Panel 5: Patient Support Programs
Panel Description: This session will help participants develop a better understanding of why PSPs are well-suited to support the development of outcomes-based agreements. It will suggest effective ways that PSPs can be developed to support data collection for HTA. Finally, current pCPA perspectives on PSPs and the opportunity for data collection within programs will be explored by this panel.
Moderator: Allison Wills
Panellists: Sang Mi Lee, Greg Rutherford, and Janey Shin
Allison Wills: I am here to moderate this panel on patient support programs (PSPs) and exploring opportunities for outcomes-based agreements (OBAs) in Canada. We are going to try and give you perspectives around how PSPs can be leveraged to explore OBAs.
What is an OBA? It is an agreement between a manufacturer and a payer in which the manufacturer will issue a refund or rebate based on how the drug performs in the real world. These agreements are still in the early days in Canada. There are major challenges to overcome such as the fact that we are a fractured system and there are resource challenges in terms of implementation. So how can we look at implementing OBAs and what are the ideal processes?
It is no secret that specialty drugs are leading the market and there is complexity in delivering them to patients. By 2020 they could make up 50% of the market in terms of expenditure. It is the fastest growing category of drugs in Canada. With specialty drugs, there are typically corresponding PSPs. Specialty drugs require many services that are needed by patients to access the drug which are not covered by the public or private systems – such as reimbursement support, access to infusion clinics or injection training. Manufacturers have filled this gap by sponsoring patient support programs. There has been some controversy with PSPs regarding the benefit to patients and the “marketing” benefit that manufacturers receive – but I challenge you to think about the investment that manufacturers have made, and what would happen to specialty drugs in the Canadian market without it. For the immediate future, PSPs are here to stay: as long as there are gaps in coverage, they will need to be filled to ensure patients can access specialty medications.
The Canadian PSPs ecosystem is well suited to support OBAs. When a specialty drug has a PSP it usually means there is a concentrated distribution which in turn supports the opportunity for data collection. For example, there is one pharmacy location managing 95% of a specialty drug for an entire province, so with this distribution model, this is a significant amount of data.
At present, most PSPs in Canada are focused on access to drug and reimbursement, and have an operational value – and are not set up to support data collection for payer usage. However, PSPs can be innovated to generate this data. We need to tap into the strategic value of these programs but there are a lot of challenges to get from level 1 to level 4 (strategic value). We need to work together and collaborate.
So the first question is for Sang Mi, what is currently happening with OBAs in Canada and how are PSPs being leveraged?
Sang Mi Lee: There isn’t a statement or policy on how we [the pCPA] utilize PSPs for OBAs, so the following are my thoughts and comments.
I like the term performance-based agreements over OBAs. We have to be careful using the term managed access agreement because some countries use this term to refer to volume-based agreements.
PSPs could have a big role in data collection. We have 198 LOIs and only have a handful of performance-based agreements. It is only in the last year or so that we started looking at them more often with a more proactive approach. If you look at how PSPs can be leveraged, we have one agreement currently in place.
We have to look at how they are being run and designed. Right now, payers are not involved in the design of PSPs and they are not set up to be utilized for performance-based agreements. There is a lot of potential, and we are looking at it more.
Allison Wills: For many years manufacturers have sponsored these programs and in what ways are manufacturers looking at PSP infrastructure and performance-based agreements?
Janey Shin: There has not been great use of PSP data to date, but there are studies that show that patients do better in PSPs. So, the question is how do we leverage this data?
To respond to Sang Mi who asked how do payers know if the quality of data is good? I think there needs to be collaboration and willingness to share data to maximize patient outcomes between the manufacturers and the payers. If we could link PSP and other data sources this could be a powerful tool to measure outcomes. ISPOR provided guidelines on how this could be done in a structured way. So the next question is what are the critical elements we need to measure? Also, this needs to be addressed early on to allow manufacturers to put the infrastructure in place and establish data elements. For us, it’s about collaboration and having an understanding of payers’ needs in order to maximize potential.
Allison Wills: Is it a little bit of the chicken and the egg?
