It’s never too early in the research and development (R&D) process to identify and measure a product’s projected clinical outcomes in order to meet the needs and expectations of payers and healthcare professionals (HCPs) who are the final arbiters of a product’s commercial success. The shift toward longer, more adequate timeframes to establish proof of concept can be as long as three to four years,1 given the complex requirements for developing and launching specialty drugs and cell and gene therapies (CGTs) for individuals with complex conditions or rare or orphan diseases.
Simply getting a patient into a physician’s practice doesn’t guarantee that a product will be utilized or paid for by health plans or self-insured companies. In fact, the introduction of CGTs heightens the need for pro-active planning and a longer runway to support optimal health plan coverage and pricing for expensive therapies as well as to prepare HCPs for prescribing decisions.
Although launching a product often feels like a long way off, given the average of 10 years to develop a potential new medicine,2 prioritizing these issues at every phase of development is key to maximizing the value creation of the pipeline asset and increasing the likelihood of success throughout the product lifecycle—from R&D and pre-commercialization phases through product launch, market access, and beyond. Consider the risk of delaying strategy development, a mistake which could result in a significant waste of time and money when the product fails to get clinician acceptance and appropriate reimbursement.
At the bare minimum, it is advisable to consider the challenges during phase one, prior to the clinical trial phase, about four to six years into development. This requires formulating and articulating a strategy, not necessarily building the actual infrastructure.
Importance of Data Collection
Many manufacturers fail to allocate sufficient time to obtain the safety and efficacy data needed to define critical endpoints. Cutting corners on data collection can be another costly error. Furthermore, leaving market access considerations until after the clinical trial phase—with the aim of launching in 12 months—is certainly the riskiest of all strategies.
While a focus on research is important, it will not be sufficient in the long term to only have clinical trial data to fall back on. The accumulation of the right data—including safety, efficacy, and outcomes—are vital to negotiations with payers as opposed to what regulators are looking for. What’s more, data collected early on in the process, such as epidemiology data, can help to inform later-stage decisions such as market strategy and pricing. The research team and its market access advisors should be tasked to compile initial thoughts on who will actually use the product, which may vary significantly from reality when regulators reduce the size of the suitable patient population or impose other constraints.
The key is to demonstrate long-term outcomes, providing real-world evidence (RWE) that supports payers with early visibility into the total health system costs related to a particular disease. This is critical because the disease burden is often not quantified for certain populations or sub-populations indicated for CGT or certain orphan drugs. Data is also needed to engage in outcomes-based contracts and establish the causal relationships between the disease and outcomes. This data will influence the adoption of value-based pricing and attract broad reimbursement.
Financial Solutions for Payers
Considerations must be given for exploring financial services that are becoming an essential component of go-to-market strategies. Many new solutions allow the high one-time costs of gene therapies and other expensive medication to be converted into small, predictable payments over time for payers. This option grows more critical as new therapies become available with price tags as high as $3.5 million for the new hemophilia gene therapy.3 As an example, one leading manufacturer is currently offering a warranty program4 that will cover the cost of any specific drug if it fails to work, marking only the second time a major pharma company has taken such a step.
Innovative copay solutions that support existing copay card programs enable manufacturers to address financial barriers of patient access. Ideally, the program should help to ensure full transparency of transactions that guard against fraud, provide complete oversight of copay fund usage, and assure that funds are safe and secure. Manufacturers should insist upon CMS-compliant programs that eliminate financial barriers to accessing treatment, with additional features such as blockchain technology to provide data security and tools that avoid “accumulators”—the coverage of two prices: the deductible and the maximum amount patients must pay out-of-pocket. These capabilities minimize risk and make certain that patients receive the full benefits of copay funds.
Securing HCP Adoption: Raising Awareness, Providing Education
It’s important to remember that HCPs typically deal with fewer specialty patients—and even fewer rare disease patients making it more challenging to diagnose rare diseases. Additional challenges include shortages of physicians specializing in rare diseases, limited facilities dedicated to rare diseases, and a lack of clinical trials for disease areas. For these reasons, it is critical to provide full support for the therapy itself by offering evidence-based information to providers and payers.
