Author: Maren Nordhoy

Challenges and Concerns Raised by EFPIA on Joint Clinical Assessments

  • April 30, 2024
  • Maren Nordhoy
  • Comments Off on Challenges and Concerns Raised by EFPIA on Joint Clinical Assessments


In the healthcare regulation sphere, proposed EU-level joint clinical assessments (JCAs) have sparked debate in the pharmaceutical industry. A recent EFPIA report highlights concerns about the rigidity of the guidelines, particularly in oncology, and suggests revisions to ensure a more efficient and adaptable evaluation process that accommodates diverse stakeholder needs across Europe.

In the realm of healthcare regulation, the proposed methods for conducting EU-level joint clinical assessments (JCAs) have stirred significant debate and concern within the pharmaceutical industry. A recent report commissioned by the European Federation of Pharmaceutical Industries and Associations (EFPIA) sheds light on the potential pitfalls and challenges companies may face under the forthcoming Health Technology Assessment Regulation ((EU) 2021/2282).

The guidelines put forth for implementing these JCAs, as highlighted by EFPIA, lack the requisite flexibility and pragmatism necessary to navigate the complexities of rapidly evolving disease areas, particularly oncology. A recent study, published on March 28, scrutinized the methodology proposed by EUnetHTA21 by applying it to three anonymized oncology products with EU regulatory approval.

One of the key findings of the report reveals that the scoping process outlined in the proposed methodology results in an overwhelming number of Population, Intervention, Comparator, and Outcome (PICO) elements that companies must address. For instance, one drug subjected to analysis had a staggering 57 PICOs initially, even after consolidation, the number remained at 23. This imposes a significant burden on companies to gather and present data for each PICO, often necessitating complex analytical methods or real-world evidence from disparate sources.

Furthermore, the report underscores the challenge posed by the wide range of comparator therapies mandated by the proposed guidelines. In the context of oncology, where treatment options vary widely across EU member states, identifying appropriate comparators becomes particularly daunting. This issue is compounded by the inclusion of off-label drug use as comparators, a practice that diverges from regulatory standards and complicates the assessment process.

JCA is not the cause of differing and partly even conflicting evidence requirements across Europe. These differences existed for decades. So far, industry coped with these requirements by planning their trials for key countries (most notably the US and Japan) and then trying to meet the evidence requirements of all other countries one after another according to their position in the (European) launch sequence. What JCA changes, is that all EU requirements have to be accounted for at once within a very restricted timeframe. This puts the timeline of life-saving innovations in jeopardy and should raise serious concerns among industry, patients, physicians, regulators as well as politicians. Doing this in oncology – a field of extreme unmet need where lives of patients are at stake and where treatment options as well as evidence requirements differ strongly across the EU  – seems to be a questionable choice.

Another critical concern raised by EFPIA relates to the requirement for mature Overall Survival (OS) data, which may not be available at the time of regulatory approval for many oncology submissions. The report suggests a reconsideration of this requirement, advocating for the inclusion of intermediate outcomes such as Progression-Free Survival (PFS) in JCAs. Additionally, it highlights discrepancies between the proposed guidelines and the endpoints commonly used in national HTA assessments, further complicating the evaluation process.

EFPIA’s apprehensions regarding the JCA process echo broader industry concerns, with organizations like the Alliance for Regenerative Medicine (ARM) also cautioning against potential impediments to the implementation of the HTA regulation at a national level.

As discussions surrounding the implementation of EU-level JCAs continue, it is evident that addressing the outlined challenges and refining the proposed guidelines will be crucial to ensuring a streamlined and effective assessment process that aligns with the diverse needs of stakeholders across the European healthcare landscape.

Joint Clinical Assessment (EU HTA) Explained

  • March 11, 2024
  • Maren Nordhoy
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A Comprehensive Exploration of Competitive Intelligence, Knowledge-Sharing, and Strategic Optimization in the Evolving Landscape of EU Health Technology Assessments.

The first companies undergoing the JCA process in 2025 will indeed be pioneers, blazing a trail for others to follow. These early adopters will be the first to gain practical experience with the JCA, providing valuable insights into the challenges and opportunities associated with the process. As they navigate the complexities of the JCA, these companies will inevitably develop strategies and best practices that contribute to their success.

