Finding treatment solutions where there are none
It is no secret that the current global pharmaceutical pipeline is focused on developing treatments to combat cancer. The reasons behind this are multiple, with a greater return on investment for companies in this therapeutic area and rising incidence for health care systems to treat. It also is an area of particular concern for the public, which is reflected in media coverage.
However, a Deloitte report on R&D notes that the greater return on investment for oncology has resulted in a much greater emphasis on developing drug candidates in this area compared to others. In its cohort study, Deloitte found that 39% of pipelines were formed of oncology treatments in 2018, compared with 18% in 2010.
The same study found that treatments for infectious diseases shrank over this period, moving from approximately 10% of pipelines to 6%.
Finding new partners
At BIO-Europe Spring, a panel discussion centred upon how this imbalance could be readdressed and how those working in the area could achieve success, despite fewer funding opportunities.
Michael Crowley, head of business development for pharma research and early development at Roche, suggested that the key to being successful in the area, even for a company as large as Roche, lay in finding the correct partners.
Crowley told attendees, “At Roche, we're invested in infectious diseases but the business model is challenging. So, reaching out to government organisations and other kinds of partners to help co-fund new antibiotics development or to be creative with the regulatory pathways is essential.”
To a certain extent, the prediction that Crowley made following this statement, that he expects to see changes in the business and regulatory environment due to partnerships between the industry, regulators, and NGOs, has already come true.
This kind of conversation led to the UK government earlier this year announcing that it would explore new payment models based on how ‘valuable’ the medicines are to the UK’s health care system, rather than by volume.
David Rossow, founding partner of the strategic investment fund at the Bill and Melinda Gates Foundation, which has become a high-profile partner for industry with its ‘venture philanthropy’ model, stated that its work researching the area of infectious diseases, alongside partners, had changed its investment strategy.
“As we learned more about the epidemiology of children that died between zero and 28 days, we recognised that we were focused on children older than 28 days that were exhibiting symptoms – while children in the former group go largely unreported and that is a huge part of the patient population, and this has informed our product development,” Rossow explained.
He furthered that this is part of being “along for the ride” with partners – being able to see the opportunities, both from an investment and development perspective.
Looking to the future
Crowley highlighted that although infectious diseases is one underserved area, rare diseases actually see a huge number of patients without treatment options across a number of different disease areas.
Outlining the statistics, he said, “There are 7,000 rare diseases and only 200 of those have any kind of treatment at all. There are 350 million people living with rare diseases worldwide.”
This is a specific area where Crowley believes partnerships can have an influence, specifically between regulators and industry.
He provided one example that may speed up access, by suggesting: “Perhaps allowing for gene therapy vectors that have been tested and proved in other diseases could be tested in other patients, such as in rare diseases.”
Dissimilar to some other underserved therapeutic areas, the financial incentive and return on investment in the rare disease area is clear – resulting in more treatments in the pipeline and a prediction that the overall market could reach $262bn (€233.4bn) by 2024, an Evaluate report suggested.