Can you elaborate on your role as a market researcher and how you incorporate patient perspectives in your work?
Market research plays a pivotal role in generating insights for pharmaceutical drug development, particularly when it comes to therapies for rare diseases. This includes providing crucial input on various elements, such as identifying the unmet needs in the rare disease market, determining the size of these unique patient populations, understanding the competitive landscape of rare disease treatments, and assessing the market readiness for a novel product or treatment approach. Furthermore, market research can give insights into the clinical and economic value propositions that are likely to resonate with key stakeholders in the rare disease community, including patients, their families, physicians, payers, and advocacy groups. This information can be critical in shaping the drug’s value proposition, informing clinical trial design specific to the nuances of the disease, and developing appropriate pricing and reimbursement strategies.
In addition, market research empowers healthcare organizations to anticipate potential challenges and opportunities that are unique to patients with rare diseases. This is achieved by gaining a profound understanding of key opinion leaders’ perspectives, patient journey insights that consider the rare disease experience, current treatment paradigms, and barriers to adoption for these uncommon conditions. It also aids in decision-making regarding further product development, such as the necessity for additional clinical trials specific to the rare disease, or the pursuit of additional indications. The incorporation of these patient perspectives in rare diseases through market research not only humanizes the drug development process but also ensures that the resulting therapies truly address the needs and improve the quality of life of these patient populations.
What motivates you to partner with patient advocacy groups and how have these partnerships benefited the patients?
Partnerships with Patient Advocacy Groups can profoundly impact the trajectory of drug development, especially when it comes to understanding and addressing the unique needs of a patient community impacted by a rare disease that is not extensively documented in the medical literature.
For one, these partnerships provide patients and their advocates with a direct voice in the drug development process. This inclusion enables a greater understanding of the lived experiences of patients, which can help in designing more patient-centric clinical trials, thereby improving clinical trial recruitment and retention rates. Working with patients helps to identify and prioritize often missed but significant unmet needs, as well as understand the impact of new therapies on patient quality of life beyond clinical measures of efficacy.
Secondly, such partnerships foster increased transparency and trust between patients and drug developers. It helps patients to understand better the intricacies of drug development and regulatory approval processes while fostering a deeper understanding of informed decision-making and participation in clinical trials.
Finally, these collaborations can expedite the development of treatments for rare and underserved conditions. By working with patients, regulatory authorities, and policymakers, we can advocate for change and bring these life-changing treatments to patients faster.
As science makes new advancements, how do you see the future treatment landscape of rare diseases evolving and what do you think needs to be done in order to make an even greater contribution to this area?
The treatment landscape for rare diseases is likely to evolve significantly in the future, given the current momentum in scientific breakthroughs and technological advancements, I foresee two key areas where we could see rapid development.
First, the application of RNA-based therapeutics on such as antisense oligonucleotides and RNA interference techniques, which work by altering the RNA messages within cells. Products such as Nusinersen (Spinraza) and Golodirsen (Vyondys 53) have been on the market for some time now and have provided much-needed hope for Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD) patients respectively. I anticipate continued focus on similar conditions in pediatric patients with high unmet need. The use of this technology for production of major COVID vaccines was a major turning point for widespread use and experience with this technology and this is expected to give a boost to drug development using this modality.
Secondly, I see continued potential in development of products based on Chimeric Antigen Receptor T-cell (CAR-T) platform. Kymriah (tisagenlecleucel), launched in 2017 was the first product of this kind and presented a cure for pediatric B-cell precursor acute lymphoblastic leukemia (ALL). More recently, Breyanzi® (lisocabtagene maraleucel) and Abecma® (idecabtagene vicleucel) indicated for diffuse large B-cell lymphoma (DLBCL), a hard-to-treat follicular lymphoma and multiple myeloma respectively and have been promising developments.
While these developments are very encouraging, there are still significant challenges in this area and I believe the following areas should be the focus of investment and research to make a tangible difference in patient lives
One of the major challenges is diagnosing difficulties. These diseases often present themselves with nonspecific symptoms and patients may go undiagnosed or misdiagnosed for years. This makes patient identification and recruitment for clinical trials challenging and can delay access to potential treatments. There is hope though – with the large-scale use of next-generation sequencing technologies, machine learning algorithms, and big data analysis, the diagnosis accuracy can improve many folds in a short span of time and that could translate into faster clinical development timelines.
Another major challenge is regulatory hurdles. The unique nature of rare diseases does not lend itself well to the traditional regulatory approval pathways. While there are several incentives, such as those outlined by the orphan drug development guidance, navigating them can be complex.
Lastly, the biggest challenge has been around reimbursement and pricing issues. Concerns about affordability and affordable access are top of mind for most patients and caregivers. While value-based pricing of orphan drugs have met with limited success, I am excited about the potential for leveraging real-world evidence in coverage decisions.
What is a professional accomplishment that you are particularly proud of, and why?
I am very proud of my contribution to the Metastatic Breast Cancer (mBC) community. Breast cancer is the most common cancer in women in the United States, and it has now surpassed lung cancer as the most commonly diagnosed cancer worldwide. An estimated 2,261,419 women worldwide were diagnosed with breast cancer in 2020. mBC has profound impacts on the lives of women and their families. Globally, breast cancer is the fifth leading cause of death. In 2020, an estimated 684,996 women across the world succumbed to breast cancer. Metastatic breast cancer accounts for the majority of deaths from the disease. The disease progression is not only a physical challenge, characterized by varying symptoms and therapeutic side effects, but also a profound psychological, social, and economic burden. Women with mBC often experience fear, anxiety, and depression related to the uncertainty of their prognosis, changes in their physical appearance, and the potential of leaving their loved ones behind.
There have been several therapeutic advances over the past decade in the treatment of metastatic breast cancer. The development of targeted therapies, immunotherapies, and improved chemotherapy regimens have extended survival and improved the quality of life for many women with this disease. Genomic testing is increasingly being used to identify the specific characteristics of each woman’s cancer, allowing for more personalized treatment approaches. Breakthroughs in understanding the biology of breast cancer have led to the discovery of new drug targets and the development of novel therapies. Despite these advances, there is still a critical need for continued research to further improve outcomes for women with mBC.
I had an opportunity to work on the “Global Status of Advanced/Metastatic Breast Cancer 2005-2015 Decade Report,” which provides a comprehensive analysis of the mBC landscape over the past decade. This report was an international collaboration between the European School of Oncology (ESO) under the framework of the International Consensus Conference for Advanced Breast Cancer (ABC). The study aimed to highlight gaps in patient care, access to resources and support, and treatment outcomes for women around the world. The report offers a critical examination of the metastatic breast cancer (mBC) landscape, underscoring disparities in patient care, accessibility to resources and support, and treatment outcomes for women across the globe, and that’s something that I am incredibly proud of.