However, as you rightly point out, not all companies will be willing to share their experiences openly. In a competitive market environment, companies may view their knowledge of the JCA process as a valuable asset and guard it closely as a trade secret. This creates a challenge for other companies seeking to understand the key success factors and optimize their approach to the JCA.

This is where competitive intelligence plays a crucial role. Competitive intelligence involves gathering, analysing, and interpreting information about competitors, market trends, and industry developments to gain a competitive advantage. In the context of the JCA, competitive intelligence can help companies learn from the experiences of others and identify strategies and tactics that contribute to success.

By skillfully gathering information from multiple sources, including public records, regulatory filings, industry publications, and market research reports, companies can gain insights into the approaches and outcomes of their competitors undergoing the JCA process. This information can help companies identify patterns, trends, and best practices that inform their own approach to the JCA.

Moreover, competitive intelligence enables companies to benchmark their performance against industry peers and identify areas for improvement. By comparing their approach to the JCA with that of their competitors, companies can identify gaps, weaknesses, and opportunities for enhancement. This allows companies to refine their strategies, optimize their resources, and increase their chances of success in the JCA process.

In addition to competitive intelligence, collaboration and knowledge-sharing among industry stakeholders are essential for navigating the complexities of the JCA. Industry associations, trade groups, and professional networks can serve as valuable forums for sharing insights, exchanging best practices, and collectively addressing challenges associated with the JCA. By fostering a culture of collaboration and knowledge-sharing, stakeholders can collectively advance their understanding of the JCA and optimize their approach to market access within the EU.

In conclusion, the introduction of the JCA represents a paradigm shift in the evaluation of medicines within the EU. As companies embark on this new journey, they face significant challenges and uncertainties. However, by leveraging competitive intelligence and fostering collaboration among industry stakeholders, companies can gain valuable insights, identify key success factors, and optimize their approach to the JCA. In doing so, they can enhance their chances of securing timely access to innovative therapies and improving patient outcomes across the EU.

Sources:

  • Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU. EUR-Lex. https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32021R2282. Retrieved Jan 30 2024.
  • HTAR (HTA-Regulation): Implementation in Austria. Austrian Institute for Health Technology Assessment GmbH. https://aihta.at/page/htar-hta-verordnung-implementierung-in-oesterreich/en. Retrieved Jan 31 2024.
  • D4.2 Practical Guideline on Scoping Process. EUnetHTA. https://www.eunethta.eu/wp-content/uploads/2022/09/EUnetHTA-21-D4.2-practical-guideline-on-scoping-process-v1.0.pdf. Retrieved Feb 14 2024.
  • D5.1 Submission Dossier Guidance. EUnetHTA. https://www.eunethta.eu/wp-content/uploads/2022/12/EUnetHTA-21-D5.1-Submission-Dossier-Guidance-v1.0.pdf. Retrieved Feb 15, 2024.
  • EUnetHTA 21 – Stakeholder Kick Off Meeting. EUnetHTA. https://www.eunethta.eu/wp-content/uploads/2021/12/EUnetHTA-21-Stakeholder-Meeting-03.12.-FOR-WEBSITE.pdf.
  • Joint HTA Work. EUnetHTA. https://www.eunethta.eu/jointhtawork/. Retrieved Jan 30 2024
  • EU Regulation on Health Technology Assessment (HTA). MedTech Europe. https://www.medtecheurope.org/news-and-events/press/eu-regulation-on-health-technology-assessment-hta/. Retrieved Feb 14 2024
  • Proposals On EU-Wide Joint Clinical Assessments Fall Short, Warn German Industry Groups. Pink Sheet Pharma Intelligence. https://pink.pharmaintelligence.informa.com/PS147635/Proposals-On-EUWide-Joint-Clinical-Assessments-Fall-Short-Warn-German-Industry-Groups. Retrieved Feb 12 2024

Rare Disease Day: Uniting Compassion, Data, and Global Action for Accessible Healthcare

  • February 29, 2024
  • Maren Nordhoy
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What rare disease day means for our work at SAI

Every leap year, on the 29th of February, the world pauses to acknowledge Rare Disease Day—a beacon of solidarity for the millions navigating the labyrinth of rare and often isolating medical conditions. It’s a day that transcends boundaries, echoing the voices of patients, caregivers, and advocates, and igniting a flame of hope for a brighter, more inclusive future.

