated with irreversible structural damage and progression over time.
Presently, there are no drugs approved that can prevent, stop, or even restrain progression of OA. Moreover, the available medications available to mitigate the pain of OA have numerous risk/benefit considerations and market research indicates that only 19% of kOA patients are satisfied with currently available treatments.
The prevalence of OA is increasing in line with the ageing population and increasing rates of obesity. By 2030, the number of people suffering from OA in the US alone is predicted to increase by 86% to 67 million.
If we assume a similar increase across the other markets defined above, even allowing for lower rates of obesity in non-US markets, it is plausible that more than 120 million people will be suffering from OA by 2030.
The osteoarthritis treatment market was valued at approximately US$5.8 billion in 2020 and is expected to witness a revenue of US$8.2 billion by 2026, with a compounding annual growth rate (CAGR) of 5.76% over the forecast period.
Paradigm Biopharmaceuticals
Paradigm Biopharmaceuticals is a late-stage drug development company with a mission to develop and commercialise Pentosan Polysulfate Sodium (PPS) for the treatment of pain associated with musculoskeletal disorders driven by injury, inflammation, ageing, degenerative disease, infection or genetic predisposition.
Paradigm is also investigating proof-of-concept for the use of PPS in respiratory and heart failure indications.