With over 2 million groups where professionals from various industries network, stay updated with the latest news, and collect feedback from their audience, LinkedIn is the ultimate B2B platform; call it “the place to be” for pharma.
Considering the vital role Linkedin groups play in pharma and the growing attention GT therapies get from the scientific community and the pharma industry, we ran a poll during the summer of 2022 in the “Gene, Cell and RNA therapy Network” group to uncover Gene Therapy (GT) challenges faced by pharma companies. The group is already counting almost 14K members, and it serves as a networking hub for professionals working in the fields of Gene / Cell / RNA Therapy.
GT promises to treat severe diseases from a wide range of therapy areas like cancer, disorders of the CNS, and musculoskeletal disorders through the three following ways:
- Replacement of disease-causing gene
- Inactivation of the disease-causing gene
- Introduction of new/modified gene in the body
Numbers around GT investments, approvals and launches are indicative of the growth the space is experiencing. In 2021, Novartis invested $800 million upfront to acquire access to Gene therapy for an age-related illness that can cause blindness. About 27 Cell and Gene therapy products had received FDA approval as of December 2022. According to forecasts, there will be more than 50 in-vivo and ex-vivo launches in GT in the future.
After this brief space overview, it is time to present the poll findings, initiate the discussion around the GT challenges, and suggest ways to overcome them.
4 Main Gene Therapy challenges unveiled and explained
Manufacturing and supply chain challenges are at least four times more likely to be an issue for pharma than other types of challenges. According to the LinkedIn poll results in which 60 people participated, the most common challenge for Gene and Cell therapy had to do with the manufacturing process and supply chain (63%), followed by the small amount of clinical data (15%), reimbursement issues (13%), and others (8%). Comments below the poll showed that the regulatory framework is a common challenge, and we will discuss it as well.
- Manufacturing process / supply chain challenges cause production cycle backlogs.
The GT manufacturing process and supply chain are unique and quite different from conventional therapies; starting material is different every time, making the operation time-consuming and expensive.
Backlogs are frequent when production cycles are established to create products for early clinical studies. Several production cycles, taking up to 2 weeks or more from receipt of initial materials, are often necessary to manufacture multiple batches of drug material and final product for readily available Cell and Gene therapies.
These manufacturing runs also come with a built-in risk of batches not meeting specifications or being lost owing to sterility problems; these risks translate into more processing steps and/or manual handling.
- Limited clinical data raise ethical and safety issues.
Cell and Gene therapies are frequently used to treat rare and orphan diseases with small patient populations. With conventional study designs, recruiting enough patients for trials to establish the safety profiles of innovative treatment platforms is difficult. Due to the low statistical power brought on by the few patients available to form treatment cohorts, it becomes challenging to determine dose-response curves. Similarly, while dealing with life-threatening conditions, traditional randomized controlled studies would not be appropriate or ethically acceptable.
- Reimbursement challenges hamper patient access to Gene Therapy.
The high cost of Cell and Gene therapy is one of the biggest obstacles to their widespread adoption, despite the fact that they can treat currently incurable diseases. Inadequately defined diagnosis codes and limited data in the field of GT make it even more difficult for payers and providers to identify the right patients. Many current reimbursement methods need to be properly designed to cover expensive therapies.
- The lack of harmonized regulations puts pressure on sponsors.
Cell and Gene therapies still need to be explored, so there need to be harmonized regulations, clear manufacturing guidance, and GMP standards. Gene treatments frequently target rare diseases and are subject to accelerated regulatory protocols, which can put sponsors under tremendous time strain. This requires doing tasks twice as quickly with only half the expertise.
How to address Gene Therapy challenges
Below we discuss ways to overcome the challenges pharma companies face regarding GT therapies as they were revealed from the LinkedIn poll.
The solution to the manufacturing process and supply chain challenges which was the most common answer (63%) in the LinkedIn poll, is to ensure the following:
- Early collaboration with material suppliers to guarantee consistency, availability, and quality of the products,
- Automation and standardization of manufacturing process early in the development cycle employing available solutions such Milenyi Biotec. Milenyi Biotec works in the design, development, manufacture, and integration of research and clinical-grade products, supporting the advancement of various Cell and Gene therapies.
- Shift to in-house manufacturing processes to avoid dependency on CDMOs as a result of long term outsourcing.
Addressing the second most common challenge (15%) regarding patient recruitment for trials in rare and orphan diseases means that:
- Pharma companies in the Cell and Gene therapy space and regulatory bodies should work closely to address clinical data challenges.
- Sponsors should opt for basket-type clinical trial designs that simultaneously cover and evaluate multiple indications in early-phase safety studies, facilitating the clinical trial enrollment process and expanding the number of eligible participants.
- Consulting firms and organizations have the expertise to provide clinical research services and support sponsors. Accenture Life Sciences provides a cloud solution, “Intient Clinical” for accelerating clinical trials to improve visibility into clinical studies performance and speed up the trials’ process with real-time understanding of patients, identification, and engagement. CMed understands the particularities of Advanced Therapy Medicinal Product (ATMP) trials and manages complex clinical trials related to Cell and Gene therapy overcoming the associated operational challenges.
According to the poll results, the third most common challenge (13%) was reimbursement issues. This challenge can be addressed by:
- Focusing on therapy areas where unmet needs call for long-term, continuing care.
- Utilizing health economics to manage payer objections and negotiate with them win-win value-based long-term agreements tailored to the specific characteristics of the product and the market. For example, Medicare for inpatient care uses Medicare Severity Diagnosis related groups (MS-DRGs), and hospitals receive a single lump-sum payment per discharge (the amount increases if the patient has certain comorbid conditions or develops complications). Outlier payments for patients receiving CGTs could also be made in addition to MS-DRG and New technology add-on payment (NTAP). Historical coding and Medicare payment for KYMRIAH and YESCARTA over the years since launch, all claims eligible for Outlier. FormularyDecisions provides qualified payer access to product information and access to health economic information on thousands of pharmaceutical products. It is also home to reports generated by the Institute for Clinical and Economic Review (ICER). ICER Reports assist decision-makers in determining the cost-effectiveness of products in specific disease states as compared to similar products.
As we mentioned above, regulatory challenges were mentioned in the comments below the poll. To address these challenges, companies need to advocate for global standardization and assurance in the regulation of Cell & Gene therapy products. Regulatory bodies are willing to facilitate Cell & Gene Therapy development, approval and monitoring. That is why engaging with regulators early and maintaining relationships with them throughout the whole process is advisable.
Overall, the LinkedIn poll gave us a sneak peek into the serious Gene Therapy challenges pharma faces. The 60 respondents agreed that manufacturing and supply chain challenges are the most common ones (63%), while the small amount of clinical data and reimbursement issues are concerns for 15% and 13% of the respondents respectively. 8% of the respondents chose “other,” and a couple of comments mentioned regulatory challenges.
Though the sample of respondents was small and hence not representative, we can see how social media play a significant role in identifying industry challenges. Therefore, Pharma companies should use social media to network and collaborate with companies who offer social media intelligence to know their stakeholders’ pain points.
Concerning Gene Therapies, addressing all the above challenges is a “need of the hour” and the pharma industry must take a holistic approach to their development, regulation, and delivery. This requires collaboration from all stakeholders, from the pharma industry and government regulators to patient advocacy groups, to ensure that these treatments are developed, approved, and available to those who need them.
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