Rare Disease Day traces its humble beginnings back to 2008, born from the collective resolve of individuals and organizations like the European Organization for Rare Diseases and the National Alliance for Rare Diseases in the United States. Since then, it has blossomed into a global movement, weaving a tapestry of empathy and action across continents.

At its core lies an urgent call for access and affordability—a lifeline that remains elusive for far too many, especially in low- and middle-income countries (LMICs) where resources are scarce, and healthcare disparities loom large. In these corners of the world, the cost of treatment can be a barrier as daunting as the illness itself, casting shadows of uncertainty over the lives of those affected.

Consider the plight of a child living in a remote African village, battling the relentless grip of sickle cell disease—a condition that affects millions worldwide, with an estimated 75% of cases concentrated in Africa. For this child, access to modern therapies is not merely a matter of affordability but a question of accessibility. The nearest physician or hospital may be days away, diagnostics are scarce, and the infrastructure needed to administer life-saving treatments, such as cooling chains, may be non-existen

The story doesn’t end there. Across the globe, in a country revered as a workbench of pharmaceutical and vaccine production, countless individuals grapple with the harsh realities of rare diseases. In India, home to over 1.3 billion people, the burden of rare diseases is substantial, with an estimated 70 million people affected. Yet, access to adequate treatments remains a distant dream for many, despite the country’s prowess in medical innovation and production.

The statistics paint a stark picture: globally, an estimated 400 million people lack access to essential health services, while 100 million are pushed into poverty each year due to healthcare expenses. In LMICs, where healthcare infrastructure is often fragile, the disparities are even more pronounced, with rural populations bearing the brunt of inadequate access and affordability.

It’s against this backdrop that the synergies between competitive intelligence, market research, and market access/pricing become not just desirable but imperative. By harnessing the power of data and insights, we can break down barriers, build bridges, and forge pathways to health.

As we stand on the precipice of another Rare Disease Day, let us not merely mark the passage of time but seize the moment to reaffirm our commitment to compassion and solidarity. Let us be architects of change, weaving webs of connection that span continents and cultures, binding us in a shared pursuit of healing and hope.

For in the tapestry of humanity, it is the threads of empathy and understanding that lend us strength—a strength that knows no borders, no boundaries, and no bounds. And in the embrace of that strength, we find our truest humanity—the capacity to uplift, to empower, and to transform lives, one rare disease at a time.

SAI MedPartners Expands Strategic Investments: Driving Growth and Elevating Consultancy Excellence

  • July 14, 2023
  • Maren Nordhoy
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In the dynamic world of pharmaceutical services, strategic investments play a vital role in shaping the future of organizations. We are happy to announce that we have recently made headlines with our strategic acquisitions of Fulcrum Research Group (FRG) and Pharmaforce International (PFI). To shed light on the motivations behind these investments and the vision driving SAI MedPartners forward, we had the privilege of sitting down with the company’s CEO, Erik Nordhoy.

“This combination furthers our goal of building SAI into a premier global consultancy to the pharmaceutical and medical device industries. We look forward to continuing to strategically invest in resources to accelerate SAI’s growth and expand our service capabilities.” – Erik Nordhoy, CEO

Why was it the right time for SAI to acquire companies like FRG and PFI?

By 2022, I began to feel that SAI had reached an inflection point in our journey toward becoming a top tier strategic consultancy and recognized thought partner for our global pharma and biotech clients. We had grown to nearly $30 million in annual revenues and built a leadership position in the competitive intelligence field with a terrific team of over 180 professionals worldwide. While we continue to see a lot of additional running room within the core CI business, I felt that the time was right to begin exploring our strategic options to add complementary services to strengthen our value proposition. The strategic investment in SAI by Northlane Capital in June of last year gave us, for the first time, the resources to really “think bigger” about investing in our company and accelerate the broadening of our client engagement model beyond the core CI business.  