Manufacturers need to anticipate requirements and barriers for HCPs to communicate with insurance providers and external pharmacies, prioritizing a goal to streamline communications between HCP, patient, and pharmacy. For example, it is critically important that HCPs are notified if a patient is non-compliant with taking medication on schedule or as prescribed.
As prescribers, HCPs play an important role in the prior authorization process, providing vital assistance to ensure that a particular drug qualifies for coverage under the terms of the pharmacy benefit plan. Any breakdown in this communication channel seriously undermines patient access to treatment.
Value-Add Components
As manufacturers develop go-to-market strategies, they should consider some additional components that strengthen the product’s value proposition and further support its introduction to payers and HCPs to ensure patient access to care. Since manufacturers do not typically have in-house capabilities for implementing these programs, many choose to work with a single-source solutions partner that provides programs that can be customized to meet the challenges of specific product launches.
This approach eliminates the need to engage with multiple vendors in order to address and accelerate all processes, including:
- Prepare for market access strategy and payer negotiations: To ensure patient access to therapy and determine pricing and reimbursement at the time of launch, you need a team of individuals with experience, expertise, industry contacts, and a track record of success.
- Consider discontinuation of therapy metrics: Pragmatic endpoints, such as time-to-treatment discontinuation (TTD), defined as the date of starting a medication to the date of treatment discontinuation or death, has been proposed as a potential efficacy endpoint for RWE trials, where imaging evaluation is less structured and standardized.5
- Gather the right clinical data: With data such as dosing regimen, treatment duration, and route of administration you can develop a safety profile during the drug development process to optimize pricing on the day of launch.
- Build a compelling value story: Identify the unmet need, communicate the burden of the disease, and showcase the effectiveness of the therapy. Doing so requires collecting data inside and outside of the R&D timeframe or clinical trial.
- Consider patient adherence and compliance with treatment: This affects short- and long-term product utilization, impact on quality and length of life, health outcomes, and overall healthcare costs for patients who often suffer from a chronic or complex condition that needs prolonged medical care. Nonadherence can account for up to 50% of treatment failures, around 125,000 deaths, and up to 25% of hospitalizations each year in the United States.6
- Identify the patient population size: Gather specific data on the eligible patient population and benchmarked prices to create a holistic projection of the product’s durable pipeline success, number of patient lives affected, and the direct financial impact on the payer.
Preparation is key to the success of your product at launch. So don’t wait to start gathering the clinical endpoints and data you will need to help in your negotiation with payers and in providing education to HCPs about your product’s benefits. Otherwise, the years a product spent in development will be all for naught.
References:
1. Alfano, Simon; Gorham, Alex; Loche, Alberto; Salazar, Pablo. “Eight Imperatives for Launching Cell and Gene Therapies.” McKinsey & Company, September 20, 2022. https://www.mckinsey.com/industries/life-sciences/our-insights/eight-imperatives-for-launching-cell-and-gene-therapies.
2. Carroll, J.P. “How Long Does it Take to Get a Drug Approved?” Biotechnology Innovation Organization, February 16, 2021. https://www.bio.org/blogs/how-long-does-it-take-get-drug-approved.
3. Naddaf, Miryam. “$3.5-Million Hemophilia Gene Therapy Is World’s Most Expensive Drug.” Nature Magazine, December 9, 2022. https://bit.ly/3l2ImPS.
4. DeFeudis, Nicole. “Sanofi Promises Money Back for Discontinued Blood Disorder Treatment – In Some Cases.” Endpoints News, January 20, 2023. https://endpts.com/sanofi-promises-money-back-for-discontinued-blood-disorder-treatment-in-some-cases.
5. Blumenthal, G.M. et al. “Analysis of Time-to-Treatment Discontinuation of Targeted Therapy, Immunotherapy, and Chemotherapy in Clinical Trials of Patients with Non-Small-Cell Lung Cancer.” Annals of Oncology, May 2019. https://www.annalsofoncology.org/article/S0923-7534(19)31160-3/fulltext.
6. Kim, Jennifer, PharmD, BCPS, BCACP, CPP; Combs, Kelsey, PharmD; Downs, Jonathan, PharmD; Tillman III, Frank, PharmD Candidate. “Medication Adherence: The Elephant in the Room.” U.S. Pharmacist, January 19, 2018. https://bit.ly/3FcdlQm.
