Market Access : Clearing the ambiguity

I must admit that during the first few months of my MSc, I was quite confused about the term "Market Access". I did know it was synonymous with translating one's skills as a health economist into practice, particularly in an industry sponsored role, but it was quite difficult to come by a definitive synopsis of what it is one actually does. What are the specific tasks and activities one is involved in? What level of complexity is involved? What skills does one need to possess? So here is my attempt at dissemination, clarification, & education. I hope it is useful to those considering it as a career and for the layperson who has mistaken it (& me) to be some sort of pharma lobbyist !

The concept of market access (in healthcare) is fundamentally similar to that in other sectors - Introduction to, and uptake by the market, of a new product offering improved value or competition to the current alternative(s). However it is the execution of this strategy which varies substantially (and requires some specialist skills). Some key questions which pose challenges - How to obtain information about target population? Which parameters of efficacy, safety, effectiveness do we measure? What competitive edge does the product hold? How to demonstrate overrall value ( to both payer & consumer)? How to negotiate in a highly restrictive regulatory environment?

Because drug development is an incredibly long process, answering these questions requires collecting data and its analysis at various stages of the products life cycle. To this effect, planning the coordination and sequential timing of activities is a core deliverable in a MA role. Pre-approval, during the clinical trial 1-3 phases, the objectives are largely to incorporate MA issues during development to create the "value proposition" and to gather data in support of  or informing product-specific decision making by upper management (i.e. invest vs disinvest, study types, trial population, approval/reimbursement strategy etc). During the phase 1/2 stage the HE/MA activities tend to focus on analysis of the disease area and associated pricing research. This includes conducting cost-of-illness studies, mapping studies, literature & systematic reviews (on outcomes/competitors disease management/policies as per discretion), and possibly even observational studies. On the pricing side, research efforts tend to be focused on assessing the payer landscape and price anchoring, providing an initial insight into the likelihood of reimbursability. Additional activities at this stage may include development of an early cost-effectiveness study, conceptual model, and patient reported outcomes (PRO) tools.

The value story of a drug starts taking shape during Phase 3 / pre-launch stage. Adjacent to maintaining up-to-date knowledge, desk research activities can now become a lot more focused on local issues and populations, and the process of dissemination through publications and scientific stakeholder or key opinion leader management begins to be emphasized. Increased use of piggy-back CE studies means that it is not uncommon to find a formal economic analysis and PRO analysis being conducted at this stage in preparation for a health technology assessment (HTA).  On the P&R front, price sensitivity studies, sequence & strategy analysis, and risk evaluations are just some of the acitivities.

Input from the MA unit is especially essential during the post approval & launch stage of the products life.  The objective now is to demonstrate the products value to payers and secure reimbursement status on formulary lists. This is most commonly done by providing the necessary evidence and data to either government agencies or independent bodies for them to conduct an HTA. A positive HTA recommendation can go a long way towards determining the success of a drug, as evidenced by the recommendations of NICE (England), SMC (Scotland), CADTH (Canada) to name a few. These dossiers need to not only present clinical evidence data, but also evidence on cost-effectiveness, comparative effectiveness and increasingly, PRO data. Since requirements can vary across geographical jurisductions, local adaptation of dossiers can be a labour intensive process. The pricing life cycle is also largely determined at this stage, as risk sharing agreements and payer negotiations occur. Reaching consensus at the optimal price - which is largely an acceptable ROI for both the manufacturer & payer - could be considered the key outcome. With eventual entry and possible acceptance of the drug, the MA professional has the on-going responsibility of collecting relevant real life effectiveness data to increase product value (and bargaining power), and updating dossiers in anticipation of HTA re-evaluation.

This is by no means an exhaustive list of activities pertainining to MA. Job descriptions can indeed vary depending on organizational capacity, and there are many soft deliverables such as scientific lobbying, patient advocacy, competitive intelligence and management of HTA decisions which need to be incorporated at various stages. However, I hope this article better illustrates the complexity and dynamic nature of MA in healthcare. If I am missing something please feel free to comment and add. I am sure input from working professionals would indeed be invaluable to newcomers in the field.

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Innovation Update

A Boost for Protein Expression:

Invitrogen has launched its GIBCO®OptiCHO Protein Expression Kit - the first kit of its kind to provide cells banked under cGMP conditions, enabling biotherapeutics manufacturers to speed regulatory submissions and time to market. The cell line development kit will provide not only the cGMP parental cells but also the components and all the protocols needed to engineer and select producing clones in a completely serum-free system. Amongst other things, cGMP requirements for production cell lines derived from parental cells include source verification, testing, history, control over cell production documentation, and ability to demonstrate chain of custody for the cells from creation through use in biotherapeutic production. Costs for testing & project management to meet these GMP guidelines can range from $30K to $50K - a significant amount for research labs.

The real kicker in this story, however, is the potentially game changing move of providing a single-fee commercial license with no royalties. Taking into account that current procurement costs for a research or commercial use license have been reported to range from $7,000 to $50,000 per year for research use licenses, and up to $500,000 per year (plus royalties on the therapeutic product) for commercial use licenses, Invitrogen’s attempt at market penetration may have a big effect on the balance sheets of cash strapped start-ups. In addition to the cost savings on licenses, royalties, documentation, & regulatory activities the GIBCO kit may also reduce productivity problems. In order to improve biosafety & regulatory approval timeline, many researchers have been removing serum and products of animal origin from cell-culture media during production of therapeutic proteins from CHO cells & using plant hydrolysates as nutrients. The drawback of using plant hydrolysates is that they often contain undefined levels of nutrients and can result in lot-to-lot variation in fed-batch performance and purification inefficiency, which are disadvantages when consistent protein yields are required. To solve this problem, the new kit makes use of chemically defined feeds which apparently not only eliminates the variability associated with using plant hydrolysates, but could also improve the productivity of biopharmaceutical protein manufacture and help move therapeutic proteins into clinical trials more rapidly.

Efficient Eye-Screening:

Using a technology originally developed at the Department of Energy's Oak Ridge National Laboratory to understand semiconductor defects, people at risk of becoming blind can now be screened for eye diseases such as diabetic retinopathy and age-related macular degeneration. The technology involves taking pictures of the retina and relaying them back to a database for comparison. Comparison with thousands of images of known retinal diseases, will determine if the patient passes the screening or is in need of follow-up care.

Manufacturers of semiconductors have used this technology for over a decade, to rapidly scan hundreds of thousands of tiny semiconductors to learn quickly about problems in the manufacturing process. "Right now, with 21 million diabetics in the United States, we need to be screening 400,000 patients for diabetic eye disease every week. Less than half of these diabetics receive the recommended annual eye exam, which is absolutely essential to minimize serious eye complications and potential blindness." says Edward Chaum, who is leading the medical portion of the project. Utilizing an automated process of this kind has the potential to not only improve ophthalmic care in rural areas but also reduce the burden on outpatient clinics & primary care physician offices in urban centre’s. The goal is to have hundreds of cameras installed in the US and beyond, for early detection of disease in a cost effective manner.

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