What was the strategic driver for SAI to acquire Fulcrum Research Group and Pharmaforce International?

Fulcrum Research Group fits the mold of what we were looking for in a first acquisition. The FRG brand and reputation is well established as a high-quality thought partner in the field of primary market research. The leadership team is fantastic and the company has grown its business rapidly largely through “word of mouth” and referrals based on the superb quality of its work. The primary market research that FRG provides to its clients dovetails beautifully with SAI’s CI offering. The two organizations interact with a common set of customer touch-points and are now able to provide our clients best-in-class support to understand the full external environment (market and competitors). We feel the combined value proposition is pretty compelling and customer feedback has been great so far. Beyond that, we felt that SAI’s broader client base, established global footprint and internal marketing/BD capabilities can accelerate FRG’s already impressive growth.

Pharmaforce International is the “go-to” source and industry standard for commercial resource benchmarking within the pharmaceutical and biotech industry. For nearly 20 years, clients have turned to PFI to support critical decisions regarding brand and portfolio commercial resourcing as they compete in the marketplace or evaluate requirements to launch products. With a focus on key areas like oncology and immunology, PFI has built a tremendous dataset which we believe has the potential to be built out into a broader tool with enhanced capabilities to help clients navigate the emerging commercial model in pharma. These capabilities and the core PFI syndicated offerings are very complementary to many of our existing commercial CI monitoring engagements. Also, the resources of PFI’s terrific US and EU-based research teams have the potential to significantly augment our consulting delivery capability.

How is the integration of FRG and PFI going?

Fulcrum Research Group: The integration of FRG with SAI is proceeding well with really great collaboration between the two teams and real progress being made in integrating Finance, HR, IT and other operational elements. Our plan is to maintain the FRG brand for the foreseeable future and the team will continue to support its clients as they have always done. In parallel, we will carefully evaluate opportunities for operational leverage, cross training of teams, integrated offering development and cross-selling across the organizations.

Pharmaforce International: While we are in the early day post-transaction, we have made good progress right out of the gate in aligning our go-to-market processes. Similar to the FRG situation, we will maintain the PFI brand while exploring the operational and cross-selling synergies. We now have an integrated view of the pipeline of opportunities for the combined organizations and are increasingly coordinating our business development efforts to leverage the very broad PFI client base. We have also been very pleased at early examples of how the PFI research team has jumped in and provided valuable support on a number of important SAI client engagements.

How do these acquisitions drive value for SAI’s clients?

We believe that our clients are increasingly looking for deeper and more strategic relationships with a smaller number of trusted partners to support their clinical and commercial strategy development. Particularly among the biotech community, the ability to engage more broadly and answer a wider range of fundamental business questions has a strong appeal for our clients. The integration of FRG’s best-in-class primary market research and PFI’s industry-standard commercial benchmarking offerings with SAI’s leadership position in CI positions us well as a single-source partner for critical decision inputs along the lifecycle of a brand.

What do these acquisitions mean for the future of SAI, and how will SAI’s current growth strategy develop with each acquisition?

Each acquisition represents a significant step in building out our decision support capabilities and moves us closer to our goal of becoming a fully integrated, best-in-class thought partner that supports commercial strategy and analytics for our clients. Going forward, we will continue to seek out other complementary functionalities, such as market access and regulatory consulting, that fit well with our core competitive intelligence and primary market research services.

Contact us to learn more about these strategic acquisitions and the full extent of our decision support capabilities!

The Evolving World of Social & Digital Media Intelligence

  • January 25, 2023
  • Maren Nordhoy
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It is no secret that social media is an essential channel for expressing opinions and sharing facts. Depicted through visual representations and the written word, the world works fundamentally through online channels, without which we would be thrown back into a rudimentary and non-progressive lifestyle.  

Social media has given the world a glimpse of the amazing accomplishments the healthcare industry has achieved, as well as what we can bring about in the future.  From various research on the selection of molecular candidates and all phases of clinical trials, to drug launches and promotional campaigns, the healthcare sector now heavily relies on insights from social media platforms. 

