Partners4Access: Sustainable Access for Therapies of Rare Diseases

Aparna Krishnan, Partner - Global Operations, Akshay Kumar, Partner

Rare diseases present a significant challenge to both patients and healthcare professionals. Although obtaining a proper diagnosis can take considerable time and the involvement of multiple medical practitioners, it is essential to acknowledge that progress is being made toward improving the situation.

Despite the pharmaceutical industry's reluctance to invest resources due to the limited patient pool, high development costs, regulatory hurdles, and market challenges, there are organisations such as Partners4Access that recognise the importance of addressing this issue and are committed to making a positive impact.

Partners4Access is a distinguished consulting firm that caters to clients seeking sustainable access to therapies designed for rare diseases, defined as conditions affecting less than 1 in 10,000 individuals. As one of the few firms exclusively dedicated to orphan drugs, rare diseases, and cell and gene therapies, it assists pharmaceutical companies in navigating the complex healthcare environment and identifying optimal access solutions for their products.

"Partners4Access helps its clients navigate the complex healthcare environment and identify the best access solutions for their products," says Akshay Kumar, partner at Partners4Access.
The company's services include market access strategy, value proposition development, pricing and reimbursement, health economics and outcomes research, and patient access programs. Pharmaceutical companies collaborating with Partners4Access can ensure that their rare disease treatments are not only accessible and affordable to health care systems and patients in need, but also generate a sustainable return on investment.

Partners4Access helps its clients to navigate the complex healthcare environment and to identify the best access solutions for their products

In addition to its services, Partners4Access houses a team of seasoned professionals with a deep passion for rare diseases and orphan drugs. The team comprises experts in market access, health economics, and patient access programs, drug commercialisation among other domains, and is fully equipped to address the complex needs of its clients. Overall, the firm plays a pivotal role in facilitating the availability of treatments for rare diseases, thereby significantly impacting the lives of patients who otherwise have serious debilitating, if not life threatening conditions.

Partners4Access's clients face numerous challenges when launching a drug for a rare disease. These challenges include designing studies to demonstrate that a new drug works in diseases that are not yet fully characterized, with small patient pools and where there are question marks around the right endpoints to study establish its efficacy. In addition, the rarity of the diseases poses a challenge for stakeholders, such as healthcare systems and insurers, who must assess whether the benefits these treatments offer justify the cost of treatment, especially from a broader societal perspective.
Despite these challenges, Partners4Access has a proven track record of helping its clients gain access to their products. Since the firm is dedicated to providing innovative solutions for the rare disease community, it tackles the unique challenges posed by this field.

In a recent example, Partners4Access worked closely with a pharmaceutical organisation that launched its first therapy in the rare disease phase. While the initial trial yielded positive results, regulators and payers advised a second trial of a longer duration to ensure the drug's benefits for patients is sustained over time. The Partners4Access team collaborated with the pharma organisation to investigate the reasons for stakeholder uncertainty. The team discovered that the need for more understanding of the disease and its impact on patients was the root of the problem. The company worked with senior disease area expert clinicians to build a compelling narrative around unmet needs. As more literature was published, the company was able to secure conditional approval/conditional reimbursement for the drug.

Partners4Access's specialism in rare diseases and cell and gene therapies sets it apart from other healthcare consulting firms. Its team of professionals has the expertise and experience necessary to support pharmaceutical companies in developing treatments for rare diseases and making these accessible to patients in a sustainable manner for healthcare systems and for pharmaceutical companies. With a range of services and a dedicated team, Partners4Access is wellpositioned to continue playing a pivotal role in the orphan drugs, cell & gene therapy landscape.