The Promising Pathways Act 2.0 is a newly proposed bill that aims to expedite and streamline approval pathways for rare disease therapies in the US. The Act is, however, facing a number of challenges in terms of implementation. The sponsors of these niche therapies tend to be small and medium-sized biotech firms which are unable to compete with the financial might of Big Pharma and also struggle to deal with the myriad regulatory hurdles they need to jump over. Monica Weldon, a frequent PharmaBoardroom contributor and rare disease patient advocate, who helped craft the bill, explains more.


A Conditional Approval Pathway

In the dynamic realm of pharmaceutical innovation, the Promising Pathways Act 2.0 has emerged as a beacon of hope for patients battling rare diseases and for small biotech firms striving to introduce novel treatments to the market. This legislation seeks to streamline regulatory pathways, offering a lifeline to innovative therapies that might otherwise languish in bureaucratic hurdles. The Promising Pathway Act 2.0, reintroduced by Senator Mike Braun and a bipartisan team, aims to expedite the approval process for treatments targeting rare and life-threatening diseases, proposing a conditional approval pathway that allows for a two-year provisional approval based on early evidence of safety and efficacy. The pathway is designed to provide quicker access to potentially life-saving drugs for patients who cannot afford to wait through the traditional, lengthy FDA approval process.



The updated version addresses concerns from the original bill by narrowing the eligibility criteria and emphasizing safety and real-world data collection during the provisional approval period​. However, its progress is being hindered by formidable opposition from entrenched interests—big pharmaceutical companies, influential patient advocacy groups, and surprisingly, the FDA itself. These challenges underscore critical concerns about the potential stifling of innovation through monopolistic control and regulatory conservatism.

A primary issue at hand is the growing power wielded by larger pharmaceutical corporations over smaller biotech firms. Recent years have seen a trend where industry giants acquire promising biotech companies, consolidating drug development efforts under their expansive portfolios. This consolidation not only reduces competition but also limits the diversity of treatment options available to patients. Acquisitions such as Gilead Sciences’ purchase of Forty-Seven Inc. and Bristol-Myers Squibb’s acquisition of Celgene illustrate how smaller innovators are often absorbed into larger entities, potentially stifling independent avenues of research and development.

Moreover, these acquisitions can lead to inflated drug prices, as consolidated control allows larger pharmaceutical companies to set pricing without the competitive pressures that arise from a diverse market. This monopolistic control not only limits patient access to affordable medications but also undermines the ability of smaller biotech firms to flourish independently, thereby deterring future innovation.


Strain on Biotechs

The struggles faced by small biotech firms further illuminate these concerns. Companies like MannKind Corporation, InterMune Inc., BioMarin Pharmaceuticals, and Sarepta Therapeutics have encountered significant regulatory obstacles and financial burdens when attempting to bring groundbreaking treatments to market.

Take, for instance, Sarepta Therapeutics’ journey with Exondys 51 for Duchenne muscular dystrophy (DMD). Despite achieving conditional approval, Sarepta faced continuous scrutiny and demands for additional studies from the FDA and larger competitors. The prolonged regulatory process and financial strain jeopardized the company’s sustainability and its capacity to advance other innovative treatments. Such challenges underscore how smaller entities are disproportionately affected by the arduous and costly approval processes imposed by regulatory agencies.

In this competitive landscape, small biotech firms are often viewed as engines of innovation, driving advancements in treating rare diseases and addressing unmet medical needs. They are celebrated for their agility, creativity, and willingness to take risks in drug development. Conversely, larger pharmaceutical companies boast substantial financial resources, established infrastructure, and extensive expertise in navigating clinical development and regulatory pathways. These advantages allow them to acquire smaller biotech firms and their promising treatments, further consolidating their market dominance.


The Role of the FDA

The FDA plays a pivotal yet complex role in this ecosystem. While tasked with ensuring public health through rigorous regulatory oversight, questions arise about whether the agency’s practices inadvertently favor well-established pharmaceutical giants over smaller innovators. The lengthy approval timelines and stringent data requirements imposed by the FDA pose significant barriers to entry for smaller firms, potentially delaying or preventing the introduction of life-saving therapies to patients in need.

Opposition to legislative initiatives like the Promising Pathways Act 2.0 from the FDA and large drug companies underscores these concerns. This bipartisan legislation aims to expedite the regulatory process for drugs treating rare diseases, offering hope to small biotech firms and patients awaiting innovative treatments. Despite its potential benefits, critics cite concerns over data sufficiency and safety, arguing that opposition may serve to maintain the status quo rather than foster meaningful innovation.

From a broader perspective, resistance to legislative reforms aligns with efforts to preserve entrenched interests and market dominance. The act threatens to disrupt the current monopolistic control held by large pharmaceutical companies by empowering smaller competitors to bring treatments to market more swiftly and independently. However, while safeguarding safety standards is paramount, the FDA’s cautious approach risks perpetuating a system that favors those with significant financial resources and established infrastructures.


Levelling the Playing Field

Addressing these challenges demands a balanced approach that prioritizes transparency, fairness, and genuine competition in pharmaceutical innovation. Congressional oversight of FDA decision-making processes and a critical examination of how regulatory practices impact innovation and patient access are essential. Encouraging affected companies to share their experiences and insights can shed light on existing barriers and challenges that hinder progress.

In conclusion, fostering a regulatory environment that supports innovation and enhances patient access requires collaborative efforts across industry stakeholders, policymakers, and regulatory bodies. By advocating for legislative reforms like the Promising Pathways Act 2.0, we can strive to level the playing field, empower smaller biotech firms, and ultimately benefit patients with a broader spectrum of treatment options. The time is ripe for constructive dialogue and meaningful reform to ensure that innovation in the pharmaceutical industry serves the best interests of public health and advances medical progress.