Information-sharing and peer support social media platforms have both directly and indirectly enabled the healthcare industry to improve the quality of medication and care, while aiding online communities and individuals dealing with various ailments.  PulseTraQ, a unique digital and social media intelligence, has been created to further advance the way pharmaceutical companies gather data and enhance efficiency. 

Through PulseTraQ we create topline social intelligence reports for our clients…

Here is an example of an intelligence report focused on Rheumatoid Arthritis.  Through the intelligence of PulseTraQ, our social media analysis revealed that within the last 5 years the treatment space for rheumatoid arthritis has evolved and seen improvements in terms of efficancy and reduced disease progression.  Additionally, we can conclude that although certain prescribed medications were observed in conjunction with rheumatoid arthritis, they also cater to other diseases such as Psoriatic Arthritis, Ankylosing Spondylitis, Crohn’s disease, Ulcerative Colitis, various forms of cancer, as well as other common and rare indications.

The range of benefits PulseTraQ offers continues to expand as the need for more specific social and digital intelligence analyses grows.  Through PulseTraQ, we can look at search trends for different brands, share of voice for brands and biosimilars, monthly brand mentions, and top keywords.  The rich, unprompted sentiments expressed by the patient and medical community through social media platforms acts as an invaluable source of input, to better the healthcare field. 

PulseTraQ is an indispensable tool for pharmaceutical and healthcare companies that we are excited to offer throughout the industry. A full social intelligence report includes everything from brand mentions, SERP analysis, and geographic splits, to historical analysis, top reporters’ data, and so much more.

To see a full social intelligence report and how PulseTraQ can help your strategic initiatives, download the full report below!

Download PulseTraQ White Paper

Challenges in Hemoglobinopathies Projects and the Support of KOL Match

  • September 19, 2022
  • Maren Nordhoy
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There are over 7,000 rare diseases described, affecting about 20-30 million people in the U.S. alone, according to the NIH. While each condition has its specificities, there are common challenges in the rare disease space, such as finding patients and leveraging centers of excellence and referral networks. This article discusses such challenges and how partnering with SAI’s subject matter experts can help overcome them.

Hemoglobinopathies are a family of rare diseases characterized by a defect in hemoglobin, the protein inside red blood cells that carries and delivers oxygen throughout the body. Although it is difficult to precisely determine the number of individuals affected by hemoglobinopathies, it is estimated that between 300,000 and 400,000 newborns every year have hemoglobin disorders.

While transient treatments have been the standard of care for most hemoglobinopathies, recently, gene therapy has strongly emerged as a potential curative treatment. In August 2022, the gene therapy beti-cel (Zynteglo, bluebird bio) has been approved by the FDA to treat adult and pediatric patients with transfusion-dependent beta-thalassemia. In addition, gene editing approaches (e.g., CRISPR/Cas) are invading the hematological space and can change the paradigm of disease therapeutics.

Despite the common defining characteristic of hemoglobinopathies, each condition has a unique underlying cause (i.e., specific genetic alterations). Thus, each condition affects unique populations and is supported by specialized healthcare professionals. Such differences, as well as the treatment methodology (curative vs. transient), determine the choice of specialists and patient population involved in a clinical study.

Our rare disease experts thoroughly understand the geographic distribution of hemoglobinopathies and the challenges associated with finding specialized centers and experts worldwide. At SAI, we utilize a curated list of top KOLs to find the best specialists driving innovation and treatment paradigms in hemoglobinopathies and other rare diseases.

KOL Match Rare Disease is SAI MedPartners’ subscription-based tool, which blends our extensive network of KOLs to our long-standing commitment to advancing rare disease research and development. This vetted KOL database supports our clients’ initiatives such as advisory board construction and clinical trial site selection, and it informs KOL interactions and engagement strategy.​

The unique challenges in the hemoglobinopathies space require rigorous, expert work such as our rare disease experts do every day, with the support of unique tools such as our KOL Match database. Whether you want to discuss our strategic consulting capabilities in the rare disease space or explore the breadth of KOL Match, please contact us today.

 

Sources:

Kavanagh PL, Fasipe TA, Wun T. Sickle Cell Disease: A Review. JAMA. 2022 Jul 5;328(1):57-68. doi: 10.1001/jama.2022.10233. PMID: 35788790.