Janey Shin: I think we need to have those conversations to know what will satisfy the payer. It’s a dialogue that needs to occur from the offset.
Sang Mi Lee: In an ideal world, you have a Health Canada approved drug with NOC/C and then pCODR recommends collecting real-world evidence and identifies that this drug would be fit for an OBA. We would then work with the manufacturer to design the PSP to make sure all the questions are answered, and an agreement would be in place shortly after HTA.
Allison Wills: Question for Greg: one of the logistical challenges is the data and the cost of collection. What opportunities exist currently to support data collection, and do you have recommendations for manufacturers?
Greg Rutherford: More often than not we bring in a PSP at launch to expedite reimbursement and maximize access. Now data has gone way past the early days when the data reporting was solely metrics-related to the program.
If you think of PSPs now, they provide the opportunity for the drug to be expedited to the right patient audience. It is about patient capture and puts in place measures to improve adherence. It drives revenue from a manufacturer perspective and presents the opportunity to collect longitudinal data. If at launch you think of the outcomes you want to collect, you can start collecting right at the point of enrollment. For example, will we see similar outcomes from the clinical trial in the real world?
Involving an adherence tool early on will help keep patients on the drug long enough to measure an outcome. If you consider more of a closed loop distribution model, while it is more of an expense, you have an opportunity to stay with the patient longer and data is more readily available. You can put all these outcomes on a digitized platform that allows you to retain data and facilitate data sharing.
Allison Wills: Let’s bring it back to OBAs. The pCPA has considered OBAs, so can you share any challenges and what brings about success in those negotiations?
Sang Mi Lee: There was a question around who owns the data, and can we publish on this data? Especially if we have a world where there is disinvestment it would be important for us to be able to publish that the real world outcome to certain drugs was poor, requiring disinvestment. If you want to move forward with these agreements, then the manufacturer should be willing to let us publish and we will let the world know it didn’t work.
Another challenge is that you cannot mandate that the patient goes into the PSP, so for those who may not be a part of the program, how will they be followed if the patient collection is a part of the funding agreement?
Other challenges; with complex agreements, it takes more time and as we do more we will get faster, but it requires a new type of expertise and resources for these kinds of agreements. Also currently PSPs rarely collect outcome data or don’t have proper patient consent to share/ use data even for research purposes etc. Lastly, the manufacturer has to convince us that the PSP can be used for this and that there is a firewall between the manufacturer and that data. How will the provinces get access to this data and how can they validate it? There are a lot of operational questions.
Allison Wills: Can we ask Janey to build on that?
Janey Shin: I think there are a lot of opportunities for the manufacturer to address the concerns the pCPA may have regarding the validity of the data. We ensure there is strong governance around how research and data collection is conducted. It is designed by the groups involved in the partnerships, it is not driven by the manufacturer.
It can be published and reported. Manufacturers are willing to report, but we need to be at the table when these discussions are being had otherwise we are going to think that pCPA is coming up with rules on how RWE is used. As long as the right data and analytic plan are in place there is a willingness to share data to the broader health system.
Allison Wills: Given where we are today, what do you think is the one greatest operational opportunity and one challenge in leveraging PSPs?
Greg Rutherford: We are going to see more diagnostic programs that share a universal platform which will allow for extrapolation of individual brand data. There is an opportunity to share data and take advantage of separate programs on that universal platform.
Janey Shin: Greatest opportunity is that PSPs do fill that gap in the system and they help patients manage their disease. We are seeing through some of CADTH’s recommendations that they are asking manufacturers to provide PSPs. In terms of challenges, the proposed PMPRB regulations will have a huge impact in this industry and how manufacturers will invest in these agreements. We need to work in a supportive policy environment. The other challenge is around data. How do we operationalize it in an OBA?
Sang Mi Lee: The greatest challenge is the capacity and the resources given the volumes we have at the pCPA. It takes time to think outside the box. Folks in the UK have said their patient access schemes are a huge commitment in terms of resources, planning and expertise. There is a lot of uncertainty, but it is an opportunity to utilize the changes to create something that is better and more efficient.