Q&A with Sean Sigmon, VP of Life Sciences Business Development
The FDA’s commitment to precision medicine continues to support innovation in orphan drugs and rare disease therapeutics1. In fact, despite the challenges of the COVID-19 pandemic, the FDA approved 53 novel therapeutics in 2020, nearly 60% of which were for rare diseases2. Executing a multi-phase rare disease clinical trial program is complicated enough, yet biopharma sponsors must also proactively plan their genomic data management and community engagement strategies earlier than ever before to ensure success in a post-market environment.
Because of the increasing interest in the development of therapeutics for genetic conditions and rare diseases, we wanted to sit down with our VP of Life Sciences Business Development Sean Sigmon to get his perspective on what some forward-thinking organizations are doing to augment their go-to-market plans. We want to help others understand some of the unique approaches that can be deployed when it comes to improving post-market access for genomic-based therapies. The following questions and answers highlight some concepts we believe biopharma marketers, and commercial and market access teams should give strong consideration to.
Q1. Why is planning for post-market access important to do?
A) Developing a new pharmaceutical of any kind is filled with incredible inherent risk. Most of those risks are steeped in not producing the data to convince the FDA, CMS, and Third Party Payers (TPP) to embrace a new drug treatment and provide a means for patients to afford it. One of the core challenges is that the FDA requires a unique set of proof points to earn an indication and “cost-effectiveness” is not one of those criteria. TPP may wait to see additional Health Economic Outcomes Research (HEOR) before granting formulary status; even on a prior authorization or trial basis for a new cell & gene therapy (C>). Figuring out what data one needs to earn TPP support is critical to understand as early as possible. TPPs have their own set of data needs and it behooves every commercial marketing and market access team working in the genomic-based condition space to consider their perspective early in the strategic planning process to help ensure post-market success.
Q2. What role do genomic-informed strategies play in increasing medical market access?
A) It’s important for market access teams to understand the entire patient experience when designing their commercial strategy. In the rare disease realm, studies are often accelerated and only sometimes have a placebo arm. These elements can create doubt in the marketplace regarding a new therapy’s benefit. Also, the greater clinical community is often not as familiar with the appropriate clinical work-up for the population as rare disease specialists are, so patients may experience a diagnostic odyssey if they don’t have easy access to an institution with specialty expertise. This can be avoided with optimal engagement by all parties.
InformedDNA has a range of genomics expertise that can be leveraged to build educational content, gather real-world evidence, and facilitate testing programs in partnership with clinical, advocacy, and patient communities. The goal is to help each group fully understand how genetic testing can inform high-quality, personalized patient care that results in optimal long-term clinical outcomes and get the right testing to patients efficiently.
Q3. What conversations are health plans having when it comes to which drugs/biologics and/or genetic tests to cover for their members?
A) When it comes to implementing new initiatives, health plans often base decisions on whether the program works towards achieving the Quadruple Aim, which includes: improving the health of populations, enhancing the patient experience of care, reducing the per capita cost of health care, and improving the work life of those who deliver care3. In our experience, application of the standard clinical utility framework to genetic testing and genomic-based therapeutics has been a challenge for many health plans, as there are numerous factors that influence the utility of genetic testing and evidence is always changing or can be somewhat limited due to small population sizes. Health plans rely on a foundation of evidence-based policies and guidelines to cover high-quality care that improves health outcomes for their members. However, they also realize that there may be a slightly different framework necessary to achieve the aforementioned goals given the nature of genomics. It requires the support of genomics experts to ensure that guidelines are indeed offering coverage based on current evidence in this rapidly evolving field, but when there is supportive evidence that the intervention improves health outcomes, health plans are motivated to cover it.
Q4. What in-depth knowledge does InformedDNA have of the FDA, genetic diseases, and genetic testing technologies, that can help biotech companies gain market access while meeting a payer’s desire for longer-term proven outcomes?
A) InformedDNA has built an experienced team of genomic experts with a wide variety of competencies including genotype/phenotype database management, evidence-based policy creation, codification of policy into algorithms and automated assessments, genetic test panel optimization, procedure code identification and matching, laboratory network consultation, study protocol design, and sponsored genetic testing/registry program implementation. The core of InformedDNA’s scalable approach is the telehealth genetic counselor engagement model. Genetic counselors are well suited to develop high trust relationships with patients and can support them during longer-term research efforts required for C>.
Q5. How do you collaborate with biotech and pharmaceutical clients to instruct clinical trial protocols that ensure post-market success?
A) It’s never too early, we suggest the following:
- Pre-clinical (early) stage – Garner payer feedback to understand how they approach managing the disease state today, and how a new therapy could impact their policies. This may shine light on supplemental protocol elements to consider that may not be required by the FDA but could enhance market embrace. InformedDNA has deep expertise here.
- Late stage – Test pivotal trial protocols with payers through primary payer research and advisory boards. InformedDNA has trusted payer relationships to do this.
- Launch and post-launch stages – Engage in reimbursement conversations earlier with payers as regulatory approvals are anticipated and evidence builds. InformedDNA is a trusted advisor to many of the country’s leading health plans and can engage with key decision-makers.
- Lifecycle management stage – Ensure payers realize their expected value by presenting relevant longer-term outcomes and identifying metrics a payer may prioritize or require to target expanded use or unrestricted formulary access over time
Q6. What would your experts say is unique to building a commercialization plan for a genetic condition compared to a more common condition? What can be done to get a payer to provide support in less time?
A) To optimize ‘post-market’ success, one cannot expect that even the most sophisticated and clever marketing imagery or ad campaigns will suffice. Relying on our genomics expertise proactively can be the very ticket to gaining an earlier approval or one with fewer challenges. That’s why we offer pharma/biotech companies and research institutions InformedACCESSTM a solution that leverages our extensive payer expertise and network to support approvals:
- Maximizes the number of patients identified as appropriate to receive a novel therapy by helping to find previously underdiagnosed patients at the community level.
- Relies on our expertise in medical writing and FDA submission support.
- Utilizes our trusted, independent genomics experts to draft appropriate reimbursement policies, navigate discussions with payers and optimize coverage criteria.
- Capitalizes on the broad base of our genomics expertise to collect, analyze, and publish genotype/phenotype and patient-reported outcomes data critical to successful formulary reviews and positioning.
Q7. How should my team reach out to learn more about InformedACCESSTM
If you’d like to chat directly with Sean about how the InformedDNA team could support your commercial or expanded access program, please contact him at email@example.com. Or, feel free to fill out the form below and we’ll be in touch.
1FDA officials update on orphan drugs, gene therapies at DIA. June 18, 2020. Available at: https://www.raps.org/news-and-articles/news-articles/2020/6/fda-officials-update-on-orphan-drugs-gene-therapie Accessed September 19, 2022.
2https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2020. Accessed September 20, 2022.
3Bodenheimer, T., & Sinsky, C. (2014). From triple to quadruple aim: care of the patient requires care of the provider. The Annals of Family Medicine, 12(6), 573-576.