Gil Bellis, Alain Parant. Beta-thalassemia in Mediterranean countries. Findings and outlook. Investigaciones Geográficas, Instituto Universitario de Geografía. Universidad de Alicante (Spain), 2021, pp.1-10. ff10.14198/INGEO.19079ff. ffhal-03447028f

Grace RF, Barcellini W. Management of pyruvate kinase deficiency in children and adults. Blood. 2020 Sep 10;136(11):1241-1249. doi: 10.1182/blood.2019000945. PMID: 32702739.

https://rarediseases.info.nih.gov/

Conference coverage with SAI: from intelligence to insight, from insight into actionable business decisions

  • August 31, 2022
  • Maren Nordhoy
  • Comments Off on Conference coverage with SAI: from intelligence to insight, from insight into actionable business decisions

Medical conference coverage requires both knowledge of the therapeutic area and the ability to synthesize key points from several individual sessions into nuanced, meaningful intelligence. At SAI, we compile the pertinent data presented at the conference, provide key takeaways from relevant presentations, and include meaningful summaries coupled with insights from KOLs and prominent experts in the field.

SAI’s therapy area experts highlight the most valuable content specific to our client’s area of interest, including updates on the current and future landscape of specific disease areas. SAI bridges the gap from intelligence to insight and insights into actionable business decisions.

Here are 10 medical conferences routinely covered by SAI which are happening this fall:

At a glance:

  1. ESMO (European Society of Medical Oncology)
    When: September 9-13, 2022
    Where: Paris, France
    Highlights: advances in antibody-drug conjugates in metastatic breast cancer, targeted treatments in colorectal carcinoma, early cancer management, cell therapies for solid tumors.
  1. EASD (European Association for the Study of Diabetes)
    When: September 19-23, 2022
    Where: Stockholm, Sweden
    Highlights: challenges and best practices in type 2 diabetes treatment, effects of SGLT2 inhibitors on the kidney, cardiovascular system, and diabetes management.
  1. ASDS (American Society for Dermatologic Surgery)
    When: October 6-10, 2022
    Where: Denver, CO
    Highlights: safe and effective combination treatments, skin cancer, regenerative medicine.
  1. ESGCT (European Society of Gene & Cell Therapy)
    When: October 11-14, 2022
    Where: Edinburgh, Scotland
    Highlights: advances in safety and effectiveness of gene delivery vectors, manufacturing and scalability challenges of gene and cell therapy products.
  1. ID Week (Infectious Disease Week)
    When: October 19-23, 2022
    Where: Washington, DC
    Highlights: Clostridium difficile bacterial infection, challenges in treating HIV patients with limited options, pediatric COVID-19 vaccines, pipeline of new antimicrobials.
  1. Congress of Clinical Rheumatology
    When: October 20-23, 2022
    Where: San Diego, CA
    Highlights: new options for treatment of systemic lupus erythematosus, the roles of IL-6 and interferons in rheumatoid arthritis, regulatory pathways in pain management.
  1. AASLD (American Association for the Study of Liver Diseases) The Liver Meeting
    When: November 4-8, 2022
    Where: Washington, DC
    Highlights: latest research on various liver disease states, patient perspectives, new developments in the space.
  1. AHA (American Heart Association) Scientific Sessions 2022
    When: November 5-7, 2022
    Where: Chicago, IL
    Highlights: focus on “improving cardiovascular health” through global education, advocating scientific discoveries, and guiding clinical practice.
    Bonus: the special focus AHA Hypertension Scientific Session takes place September 7-10, 2022, in San Diego, CA.
  1. ACAAI (American College of Allergy, Asthma & Immunology)
    When: November 10-14, 2022
    Where: Louisville, KY
    Highlights: non-Th2 asthma management, new approvals in biologics treatments, immunodeficiency beyond immunoglobulin replacement.
    Bonus: the Food Allergy Symposium will be happening parallel to ACAAI meeting.
  1. ASH (American Society of Hematology) Annual Meeting
    When: December 10-13, 2022
    Where: New Orleans, LA
    Highlights: latest scientific developments, late-breaking drug approvals, precision medicine in the hematology space.

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